Yuta Lee, CEO

April 9 | 1:45pm | Salone dei Cavalieri, Section 2

Philadelphia, PA

(Private)

Accelerated Biosciences is a regenerative medicine company with freedom-to-operate for the human Trophoblast Stem Cell (hTSC) platform with 49 patents granted. We are providing GMP cells with a complete regulatory package to help cell and gene therapy developers accelerate their product time to market by 3 to 5 years with a potential $10M in savings. We have effectively de-risked the starting cell source decision for the regenerative medicine industry. We are available for partnering and licensing opportunities.

www.acceleratedbio.com

Andrew Knudten, CEO 

April 11 | 9:00am | Salone dei Cavalieri, Section 2 

Durham, North Carolina 

(Private) 

Advanced Medicine Partners delivers what patients deserve and what regulators expect.

ampgtx.com 

Laurent Fischer, M.D., CEO

April 9 | 1:45pm | Salone dei Cavalieri, Section 3&4

Redwood City. CA

(NASDAQ: ADVM)

Adverum Biotechnologies is a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases with the aspiration of developing functional cures to restore vision and prevent blindness. Leveraging the capabilities of its proprietary intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians’ offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ixoberogene soroparvovec (Ixo-vec, formerly referred to as ADVM-022), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. Additionally, by overcoming the challenges associated with current treatment paradigms for debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe.

www.adverum.com

Tom Lynch, Ph.D., Director, Technical Sales & Scientific Support

April 10 | 10:00am | Salone dei Cavalieri, Section 2 

Fargo, ND

(NASDAQ: DHR)

Aldevron serves the biotechnology industry with custom production of nucleic acids and proteins. Thousands of clients use Aldevron-produced plasmids, RNA and gene editing enzymes for projects ranging from research grade to clinical trials to commercial applications. Aldevron specializes in cGMP manufacturing and is known for inventing the GMP-Source® quality system. Aldevron operates the largest cGMP plasmid DNA manufacturing facility in the world at company headquarters in Fargo, North Dakota, with additional facilities in Madison, Wisconsin, and Lincoln, Nebraska. Aldevron is proud to be part of Danaher. Danaher’s science and technology leadership puts Aldevron’s solutions at the forefront of the industry, so they can reach more people. Being part of Danaher means we can offer unparalleled breadth and depth of expertise and solutions to our customers. Together with Danaher’s other businesses across Biotechnology, Diagnostics and Life Sciences, we unlock the transformative potential of cutting-edge science and technology to improve billions of lives every day.

www.aldevron.com

Harald Stover, Founder and CEO, Allarta Life Science Inc., Professor of Chemistry and Chemical Biology, McMaster University

April 10 | 11:15am | Salone dei Cavalieri, Section 2

Hamilton, Canada

(Private)

Allarta is a pre-clinical company developing cell therapeutics for people with chronic endocrine disorders, combining 30+ years foundational polymer science with next-gentherapeutic cells. Allarta’s hydrogels are shape-agnostic, high surface area constructs that offer durable immune-privileged encapsulation of therapeutic cells. They are manufactured using commercially available 3D printers and enable minimally invasive delivery. Our robust hydrogels show visible blood vessels early post-implant and have high integrity and retrievability one-year post-implant. Allarta’s hydrogels are compatible with genetically modified stem cells, including suicide gene technologies, offering high cell containment and minimal fibrosis up to 95 days post-implant. For our lead indication, Type I Diabetes, our hydrogels show rapid and sustained blood glucose control in immunocompetent rodents using donor islet xenografts without immunosuppression, and human C-peptide expression in large animals. We currently aim for extended rat and large animal studies, and clinical translation, both with donor-derived and stem cell-derived islets.

www.Allarta.com

Mark Farmery, Ph.D., Chief Development Officer 

April 10 | 2:15pm | Salone dei Cavalieri, Section 2

Stockholm, Sweden 

(Private) 

Anocca is a rapidly growing Swedish biotechnology company dedicated to decoding the complexity of the human immune system to recode powerful T cell immunotherapies (TCR-T). Our cell-based Discovery Engine pushes the frontiers of T-cell biology to expand the scope and scale of TCR-T delivering libraries of medicines that can have a significant impact on patients with cancer. To date, Anocca has generated more than 40 preclinical TCR-T assets against high value cancer targets. Our cell engineering, molecular and genetic tools, automation, and software technologies analyze the complex interactions between real immune cells to identify biologically relevant disease targets and safe, potent TCR-Ts at an industrial scale (the platform can also be leveraged for vaccine design and autoimmune research). A first clinical trial of several KRAS TCR-Ts for hard-to-treat solid tumor cancers and manufactured in-house will begin in 2024.

www.anocca.com

Marco Alessandrini, Ph.D., CEO 

April 11 | 9:45am | Salone dei Cavalieri, Section 2 

Geneva, Switzerland 

(Private) 

Antion Biosciences SA is a Swiss-based biotechnology company developing universal cell and gene therapy products for the treatment of diseases with significant unmet medical needs. Antion’s proprietary gene silencing and co-expression gene constructs allow for a modular and tunable approach to multiplex engineering of therapeutic cells. By developing off-the-shelf therapies with enhanced potency, our vision is to make cell and gene therapies universal and broaden access to these life-changing therapies for all patients.

www.antionbio.com 

Devyn Smith, Ph.D., CEO 

April 9 | 2:00pm | Salone dei Cavalieri, Section 2

Cambridge, MA 

(Private) 

Arbor Biotechnologies is a next-generation gene editing company focused on discovering and developing potentially curative genomic medicines, with the most extensive toolbox of proprietary genomic editors in the industry to date. Founded by Feng Zhang, David Walt, David Scott, and Winston Yan, Arbor’s proprietary discovery engine is focused on discovering and evolving technologies that enable gene knockout, RT editing, precise excisions and large insertions into endogenous loci. Leveraging our wholly owned nucleases as the chassis for genetic modification, Arbor has the unique ability to work backward from disease pathology to choose the optimal editors or combination of editors for the disease in question, with a focus on areas of high unmet need. As Arbor continues to advance its pipeline toward the clinic with an initial focus in liver and CNS disease, the Company has also secured several partnerships around gene editing and ex vivo cell therapy programs to broaden the reach of its novel nuclease technology.

www.arbor.bio

Tamer Mohamed, President and CEO 

April 9 | 9:45am | Salone dei Cavalieri, Section 2

Vancouver, Canada 

(Private) 

Aspect Biosystems is a biotechnology company pioneering the development of bioprinted tissue therapeutics to transform the treatment of currently incurable diseases. Aspect is creating these next generation cell therapies by applying its full-stack tissue therapeutic platform, which combines the company’s proprietary bioprinting technology, therapeutic cells, biomaterials, and computational design. Aspect’s bioprinted tissue therapeutics are designed to replace, repair, or supplement biological function inside the body. The company’s current therapeutic pipeline is focused on metabolic and endocrine diseases such as diabetes, obesity, and congenital liver disorders.

www.aspectbiosystems.com

Richard Wilson, SVP, Primary Focus Lead, Genetic Regulation 

April 10 | 3:15pm | Salone dei Cavalieri, Section 3&4

Tokyo, Japan 

(OTCMKTS: ALPMY)     

Astellas Pharma Inc. is an innovative pharmaceutical company with global headquarters in Tokyo, Japan, conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into value for patients.

www.astellas.com 

Greg Kunst, CEO 

April 11 | 11:30am | Salone dei Cavalieri, Section 2

Seattle, WA 

(Private) 

Aurion Biotech is a clinical-stage biotech company, whose mission is to restore vision to millions of patients with life-changing regenerative therapies. Aurion Biotech is the recipient of the prestigious Prix Galien award for Best Start-Up in Biotech. The Company’s first candidate, AURN001, is an allogeneic cell therapy for the treatment of corneal edema secondary to corneal endothelial disease. It is the first off-the-shelf, clinically validated cell therapy for corneal care, having received regulatory approval in Japan. The Company has initiated clinical trials in the U.S. in 2023. Privately held, Aurion Biotech is backed by leading investors that include Deerfield, Alcon, Petrichor, Flying L Partners, Falcon Vision / KKR, and Visionary Ventures.

www.aurionbiotech.com 

Christopher Vann, Ph.D., Chief Operating Officer

April 10 | 4:00pm | Salone dei Cavalieri, Section 3&4

London, United Kingdom

(NASDAQ GS: AUTL)

Autolus is a CAR T cell therapy company. We are applying our extensive programming capabilities to develop advanced autologous T cell therapies that have the potential to deliver life-changing benefits to cancer patients. Autolus is a leader in T cell programming, underpinning the broad technology toolbox and manufacturing technology. Autolus is developing cancer therapies which we believe will offer patients substantial benefits over the existing standard of care.

www.autolus.com

Michael Maguire, CEO

April 11 | 10:30am | Salone dei Cavalieri, Section 2

Dublin, Ireland

(Private)

Avectas has developed Solupore, a ground-breaking non-viral cell engineering platform for next-gen cell therapies. It excels at yielding cells with high viability, functionality, and potent cytotoxicity for patient dosing, after complex gene editing. Solupore gives therapeutic developers new manufacturing options including 

www.avectas.com

Lisa Deschamps, CEO, Executive Board Member AviadoBio

April 10 | 1:30pm | Salone dei Cavalieri, Section 2

London, United Kingdom

(Private)

AviadoBio’s mission is to develop and deliver potentially transformative gene therapies for people living with devastating neurodegenerative diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working relentlessly to overcome the challenges of delivering the right drug to the right place. Our innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery and commercialization experience which uniquely positions the Company for success in bringing transformative medicines to patients.

www.aviadobio.com

Louisa Petropoulos, Ph.D., CEO

April 11 | 11:45am | Salone dei Cavalieri, Section 2

Montreal, Canada

(Private)

C3i is a global full-service CDMO manufacturing from process development to phase 3 in EU, US and Canada with commercial manufacturing launch in 2024 without the need to tech transfer to a 2nd facility. C3i offers a unique model offering shared risk with innovative companies to help them advance through funding gaps. This unites the company and C3i with a common goal ensuring success.

• Strong process development team with diverse experience in cell manufacturing including CAR-T, TcR, B-cell, NK, MSC, IPSC, and allogeneic.
  • viral & non-viral transduction
  • CRISPR editing
• Strong scale-up and scale-out ability for commercial manufacturing
• High production success rate of ~99%
• Validated Qc testing in-house
• CAP-accredited immune monitoring and diagnostic testing services
• CRO services specializing in cell therapy clinical trials
• Attractive COGS (>25% cheaper than other US/EU CDMOs)
• Partnership with leading research hospital and access to KOLs for protocol development, clinical trial launch, and DSMB review.
• C3i and company work as a team while keeping the patient in mind.

www.centreC3i.com

Tom Wilton, Chief Business Officer

April 9 | 2:45pm | Salone dei Cavalieri, Section 2

Philadelphia, PA

(NASDAQ: CARM)

Carisma Therapeutics is a clinical stage biopharmaceutical company focused on utilizing our proprietary macrophage and monocyte cell engineering platform to develop transformative immunotherapies to treat cancer and other serious diseases. We have created a comprehensive, differentiated proprietary cell therapy platform focused on engineered macrophages and monocytes, cells that play a crucial role in both the innate and adaptive immune response.

www.carismatx.com

Lee Dunham, Director of Business Development

April 9 | 9:15am | Salone dei Cavalieri, Section 2

London, UK

(Private)

The Cell and Gene Therapy Catapult is an independent innovation and technology organization committed to the advancement of cell and gene therapies with a vision of a thriving industry delivering life changing advanced therapies to the world. Its aim is to create powerful collaborations which overcome challenges to the advancement of the sector. With over 400 experts covering all aspects of advanced therapies, it applies its unique capabilities and assets, collaborates with academia, industry and healthcare providers to develop new technology and innovation. The Cell and Gene Therapy Catapult works with Innovate UK.

https://ct.catapult.org.uk

Stefan Braam, Ph.D., President and Chief Technology Officer

April 10 | 11:45am | Salone dei Cavalieri, Section 2

Mont-Saint-Guibert, Belgium

(Private)

Cellistic traces its origin to a single imperative: we are accelerating allogeneic cell therapy development through reliable manufacturing processes, at lower costs. Through facilities purpose-built for that mission, we help clients simplify their cell therapy development with our iPSC-based technologies and pre-built processes, using our proprietary Pulse & Echo platforms. Our Pulse platform for cell line development provides iPSC and iPSC-based cell lines that are ready for client’s unique edits, to create product-specific working and master cell banks. Our Echo platform for cell therapy manufacturing is centered on a highly efficient family of feeder-free differentiation and expansion processes for NK cells, α/β T cells, γ/δ T cells, macrophages, and other related lineages. The combination of these two platforms ensures customers to cut years of their product development timeline at a much lower cost.

www.cellistic.com

Rodolphe Clerval, CEO

April 10 | 1:45pm | Salone dei Cavalieri, Section 2

Paris, France

(Private)

Coave Tx is a genetic medicine company, based on a unique technology (ALIGATER) for functionalizing LNPs and AAVs, by conjugation of rationally designed ligands onto vectors surface, improving tissue targeting and efficient payload delivery. Our therapeutics pipeline, based on ALIGATER vectors, is focused on eyes and CNS disorders.

www.coavetx.com

Lijun Wang, Ph.D., CEO 

April 9 | 11:45am | Salone dei Cavalieri, Section 2 

Philadelphia, PA  

(Private) 

CoJourney is an end-to-end global gene therapy CDMO that integrates the services from process development, cGMP manufacture, analytical methods development, and release testing for plasmids, viral vectors and mRNA, in our state-of-the-art facilities that meet FDA, EU, NMPA guidelines. Some points to consider why CoJourney is a great fit for your organization to partner with to take your research into commercial scale. Robust, scalable non-chromatographic downstream platform for high purity plasmid production, leads to a substantial cost reduction, approximately 60% lower, in comparison to a process that relies on 2-3 chromatography purifications. GMP grade plasmid manufacturing: 5 production trains in 6L, 30L fermentation scales, 350 batches per year GMP grade viral vector manufacturing using Industry-leading AAV triple transfection production process yields an upstream 0.5-1.0E12 vg/mL crude titer, in a 200L or 500L bioreactor. mRNA production scale IVT 1L to 50L.Serving 20+ clients since inception.

www.cojourney.com 

Mark Flower, Vice President – Business Development, Cryopreservation

April 10 | 1:15pm | Salone dei Cavalieri, Section 2

Brentwood, TN

(NASDAQ: CYRX)

Cryoport Systems is the market leader in globally integrated temperature-controlled supply chain solutions for critical, irreplaceable products and materials in the life sciences industry. We are already trusted to support 650+ active clinical trials and leading commercial products worldwide – delivering unparalleled supply chain services for products, therapies and treatments requiring unique, specialized temperature-controlled management. And, as the life sciences constantly evolve and advance, so do we.

www.cryoport.com

Timothy Schroeder, Chairman and CEO

April 10 | 9:30am | Salone dei Cavalieri, Section 2

Cincinnati, OH

(Private)

CTI Clinical Trial and Consulting Services is a global, privately held, research service organization, delivering a complete spectrum of clinical trial and consulting services throughout the lifecycle of development, from concept to commercialization. CTI’s focused therapeutic approach provides pharmaceutical, biotechnology, and medical device firms with clinical and disease area expertise in rare diseases, regenerative medicine / gene therapy, immunology, transplantation, nephrology, hematology / oncology, neurology, infectious diseases, hepatology, cardiopulmonary, and pediatric populations. CTI also offers a fully integrated multi-specialty clinical research site and complete global laboratory services. Now in its third decade, CTI is one of the 20 largest contract research organizations in the world, with associates in more than 60 countries across six continents. CTI is headquartered in the Greater Cincinnati area, with operations across North America, Europe, Latin America, MEA, and Asia-Pacific.

www.ctifacts.com

John Lewis, Ph.D., CEO

April 9 | 2:15pm | Salone dei Cavalieri, Section 2

Edmonton, Alberta, Canada

(Private)

Entos Pharmaceuticals Inc. is a clinical-stage genetic medicines company founded in 2016.  Our proprietary Fusogenix™ Proteo-Lipid Vehicle™ (PLV™) platform, enables precision non-viral, re-dosable delivery of all nucleic acids payloads (DNA, RNA, or combinations), ushering in the new era of genetic medicine.  The Fusogenix platform combines the best attributes of current viral and nonviral delivery technology that allows for delivery of all nucleic acid modalities including gene editing tools. The Fusogenix platform can target specific tissues, including extra-hepatic targets with precision, avoiding the liver altogether if desired. Entos is leveraging its groundbreaking Fusogenix platform to solve the delivery problem holding back the advancement of genetic medicines. Entos is developing these therapies through its internal pipeline and through partnerships with other biotech/pharma companies.

www.entospharma.com

Romain de Rauville, M.D., Chief Business Officer

April 11 | 10:15am | Salone dei Cavalieri, Section 2

Liege, Belgium

(Private)

EXO Biologics is a Belgian biotech company committed to developing biopharmaceuticals using Extra Vesicles/Exosomes to treat rare diseases with unmet medical needs. In 2023, EXO Biologics received the authorization by EMA to start EVENEW: its clinical phase I trial for BPD (Broncho Pulmonary Dysplasia). EXO Biologics hence became the first company in the world to ever obtain an MSC-derived Extracellular Vesicles interventional study approved by the European Medicines Agency. EXO Biologics is now a world leader within the exosomes space as it successfully resolved the process development and regulatory challenges for starting clinical trials. The company is therefore now paving the way for the young but promising exosome field not only as a therapeutic but also as delivery vehicle.

www.exobio.be/en

Tim Miller, Ph.D., CEO, President & Co-Founder

April 10 | 11:30am | Salone dei Cavalieri, Section 2

Grove City, OH

(Private)

Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company focused on enabling access to life-changing gene therapies. Forge was founded in 2020 and is headquartered in the cell and gene therapy hub of Columbus, Ohio. Its 200,000 square foot facility, the Hearth, is dedicated to AAV manufacturing with 20 custom-designed cGMP suites. Offerings include scalable, end-to-end manufacturing services including process and analytical development, cGMP viral vector manufacturing, final fill, plasmid DNA manufacturing, as well as regulatory consulting support to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. Forge aims to accelerate the timelines of transformative medicines for patients with genetic diseases.

www.forgebiologics.com

Eric Blair, Chief Business Officer and Singapore Country Executive

April 9 | 4:45pm | Salone dei Cavalieri, Section 2

Nucleos, Singapore

(Private)

GeneFab is a contract research and manufacturing organization focused on cell and gene therapies. GeneFab was formed in 2023 with a vision to combine industry leading expertise in synthetic biology with advanced cGMP capabilities in order to accelerate the development and commercialization of genetic medicines. GeneFab capabilities spans early-stage product design, technical development and cGMP compliant production including CAR-T/CAR-NK Cells, iPSCs, engineered cell banks, AAV, lentiviral vectors, gamma-retroviral vectors, mRNA, LNP and pDNA. GeneFab’s technology platform includes bioinformatic-guided discovery of cell type promoters, directed evolution of small molecule-regulated gene switches, the engineering of highly sensitive kill switches for enhanced safety and control of cellular therapies, and innovator-centric manufacturing platforms to accelerate novel therapies to patients in need. Across two facilities in Alameda and South San Francisco and facility soon in Singapore, GeneFab employs approximately 100 employees, including experts in cell, gene, and nucleic acid therapy, CMC, analytical development, process development and synthetic biology.

www.genefab.com

Angela Columbano, Ph.D., Head of Business Development and Partnership

April 9 | 11:30am | Salone dei Cavalieri, Section 2

Évry, France

(Private)

A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a unique non-profit organization created by a patient association, the AFM- Telethon. A first gene therapy drug, to which Genethon contributed, has obtained marketing for spinal muscular atrophy. With 200+ scientists and professionals, Genethon is pursuing its mission to bring life-changing therapies to patients suffering from rare genetic diseases. Thirteen products resulting from Genethon’s research are in clinical trials for liver, blood, immune system, muscle and eye, diseases. A further six products are in the preparation phase for clinical trials over the next five year.

www.genethon.com

Sumant Ramachandra, M.D., Ph.D., CEO

April 9 | 12:00pm | Salone dei Cavalieri, Section 2

Los Angeles, CA,

(Private)

ImmPACT Bio USA, Inc., is a clinical-stage company dedicated to the discovery of transformative chimeric antigen receptor (CAR) T-cell therapies that address key biological challenges in treating cancer and autoimmune diseases. The company’s logic-gate-based CAR T-cell platforms, licensed from University of California, Los Angeles (UCLA), are specifically designed to prevent antigen escape, overcome the immunosuppressive tumor microenvironment, and deplete pathogenic B cells. The company’s technology is based on the work of pioneering UCLA scientists Yvonne Chen, Ph.D., and Antoni Ribas, M.D., Ph.D.

www.immpact-bio.com

Kate Rochlin, Ph.D., Chief Operating Officer

April 9 | 10:15am | Salone dei Cavalieri, Section 2

New York, NY

(NASDAQ: INAB)

IN8bio is a clinical-stage biopharmaceutical company focused on developing novel gamma-delta T cell therapies for solid and hematological tumors. The company’s lead, INB-400, is in a Phase 2 clinical trial in which autologous gamma-delta T cells, which are engineered to be chemotherapy resistant, will be assessed in newly diagnosed glioblastoma. The Company has two Phase 1 clinical trials: INB-200, for the treatment of patients with newly diagnosed GBM, and INB-100, for the treatment of patients with hematologic malignancies that are undergoing hematopoietic stem cell transplantation. INB-100 is the first allogeneic gamma-delta T cell therapy to show in-vivo persistance and expansion for 365 days and 100% of evaluable treated patients remain in remission, with 6 past the one-year mark. In addition, the company’s DeltEx platform has yielded a broad portfolio of preclinical programs, including INB-300 and INB-500, addressing other solid and hematological tumor types through non-signaling CAR-T’s and iPSCs.

www.in8bio.com

Michelle Duquette, Ph.D., Chief Scientist- Cell Therapy

April 11 | 11:15am | Salone dei Cavalieri, Section 2

San Diego, CA

(Private)

Invetech helps solve commercial manufacturing challenges for the global cell and gene therapy market. With dozens of successful collaborations with the world’s leading regenerative medicine companies over the past 20 years, we’re a proven partner committed to helping you successfully transition from clinical to commercial-scale manufacturing. Our automation platforms and manufacturing systems provide Assured Pathways to integrated commercial manufacturing, from pre-clinical phase to large-scale manufacturing, without any substantive process changes. We configure solutions and provide the systems and resources you need to achieve large-scale, end-to-end cell and gene therapy manufacturing with a single point of accountability.

www.invetechgroup.com

Miguel Forte, M.D., Ph.D., CEO

April 9 | 2:30pm | Salone dei Cavalieri, Section 2

Paris, France

(Private)

Kiji Therapeutics develops state-of-the-art off-the-shelf engineered cell therapies for multiple life-threatening diseases. Incorporated in France and Spain in 2023 with an initial seed from AdBio Partners. Kiji Tx has international Scientific Founders, Management and Scientific/Clinical Advisory Board, experienced in C&GT and product clinical development. With access to R&D and GMP manufacturing facilities in Madrid and Navara, Spain, Kiji Tx has operational readiness to develop the cell engineered platform and move to the clinic with its lead product, with a first patient expected in 2024. The company’s vision is to gene engineer iPSC-derived MSC cells for a targeted and optimized therapeutic benefit in a broad set of disease areas.

www.kiji-tx.com

Kevin McMahon, Solutions Consulting – Therapeutics Lead

April 9 | 11:15am | Salone dei Cavalieri, Section 2

Austin, TX

(Private)

L7 Informatics reimagines data intelligence for modern life sciences and healthcare organizations, optimizing the flow of information between processes and people, unlocking innovation at every stage of the clinical, research, and manufacturing value chains. Our comprehensive operating environment and software stack bring flexible, secure, and collaborative data+intelligence to life sciences organizations. Our end-to-end transformation engine helps solve today’s challenges and realize tomorrow’s opportunities.

www.l7informatics.com

David Venables, Ph.D., CEO

April 9 | 4:15pm | Salone dei Cavalieri, Section 2

London, UK

(Private)

Laverock Therapeutics is developing the next generation of programmable cell therapies though our novel miRNA platform (GEiGS). Gene Editing induced Gene Silencing (GEiGS) utilizes the cells own profiles of endogenous miRNA and redirects specified, recoded miRNAs to knock down novel target genes of choice, generating silencing in a stable, consistent, tuneable and context-specific manner. Cells can be programmed to induce gene silencing in response to defined contexts, such as the stage of cell development, its’ activation, or in response to in vivo disease related environmental cues. Laverock is developing the technology to control  the polarisation of programmed macrophages and the prevention of exhaustion and enhance persistence of (CAR)-T cells targeting the solid tumour micro-environment, plus the design of conditionally hypoimmune stem cell-derived islets for the T1D regenerative medicine application.

www.laverocktx.com

Maher Masoud, CEO

April 9 | 2:00pm | Salone dei Cavalieri, Section 3&4

Rockville, MD

(NASDAQ: MXCT)

MaxCyte is a leading cell-engineering company providing enabling platform technologies to advance the discovery, development and commercialization of next-generation cell therapeutics and to support innovative, cell-based research. MaxCyte builds best-in-class technology designed to facilitate complex engineering of a wide variety of cells and to drive the rapidly expanding cell therapy market forward. For over 20 years, MaxCyte has been a leader in developing transfection workflows and ventures beyond today’s processes to innovate tomorrow’s solutions.

www.maxcyte.com

David Rubinstein, Ph.D., Senior Vice President, Business Development

April 9 | 1:30pm | Salone dei Cavalieri, Section 3&4

New York, NY

(NASDAQ: MGTX)

MeiraGTx is a clinical-stage, fully integrated, gene therapy company with six programs in clinical development across ocular, neurodegenerative and salivary gland conditions. MeiraGTx has core in-house capabilities in viral vector design and optimization, as well as in-house end-to-end cGMP manufacturing facilities. MeiraGTx has developed a proprietary and transformative riboswitch-based gene regulation technology, which allows precise dose-responsive control of gene expression with oral small molecule inducers. This technology can be applied to control the expression of any transgene, including in gene therapy gene editing and cell therapy. MeiraGTx has also developed an extensive array of proprietary constitutive and cell-specific synthetic promoters, by combining rational, high-throughput, and AI/ML approaches for promoter screening and design.

www.meiragtx.com

Anil Narasimha, Ph.D., CEO

April 11 | 9:30am | Salone dei Cavalieri, Section 2

Alameda, California

(Private)

Mekonos is a CGT platform company building the future of cell therapies on a chip. Our technology enables the ex vivo delivery of any molecular cargo to any cell, supporting cell and gene therapy programs from the R&D stage through clinical and commercial manufacturing. Mekonos’ uses silicon nanoneedles to enable high efficiency cargo delivery into the most fragile cells, including primary immune cells, iPSCs and HSCs. Our unique chip architecture aligns single nanoneedles to individually trapped cells (“one needle, one cell”), ensuring that each cell receives the same amount of cargo and resulting in a highly homogenous population of modified cells.  This novel approach enables single cell precision at scale and unlocks cell therapy applications which are unachievable with conventional delivery technologies, like electroporation and viral vectors. We address the key challenges in cell and gene therapy – targeted delivery, cell survival and dose regulation (which translates to clinical safety).

www.mekonos.com

Sean Kevlahan, Ph.D., CEO

April 10 | 5:00pm | Salone dei Cavalieri, Section 2

Boston, MA

(Private)

Nanite is a next-generation non-viral gene delivery company developing a new class of programmable polymer nanoparticles for a range of modalities and indications. The company’s AI-driven platform, SAYER, combines cutting-edge high-throughput experimental and computational methods to design fit-for-purpose delivery vehicles delivering a broad range of genetic cargoes with tissue specificity.

www.nanitebio.com

Rathish Ravindrakumar, Director Business Development 

April 9 | 4:00pm | Salone dei Cavalieri, Section 2 

Basel, Switzerland 

(NYSE: NVS) 

At Novartis contract manufacturing, we partner with biopharmaceutical companies to bring high-quality medicines to patients globally. We strive to serve as the manufacturing partner of choice by solving our clients’ manufacturing challenges leveraging the experience of our people and state-of-the-art facilities at EU, US and Singapore. Our services and capabilities include:

• Cell therapy: Autologous 
• Viral vectors: LV, AAV 
• plasmid DNA, mRNA, LNP 
• Fill & Finish  
• Mammalian (Small and large scale) 
• Microbial (Small and large scale) 
• Solid phase synthesis

www.novartis.com/about/manufacturing/contract-manufacturing

Madan Jagasia, M.D., CEO

April 10 | 4:30pm | Salone dei Cavalieri, Section 2

Cambridge, MA

(Private)

At Obsidian, we are pioneering engineered cell and gene therapies to deliver transformative outcomes for patients. OBX-115, our lead product candidate, is an autologous tumor-infiltrating lymphocyte (TIL) cell therapy manufactured using a portion of the patient’s tumor tissue, from which TIL are isolated and engineered with a transgene to express membrane-bound human IL15 (mbIL15). The transgene construct encodes for a fusion protein that couples mbIL15 with a drug-responsive domain (DRD). The DRD is stabilized by administration of the small-molecule drug, acetazolamide (ACZ), which upon binding stabilizes the fusion protein, and allows for expression of mbIL15 on the cell surface of the TIL. mbIL15 drives the expansion and persistence of TIL, promoting elimination of tumor cells.

www.obsidiantx.com

Eric Halioua, President and CEO

April 9 | 11:45am | Salone dei Cavalieri, Section 2

Liège, Belgium

(Private)

Founded in 2014 as a spin-off of the French Blood Bank (EFS), PDC*line Pharma is a Belgian-French clinical-stage biotech company that develops an innovative class of active immunotherapies for cancers, based on a GMP-grade allogeneic therapeutic cell line of Plasmacytoid Dendritic Cells (PDC*line). PDC*line is much more potent than conventional dendritic cell-based vaccines in priming and boosting antitumor antigen-specific cytotoxic T-cells, including the T-cells specific for neoantigens, and is synergistic with checkpoint inhibitors. The technology can potentially be applied to any type of cancer. Following a first-in-human phase I feasibility study in melanoma, PDC*line Pharma is now focused on the development of PDC*lung01, its candidate for Non-Small-Cell Lung Cancer (NSCLC), currently in phase I/II trials, and PDC*neo with neoantigens, in preclinical development. The company has a staff of 42, with an experienced management team. It has raised close to €56M in equity and non-dilutive funding. In March 2019, PDC*line Pharma granted the LG Chem Life Sciences company an exclusive license in Asian countries for the development and commercialization of the PDC*lung01 cancer vaccine for lung cancer. The total deal is worth €108M, plus tiered royalties on net sales in Asia. 

www.pdc-line-pharma.com

Kersti Alm, Ph.D., Chief Scientific Officer

April 11 | 11:00am | Salone dei Cavalieri, Section 2

Lund, Sweden

(FRA: L5W)

Phase Holographic Imaging (PHI) is a MedTech company that develops and markets a new generation of microscope-based research instruments for non-invasive time-lapse imaging. PHI offers a cell-friendly, QPI-based alternative to traditional cell measurement methods. QPI technology is used and referenced worldwide for long-term quantitative analysis of living cell cultures, particularly in preclinical research. PHI now develops QPI technology for regenerative medicine and advanced cell quality control. We cooperate with numerous organizations and companies to develop a relevant and robust cell quality control system that will aid in the transition of biomanufacturing from a craft to a highly efficient industrial process.

www.phiab.com

Daniel Shelly, Ph.D., Chief Business Development Officer

April 11 | 3:45pm | Salone dei Cavalieri, Section 2

Gdansk, Poland

(PTG:WSE)

PolTREG S.A. is a clinical-stage European biotechnology company developing T-regulatory cell (Tregs) therapies for autoimmune diseases including Type-1 Diabetes (T1D), Multiple Sclerosis (MS), and AMS. PolTREG has completed four clinical trials with more than 100 patients treated with Tregs. PTG-007 autologous treatment for early-onset T1D is ready for Phase 2/3 clinical trial, after a Phase 2 trial showed half of patients in clinical remission 24 months into the study. The company will launch a Phase 2 clinical trial for PTG-007 presymptomatic T1D in the second half of 2024. In MS, the company will launch Phase 2 trials for PTG-007 in the second half of 2024 for RRMS and PPMS. PolTREG is also developing engineered Tregs, including CAR-Tregs, antigen-specific Tregs and TCR-Tregs, all of which are in the preclinical stage. PolTREG has completed construction on one of the largest, state of the art, cGMP manufacturing facilities in Europe.

www.poltreg.com

Helen Sabzevari, Ph.D., President and CEO

April 10 | 2:30pm | Salone dei Cavalieri, Section 2

Germantown, MD

(NASDAQ: PGEN)

Precigen is a dedicated discovery and clinical stage biopharmaceutical company advancing the next generation of gene and cell therapies using precision technology to target the most urgent and intractable diseases in our core therapeutic areas of immuno-oncology, autoimmune disorders, and infectious diseases. Our technologies enable us to find innovative solutions for affordable biotherapeutics in a controlled manner. Precigen operates as an innovation engine progressing a preclinical and clinical pipeline of well differentiated therapies toward clinical proof-of-concept and commercialization.

www.Precigen.com

Kara Poon, Ph.D., Senior Scientist

April 10 | 9:00am | Salone dei Cavalieri, Section 2

Stockholm, Sweden

(Private)

QuTEM (formerly Vironova BioAnalytics) specializes in quantitative nanoparticle analysis using Transmission Electron Microscopy (TEM) primarily for gene therapy, vaccine and drug delivery fields. Operating a GMP-compliant lab in Stockholm, Sweden, QuTEM provides crucial insights into product safety, efficacy, and stability for clients.

www.qutem.com

Peter Francis, M.D., Ph.D., Chief Scientific and Medical Officer

April 10 | 4:00pm | Salone dei Cavalieri, Section 2

April 12 | 9:30am | Dark Horse Consulting Ballroom

San Francisco, CA

(Private)

Ray Therapeutics (RayTx) is a Bay Area CIRM-funded biotechnology company developing next generation optogenetic therapies for vision restoration in patients with retinal blindness. In May 2023, RayTx closed a $100M series A. Early-stage clinical trials with the first iteration of optogenetic gene therapies have provided strong signals of biological activity. Building on this foundation, Ray Therapeutics (RayTx) has bioengineered a highly sensitive optogenetic protein, ChRown, which for the first time, has optimal characteristics for human vision.  Delivered using a AAV vector variants developed for low dose intravitreal delivery to the retina, RayTx’s assets, RTx-015 & -021 have the promise to realize the full potential of visual optogenetics and provide patients higher visual function than previously contemplated. ChRown is also sensitive enough to be active in ambient lighting conditions without the use of light-intensifying “goggles”. RayTx first clinical trial is anticipated to initiate by in 2024.

www.raytherapeutics.com

Kinnari Patel, President and Chief Operating Officer

April 10 | 3:30pm | Salone dei Cavalieri, Section 3&4

Cranbury, N.J.

(NASDAQ: RCKT)

Rocket Pharmaceuticals is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. The Company’s platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket’s clinical programs using lentiviral (LV) vector-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rocket’s first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. Rocket also is developing AAV-based gene therapy programs in PKP2-arrhythmogenic cardiomyopathy (ACM) and BAG3-associated dilated cardiomyopathy (DCM).

www.rocketpharma.com

Mark Missen, Head of Manufacturing Operations

April 9 | 3:45pm | Salone dei Cavalieri, Section 2

Edinburgh, Scotland

(Private)

RoslinCT is a leading global contract development and manufacturing services organization (CDMO) focused on Advanced Cell and Gene Therapies. Established in 2006 and built upon the ground-breaking technology cloning of Dolly the Sheep at the Roslin Institute in 1997, RoslinCT has harnessed cutting-edge science to advance the development of human medicines. With a remarkable heritage in the field, the company has achieved significant milestones. These include being among one of the first in the world to produce clinical-grade human pluripotent stem cells. In collaboration with the partners, RoslinCT also developed the first CRISPR-edited cell therapy product for a major inherited disorder, taking it from early development to commercialization. Equipped with 22 purpose-built cGMP cell therapy processing suites in Edinburgh, Scotland, and Hopkinton, Massachusetts, RoslinCT provides innovative process and analytical development, cGMP clinical and commercial manufacturing for a range of cell types for both autologous and allogeneic processes, and cGMP iPSC cell line development, gene editing, and differentiation. With tailored CDMO solutions, RoslinCT enables partners to efficiently progress from development to commercialization and deliver life-saving Cell and Gene Therapies worldwide. RoslinCT is a GHO Capital portfolio company.

www.roslinct.com

Tom Lowery, Ph.D., President and Chief Technology Officer

April 10 | 4:15pm | Salone dei Cavalieri, Section 2

Watertown, MA,

(Private)

Satellite Bio is a well-funded preclinical regenerative medicine company with cutting-edge hepatocyte technologies designed to tackle a broad range of liver diseases. Our platform encompasses a proprietary primary hepatocyte expansion technology that allows the dosing of over 1000 patients from a single donor liver. Additionally, we specialize in stabilizing hepatocytes in heterocellular aggregates, enabling cryopreservation for long-term stability and shipment, and significantly improved in vivo engraftment and durability. Moreover, our expertise also extends to the generation of engineered hepatocytes optimized for the endogenous expression of therapeutic proteins. This presentation will offer an overview of Satellite Bio’s technology and manufacturing process.

www.satellite.bio

Valérie Salentey, Regulatory Affairs and Quality Assurance Director  

April 9 | 5:00pm | Salone dei Cavalieri, Section 2

Paris, France 

(NASDAQ: ALSEN) 

Sensorion is a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing disorders, a significant global unmet medical need. Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases, enabling it to select the best targets and mechanisms of action for drug candidates. It develops two gene therapy programs (SENS-501 and GJB2-GT) aimed at correcting hereditary monogenic forms of deafness, developed in the framework of its broad strategic collaboration focused on the genetics of hearing with the Institut Pasteur. Sensorion’s clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) progressing in a planned Phase 2 proof of concept clinical study of SENS-401 in Cisplatin-Induced Ototoxicity and, with partner Cochlear Limited, in a study of SENS-401 in patients scheduled for cochlear implantation.

www.sensorion.com 

Cynthia Pussinen, CEO

April 9 | 10:30am | Salone dei Cavalieri, Section 2

London Ontario, Canada

(TSX: SVA; OTCQB: SEOVF; FSE/XETRA: PSH)

Sernova is a clinical-stage biotechnology company that is developing therapeutic cell technologies for chronic diseases, including insulin-dependent diabetes, thyroid disease, and hemophilia A. The Company is focused on developing a ‘functional cure for insulin-dependent diabetes with its lead asset, the Cell Pouch System, a novel implantable and scalable cell therapy platform transplanted initially with human donor islets but ultimately using an iPSC islet like cluster technology exclusively licensed from Evotec SE. Sernova is progressing a Phase 1/2 clinical trial evaluating the Cell Pouch System in patients with T1D with impaired hypoglycemia awareness. Of six patients in the first cohort of the trial, five are now insulin-independent, with the first patient at almost four years without the need for insulin injections and a normalized HbA1c. Enrollment in a second cohort of patients, which employs a higher capacity Cell Pouch System, to be completed shortly with data to come in 2024.

www.sernova.com

Alain Lamproye, Division Head 

April 11 | 9:15am | Salone dei Cavalieri, Section 2 

Cordova, CA 

(Private) 

Built on 80 years of experience, SK pharmteco is a trusted partner specializing in the manufacture of APIs and intermediates, cell and gene therapy technologies, registered starting materials and analytical services for the pharmaceutical industry worldwide. Our global operations have the capability and capacity to support your needs from development through commercial production.

www.skpharmteco.com 

Niklas Prager, CEO

April 10 | 3:15pm | Salone dei Cavalieri, Section 2

Tullinge, Sweden

(Private)

SmartCella is a world-leading biotech & medtech company pioneering the future of targeted therapies by combining first-in-class delivery platforms with cutting-edge cell and gene therapies. The company has three units:Â Procella with advanced capabilities within stem cell therapy development and cGMP manufacturing inhouse. Procella collaborates with AstraZeneca on a globally licensed cardiac stem cell therapy to repair parts of the heart damaged from a heart attack and has a growing pipeline of stem cell therapies. In combination with these therapeutic endeavors, SmartWise with the FDA-approved Extroducer, an endovascular device that enables direct tissue or organ infusion seeks to address indications impacting hard-to-reach organs and tumors. SmartCella Solutions creating novel mRNA therapies and platforms that enable cell-mediated delivery of protein and mRNA to specific regions, even within organs. SmartCella, founded in 2014, is based in Tullinge, Sweden and has approximately 60 employees, mainly scientists and researchers, with around 20 different nationalities represented.

www.smartcella.com

Peter Webster, Scientist II

Charlestown, MA

(NASDAQ: SLDB)

Solid Biosciences is a life sciences company focused on advancing a portfolio of gene therapy candidates and neuromuscular and cardiac programs, including SGT-003, for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, AVB-202-TT for the treatment of Friedreich’s ataxia, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases.

www.solidbio.com

Daniel Chung, Chief Medical Officer

April 10 | 2:00pm | Salone dei Cavalieri, Section 2

Paris, France

(Private)

SparingVision is a global ophthalmology leader bringing new hope to millions affected by retinal diseases, for which there are currently no viable treatments. The Company has assembled a suite of cutting-edge technologies from gene therapy to CRISPR, enabling the company to deploy the right technology to the right disease and ensure the delivery of breakthrough treatments to millions of patients. Both of its products, SPVN06 and SPVN20 go beyond single gene correction therapies to deliver new gene-agnostic treatments for Retinitis Pigmentosa , a group of inherited retinal diseases which are the leading cause of blindness globally. The Company also has a strategic collaboration with Intellia Therapeutics (NASDAQ:NTLA) to develop novel genome editing-based treatments for ocular disease utilizing CRISPR-Cas9 technology. SparingVision is backed by high-quality international investors including 4BIO Capital, Advent France Biotechnology, Bpifrance, Foundation Fighting Blindness (US), Fondation Voir & Entendre, Intellia Therapeutics, UPMC Enterprises, Jeito Capital, Ysios Capital.

www.sparingvision.com

Gerard Caelles, Chief Business Officer

April 10 | 11:00am | Salone dei Cavalieri, Section 2

Barcelona, Spain

(Private)

SpliceBio is a genetic medicines company harnessing its proprietary Protein Splicing platform to develop the next generation of gene therapies. Our platform offers the potential to address diseases that currently cannot be treated with gene therapies because the necessary gene is too large to be delivered by AAV vectors. Our lead program SB007 targets Stargardt disease, a genetic eye disease that causes vision loss in children and adults. SB007 has achieved high-level expression of functional, full-length human ABCA4 in vitro and in vivo after subretinal injection across species including rodents, minipigs and NHP macaques, with dose-dependent pharmacological activity in animal disease models of STGD1. SpliceBio is also developing SB008 for an IRD and SB011 for a monogenic CNS disease. The company raised a $57M series A in 2022 (UCB, Ysios, NEA, Gilde, Novartis and Asabys) and a partnership with Spark (Roche) in October 2023 to develop a novel gene therapy for an undisclosed IRD.

www.splice.bio

Laura Koivusalo, Ph.D., CEO and Founder

April 10 | 4:45pm | Salone dei Cavalieri, Section 2

Tampere, Finland

(Private)

StemSight is developing a unique cell-based approach to treat corneal blindness using allogeneic human induced pluripotent stem cells (iPSCs). Our first product STE-001 is targeting the rare disease limbal stem cell deficiency (LSCD), for which iPSC-derived therapeutic limbal stem cells (LSCs) will be transplanted to restore the natural renewal capacity of the corneal epithelium. We further aim to develop additional products for the treatment of different forms of corneal blindness by further cell therapy products, such as iPSC-derived corneal endothelial cells (CEnC) (STE-002). The company was established around IP developed in Tampere University (Finland) by Professor Heli Skottman and her team. Â Professor Skottman is one of the world’s leading experts in ocular stem cell biology.

www.stemsight.fi

Dagmara Szkolnicka, Ph.D., Co-founder and Chief Scientific Officer

April 9 | 1:30pm | Salone dei Cavalieri, Section 2

Copenhagen, Denmark

(Private)

The company is developing a scalable and effective treatment for liver diseases using liver tissue implants developed from pluripotent stem cells (PSCs). Stimuliver has developed revolutionary proof-of-concept data which demonstrates that human liver tissue can be administered under the skin, providing long-term host liver support. For chronic liver disease patients, there are benefits of implanting cells subcutaneously, as the liver is highly inflamed making it a hostile environment for cell-based therapies. By utilizing PSCs as a source of tissue, the technology Stimuliver has developed is not dependent on donor organ availability. This may in the future provide a treatment which is available without waiting time and has the potential to reverse liver disease and prevent prolonged or recurrent hospitalizations. The research is based on technology developed at the University of Edinburgh’s Centre of Regenerative Medicine, is incubated at the BioInnovation Institute and was funded by the Novo Nordisk Foundation.

www.stimuliver.bio

Matthew Lakelin, Ph.D., VP Scientific Affairs & Product Development, Co-Founder

April 9 | 9:30am | Salone dei Cavalieri, Section 2

Cardiff, United Kingdom

(Private)

TrakCel is the first provider of integrated cellular orchestration solutions for the precise management, control and tracking of cell and gene therapy products. With the largest number of live deployments supporting the broadest number of therapy classes in industry, TrakCel has built a deep understanding of the unique challenges faced by advanced therapy developers globally. OCELLOS by TrakCel is a powerful, integrated cellular orchestration solution for the precise management, control and tracking of advanced therapy supply chains, patient by patient. OCELLOS supports autologous, allogeneic and personalised cancer vaccine therapies at all clinical and commercial stages whilst OCELLOS Lite has been developed for quick, cost-effective deployment for early-stage clinical therapies. Our brand-new product OCELLOS Core is an out-of-the-box Chain of Identity and Chain of Custody solution for therapy developers from first in human clinical trials. TrakCel’s cell orchestration platforms help to establish and safeguard the therapy Chain of Identity and Chain of Custody whilst generating full, reportable, electronic audit trails in real-time for each patient. Dashboards and reports unite therapy developers with healthcare providers and supply chain partners, sharing the information needed to guide decisions and creating alerts and notifications to progress the journey. TrakCel SaaS cellular orchestration solutions are built on the Salesforce platform and offer visibility, insight and scalability, streamlining the delivery of more therapies, to more patients at their most vulnerable.

www.trakcel.com

Frederic Desdouits, Ph.D., CEO

April 9 | 4:30pm | Salone dei Cavalieri, Section 2

Pessac, France

(Private)

TreeFrog Therapeutics is a French-based biotech company set to unlock access to cell therapies for millions of patients. TreeFrog is unique in its approach to cell therapy development bringing together biophysicists, cell biologists and bioproduction engineers to address the challenges of the industry – producing and differentiating cells of quality at unprecedented scale, cost-effectively. To realize their mission of Cell Therapy for all, TreeFrog operates a hybrid business model that includes its own therapeutic programs and partnerships with leading biotech and industry players in other areas. In the last 3 years, the company has raised $82 million to advance a pipeline of stem cell-based therapies in regenerative medicine and immuno-oncology. In 2022, the company opened technological hubs in Boston, USA, and Kobe, Japan, to drive the adoption of the C-StemTM platform and establish strategic alliances with leading academic, biotech and industry players.

www.treefrog.fr

David Fontana, Ph.D., Chief Operating and Business Officer

April 10 | 3:45pm | Salone dei Cavalieri, Section 2

Seattle, WA

(Private)

Umioja Biopharma is the leader in in vivo CAR T delivery with its novel platform named VivoVec. As a private company we employ ~170 employees between our Seattle HC and our state of art GMP manufacturing facility in CO. We have recently shared best in field non-human promate data and two of our in vivo products will be in the clinic in 2024. Umoja also has an iPSC platform.

www.umoja-biopharma.com

Richard Porter, Ph.D., Chief Business and Scientific Officer

April 10 | 3:45pm | Salone dei Cavalieri, Section 3&4

Amsterdam, Netherlands

(NASDAQ: QURE)

uniQure is delivering on the promise of gene therapy a single treatment with potentially curative results through an innovative adeno-associated virus (AAV)-based technology platform supported by proprietary commercial-grade manufacturing. The recent approvals of our gene therapy for hemophilia B an historic achievement based on more than a decade of research and clinical development represent a major milestone in the field of genomic medicine and usher in a new treatment approach for patients living with hemophilia. We’re now advancing a clinical program in Huntington’s disease with a growing list of early clinical and preclinical candidates.

www.uniqure.com

Petter Bjorquist, Ph.D., CEO

April 9 | 3:00pm | Salone dei Cavalieri, Section 2

Gothenburg, Sweden

(Private)

VERIGRAFT AB is a Swedish biotechnology company run by experienced entrepreneurs and mastering a unique, breakthrough technology in the field of advanced therapies and regenerative medicine. We make transplantation possible without the severe risks of immunosuppression, and develop advanced therapies and tissue engineered products that will be able to help millions of patients with serious diseases. Our first product, the Personalized Tissue Engineered Vein (P-TEV) is a human vein segment graft for use in transplantation to replace a defective or missing part of a patient’s vein. A P-TEV with functioning valves is implanted to replace a non-functioning venous valve in the femoral vein of a patient suffering from severe chronic venous insufficiency (CVI). The first-in-man clinical trial is ongoing, and results are very positive. These patients do not receive any curative treatment today.

www.verigraft.com

Paulash Mohsen, CEO

April 10 | 10:45am | Salone dei Cavalieri, Section 2

Cambridge, MA

(Private)

Vesigen Therapeutics is a biotechnology company developing a novel, non-viral delivery technology for gene editing, RNA, and protein-based therapeutics. Vesigen’s patented technology, called ARMMs (ARRDC1 Mediated Microvesicles), can be used to precisely deliver a wide range of payloads to a unique set of tissue and cell types. Vesigen has demonstrated highly efficient in vitro and in vivo functional delivery of a range of payloads across multiple cell types and is committed to developing transformative medicines that address current unmet medical needs. ARMMs were discovered and engineered into a drug delivery system at the Harvard T.H. Chan School of Public Health.

www.vesigen.com

John Hadden, CEO

April 11 | 10:00am | Salone dei Cavalieri, Section 2

London, United Kingdom

(Private)

ViroCell Biologics is an innovation-driven Contract Development and Manufacturing Organization focused exclusively on the design and GMP manufacture of viral vectors for clinical trials. Built around one of the most prolific academic viral vector manufacturing teams, ViroCell was created to address the global demand for precisely engineered viral vectors and aims to become the partner of choice for companies developing cell and gene therapies. The team leverages its deep track record to help clients to de-risk and accelerate novel cell and gene therapies into and through clinical development, with a mission of being the partner of choice for corporate and academic innovators in this field. Focused initially on manufacturing lentivirus and gamma-retrovirus vectors, ViroCell enables clients to start clinical trials on a scalable platform, delivering value by reducing costs, time and regulatory risk.

virocell.com

Jean -Philippe Combal, Ph.D., CEO and Co-Founder

April 9 | 10:00am | Salone dei Cavalieri, Section 2

Paris, France

(Private)

Vivet Therapeutics is a clinical-stage emerging biotechnology company developing novel gene therapy treatments for rare, inherited liver metabolic diseases. Vivet is building a diversified gene therapy pipeline based on novel rAAV technologies. Vivet’s lead program, VTX-801, is currently under clinical development (GATEWAY) with first key results expected in 2024. VTX-801 is a novel investigational gene therapy for Wilson Disease, a rare genetic disorder caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurologic symptoms and potentially death. Vivet’s additional gene therapy products at non clinical stage are focusing on orphan & ultraorphan indications as VTX-803 for PFIC3, Cerebro-Tendinous Xanthomatosis, PKU and liver fibrosis. Vivet is also working on technological platforms addressing key challenges of gene therapy, including sustained therapeutic gene expression in young patients, adults and ability to treat Nabs positive patients with VTX-PID as clinical stage program.

www.vivet-therapeutics.com

Robert Ang, M.D., President and CEO

April 10 | 3:30pm | Salone dei Cavalieri, Section 2

Cambridge, MA

(NASDAQ: VOR)

Vor Bio is a clinical-stage cell and genome engineering company that aims to change the standard of care for patients with blood cancers by engineering hematopoietic stem cells to enable targeted therapies post-transplant.

www.vorbio.com

Dr. Sebastian Schuck, Ph.D., Sr. Director – Client and Contract Management

April 11 | 9:15am | Salone dei Cavalieri, Section 2

San Diego, CA

(OTCMKTS: WKCMF)

Wacker Biotech is your CDMO partner for the entire manufacturing chain of plasmid DNA (pDNA) and messenger RNA (mRNA) to lipid nanoparticle (LNP) formulations. We leverage our global team of development, process engineering, and manufacturing experts and a network of state-of-the-art facilities to accelerate our partners’ programs from research phase to the market.

www.wacker.com