Company Presentations

Slate of Company Presenters Announced!

See the list of 50+ companies presenting at the inaugural Cell & Gene Meeting on the Mediterranean who will also be available for one-on-one partnering at the program. Collectively, these leading companies are working on a number of unique products with an extensive range of possible therapeutic applications. Don’t miss your chance to meet with senior executives from the following companies – register for the conference today!

2019 Company Presenters





ADAPTIMMUNE
Adrian Rawcliffe, Chief Financial Officer
Philadelphia, PA and Oxford, UK
(NASDAQ: ADAP)
Adaptimmune, a leader in T-cell therapy, has clinical trials ongoing for three wholly-owned SPEAR T-cells (ADP-A2M4 (MAGE-A4), ADP-A2M10 (MAGE-A10), and ADP-A2AFP (AFP)) in multiple solid tumor indications. The company’s unique SPEAR (Specific Peptide Enhanced Affinity Receptor) T‑cell platform enables the engineering of T-cells to target and destroy cancer, including solid tumors. www.adaptimmune.com

ADVERUM BIOTECHNOLOGIES
Leone Patterson, President and CEO
Menlo Park, CA
(NASDAQ: ADVM)
Adverum is a clinical-stage gene therapy company targeting unmet medical needs in serious rare and ocular diseases. Adverum has a robust pipeline that includes product candidates designed to treat rare diseases alpha-1 antitrypsin (A1AT) deficiency and hereditary angioedema (HAE) as well as wet age-related macular degeneration (wAMD). Leveraging a next-generation adeno-associated virus (AAV)-based directed evolution platform, Adverum generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with Regeneron Pharmaceuticals to research, develop, and commercialize gene therapy products for ophthalmic diseases, and Editas Medicine to explore the delivery of genome editing medicines for the treatment of inherited retinal diseases. Adverum’s core capabilities include clinical development and in-house manufacturing expertise, specifically in process development and assay development. www.adverum.com

AGTC
Sue Washer, President and CEO
Alachua, FL
(NASDAQ: AGTC)
AGTC is a clinical-stage biotechnology company that uses a proprietary gene therapy platform to develop transformational genetic therapies for patients suffering from rare and debilitating diseases. Its initial focus is in the field of ophthalmology, where it has active clinical trials in X-linked retinitis pigmentosa (XLRP), achromatopsia (ACHM CNGB3 & ACHM CNGA3), and X-linked retinoschisis (XLRS). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS), other ophthalmology and otology indications. The optogenetics program is being developed in collaboration with Bionic Sight. In addition to its product pipeline, AGTC has a significant intellectual property portfolio and extensive expertise in the design of gene therapy products including capsids, promoters and expression cassettes, as well as expertise in the formulation, manufacture and physical delivery of gene therapy products. www.agtc.com

ANDALUSIAN INITIATIVE FOR ADVANCED THERAPIES
Roke Iñaki Oruezabal-Guijarro, Head of Innovation and Development
Sevilla, Spain
(Private)
Andalusian Initiative for Advanced Therapies is an initiative by the Government of Andalusia, created in October 2008 with the mission of promoting the development of new therapies in order to improve the population’s health and incorporating advanced therapies in Andalusia as an innovative element in the healthcare and as progress of the region. Being part of the Andalusian Public Healthcare System as well as a strategy of the Regional Ministry of Economy and Knowledge and the Regional Ministry of Employment, Enterprise and Trade, the Coordination Unit of the Andalusian Initiative for Advanced Therapies acts as an interface between academia and industry, facilitating translational research, knowledge, and technology transfer in this field. www.juntadeandalucia.es/terapiasavanzadas

ASPECT BIOSYSTEMS
Tamer Mohamed, President and CEO
Vancouver, Canada
(Private)
Aspect Biosystems is a privately-held biotechnology company operating at the leading edge of 3D bioprinting and tissue engineering. The company’s proprietary Lab-on-a-Printer™ platform technology is enabling advances in understanding fundamental biology, disease research, development of novel therapeutics, and regenerative medicine. In addition to its internal programs, Aspect is focused on strategically partnering with pharmaceutical and biotechnology companies, as well as academic researchers, to create physiologically and commercially relevant tissues. These tissues are used to advance and accelerate the discovery and development of new drugs and therapies. www.aspectbiosystems.com

AUDENTES THERAPEUTICS
Matt Patterson, Chairman and CEO
San Francisco, CA
(NASDAQ: BOLD)
Audentes Therapeutics (Nasdaq: BOLD) is a biotechnology company focused on developing and commercializing innovative gene therapy products for patients living with serious, life-threatening rare diseases. The company is currently conducting Phase I/II clinical studies of its lead product candidates, AT132 for the treatment of X-linked Myotubular Myopathy (XLMTM), and AT342 for the treatment of Crigler-Najjar syndrome. The company has two additional product candidates in development, including AT982 for the treatment of Pompe disease, and AT307 for the treatment of the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia (CASQ2-CPVT). They are a focused, experienced, and passionate team committed to forging strong, global relationships with the patient, research, and medical communities. www.audentestx.com

AVECTAS
Michael Maguire, Ph.D., CEO
Maynooth, Ireland
(Private)
Avectas developed Solupore® a clinical grade ex vivo cell engineering technology initially validated through partnership and licensing agreements with cell therapy companies. In parallel, Avectas is embarking on an ‘own product’ strategy through strategic placement of the technology in leading immunology academic centers, in-licensing and acquisition. Solupore® delivers one or more molecular cargos into cells with minimal perturbation and high efficiency without the known downsides of other methods. Solupore® is highly effective for delivering gene editing constructs and CAR-type surface protein engineering. The resulting cells are phenotypically better than cells modified by other delivery systems and proliferate similar to unmodified cells. A scaled system is in advanced development for clinical use. www.avectas.com

AXOVANT
Pavan Cheruvu, M.D.,CEO
Basel, Switzerland
(NASDAQ: AXON)
Axovant is a clinical-stage gene therapy company focused on developing a pipeline of innovative product candidates for debilitating neurological and neuromuscular diseases such as Parkinson’s disease, oculopharyngeal muscular dystrophy (OPMD), amyotrophic lateral sclerosis (ALS), frontotemporal dementia, and other indications. The company leverages multiple, innovative gene therapy approaches to address the underlying biology of diseases for potentially transformative effects for patients. Axovant is driven by the needs of patients to develop these gene therapies with a focus on rapid and high-quality clinical execution. www.axovant.com

BIOSTAGE
William Fodor, Ph.D., Chief Scientific Officer
Holliston, MA
(OTCMKTS: BSTG)
Biostage is a preclinical biotechnology company focused on tissue engineering and the production of combination products designed to restore function of the GI tract following resection and reconstruction. Biostage’s expertise in material sciences, polymer electrospinning and stem cell biology uniquely position Biostage in the tissue engineering and biotechnology industry. Large animal proof of concept studies, large animal GLP acute and chronic toxicology studies and the first in human case using Biostage’s lead product candidate, Cellspan Esophageal Implant (CEI), have demonstrated the feasibility, safety, and efficacy of their technology. www.biostage.com

BIOTIME
Brian Culley, CEO
Alameda, CA
(NYSE: BTX)
BioTime is a clinical-stage biotechnology company focused on the development and commercialization of novel therapies for the treatment of degenerative diseases. BioTime’s pipeline is based on two platform technologies which encompass cell replacement and cell/drug delivery. BioTime’s lead cell replacement product candidate is OpRegen®, a retinal pigment epithelium transplant therapy in Phase II development for the treatment of dry age-related macular degeneration, the leading cause of blindness in the developed world. BioTime’s lead cell delivery clinical program is Renevia®, an investigational medical device being developed as an alternative for whole adipose tissue transfer procedures. BioTime also has significant equity holdings in two publicly traded companies, Asterias Biotherapeutics (NYSE American: AST), and OncoCyte Corporation (NYSE American: OCX), and a private company, AgeX Therapeutics. www.biotimeinc.com

BLUEBIRD BIO
Jeffrey Walsh, Chief Financial and Strategy Officer
Cambridge, MA
(NASDAQ: BLUE)
With its lentiviral-based gene therapies, T-cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D™ product candidate for the treatment of cerebral adrenoleukodystrophy and its LentiGlobin™ BB305 product candidate for the treatment of transfusion-dependent β-thalassemia and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T-cell engineering. bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR-T program partnered with Celgene. bluebird bio also has discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. www.bluebirdbio.com

BLUEROCK THERAPEUTICS
Emile Nuwaysir, Ph.D., President and CEO
Cambridge, MA
(Private)
BlueRock Therapeutics is an engineered cell therapy company with a mission to develop regenerative medicines for intractable diseases. BlueRock’s cell and gene platform harnesses the power of cells for new medicines across neurology, cardiology and autoimmune indications. BlueRock’s cell differentiation technology recapitulates the cell’s developmental biology to produce native cell therapies which are further engineered for additional function. Utilizing these cell therapies to replace damaged or degenerated tissue brings the potential to restore or regenerate lost function. BlueRock was founded in 2016 by Versant Ventures and capitalized with one of the largest-ever Series A financings in biotech history by Bayer AG and Versant. BlueRock’s culture is defined by scientific innovation, the highest ethical standards and an urgency to bring transformative treatments to all who would benefit. www.bluerocktx.com

B-MOGEN BIOTECHNOLOGIES
David Largaespada, Ph.D., Chairman
Minneapolis, MN
(Private)
B-MoGen Biotechnologies is a genome engineering company established to address complex cell engineering challenges in order to accelerate the study of human diseases and the development of novel therapies. The B-MoGen team is equipped with proprietary tools and knowledge that have been developed into finely honed methods for efficient cellular engineering. The company utilizes this unique expertise to provide three distinct services that address highly complex gene delivery and gene editing. They offer a non-viral transposon-based gene delivery platform to support the T-cell immunotherapy industry; they are the first company in the world to demonstrate genome engineering of the mitochondria genome; and they offer fee-for-service custom cell engineering of both cell lines and, uniquely, primary human lymphohematopoietic cells for cancer research, drug screening, and antibody validation. www.bmogen.com

BONE THERAPEUTICS
Thomas Lienard, CEO
Gosselies, Belgium
(Euronext Brussels and Paris: BOTHE)
Bone Therapeutics is a leading cell therapy company addressing high unmet medical needs in the field of orthopedics and bone diseases. The company has a broad, diversified portfolio of bone cell therapy solutions and viscosupplement in clinical development across a number of disease areas targeting markets with large unmet medical needs and limited innovation. The company’s technology is based on a unique, proprietary approach to bone regeneration which turns undifferentiated stem cells into “osteoblastic”, or bone-forming cells. These cells can be administered via a minimally invasive procedure, avoiding the need for invasive surgery. The company’s primary clinical focus is ALLOB, an allogeneic “off-the-shelf” cell therapy product derived from stem cells of healthy donors, which is in Phase II studies for the treatment of delayed-union fractures and spinal fusion. In addition, Bone Therapeutics is also developing an enhanced viscosupplement, JTA-004, for the treatment of knee osteoarthritis. www.bonetherapeutics.com

CARIBOU BIOSCIENCES
Timothy Herpin, Ph.D., Chief Business Officer
Berkeley, CA
(Private)
Caribou is a leading company in CRISPR genome editing founded by pioneers of CRISPR-Cas9 biology and using a next-generation gene editing technology to develop a pipeline of off-the-shelf CAR-T candidates and microbiome-based therapies. Caribou’s tools and technologies provide transformative capabilities to therapeutic development, agricultural biotechnology, industrial biotechnology, and basic and applied biological research. www.cariboubio.com

CELLERANT THERAPEUTICS
Ram Mandalam, Ph.D., President and CEO
San Carlos, CA
(Private)
Cellerant Therapeutics is a clinical-stage company developing innovative cell- and antibody-based immunotherapies for hematologic malignancies and other blood-related disorders. We have two products in development. Our lead product, romyelocel-L, is a universal cell therapy to prevent infections during prolonged neutropenia. We plan to initiate a Phase III trial for romyelocel-L in 2019, and the U.S. Food & Drug Administration (FDA) has granted romyelocel-L Regenerative Medicine Advanced Therapy (RMAT) designation. Our second product, CLT030 is an antibody-drug conjugate in preclinical development to treat AML by targeting and killing leukemic stem cells, which are believed to be the cause of relapse in patients. CLT030 is directed at the C-type lectin-like molecule-1 (CLL1), which is highly expressed on leukemic stem cells, but not on normal hematopoietic stem or progenitor cells. www.cellerant.com

CHIMERIC THERAPEUTICS
Jacqueline Barry, Ph.D., Chief Clinical Officer
London, UK
(Private)
Chimeric Therapeutics is a virtual company operated by the Cell and Gene Therapy Catapult, which is developing a novel autologous CAR-T therapy targeting CLEC14A for the treatments of patients with advanced malignancies expressing the tumor endothelial marker CLEC14A. www.ct.catapult.org.uk

CRYOPORT
Mark Sawicki, Ph.D., Chief Commercial Officer
Irvine, CA
(NASDAQ: CYRX)
Cryoport is the life sciences industry’s most trusted global provider of temperature-controlled logistics solutions for temperature-sensitive life sciences commodities, serving the biopharmaceutical market with leading-edge logistics solutions for biologic materials, such as regenerative medicine, including immunotherapies, stem cells and CAR-T cells. Cryoport’s solutions are used by points-of-care, CRO’s, central laboratories, pharmaceutical companies, manufacturers and university researchers. Cryoport’s proprietary Cryoport Express® Shippers, Cryoportal® Logistics Management Platform, leading-edge SmartPak II™ Condition Monitoring System and geo-sensing technology, paired with unparalleled cold chain logistics expertise and 24/7/365 client support, make Cryoport the end-to-end cold chain logistics partner that the industry trusts. www.cryoport.com

CYNATA THERAPEUTICS
Ross Macdonald, Ph.D., CEO
Melbourne, Australia
(ASX:CYP)
Cynata Therapeutics Limited is an Australian clinical-stage cell therapy company focused on the development of products based on Cymerus™, a proprietary therapeutic stem cell platform technology. Cymerus overcomes the challenges of other production methods by using induced pluripotent stem cells (iPSCs) to achieve economic manufacture of cell therapy products, including mesenchymal stem cells (MSCs), at commercial scale and without the limitation of multiple donors. Cynata’s lead product candidate CYP-001 met all clinical endpoints and demonstrated positive safety and efficacy data for the treatment of steroid-resistant acute graft-versus-host disease (GvHD) in a Phase I trial. Cynata plans to advance its Cymerus MSCs into Phase II trials for GvHD and critical limb ischemia. In addition, Cynata has demonstrated utility of its Cymerus MSC technology in preclinical models of asthma, critical limb ischemia, diabetic wounds, heart attack and cytokine release syndrome, a life-threatening condition stemming from cancer immunotherapy. www.cynata.com

DISCGENICS
Flagg Flanagan, Chairman and CEO
Salt Lake City, UT
(Private)
DiscGenics is a privately-held, clinical-stage regenerative medicine company focused on developing cell therapies that alleviate pain and restore function in patients with degenerative diseases of the spine. DiscGenics is harnessing the restorative potential of intervertebral disc cells to develop therapies that the company believes will have a profound impact on millions suffering from the debilitating effects of back pain. The company’s first product candidate, IDCT, is a homologous, allogeneic (off-the-shelf), injectable cell therapy for the treatment of patients with degenerative disc disease (DDD). www.discgenics.com

FUJIFILM CELLULAR DYNAMICS
Nicholas Manusos, SVP, Strategy and Business Development
Madison, WI
(Private)
Cellular Dynamics International (CDI), a FUJIFILM company, produces human cells for basic, translational and pharmaceutical discovery and regenerative medicine applications. The company’s iPSC-derived iCell and MyCell products provide immediate access to healthy and diseased cellular models, such as neurons, cardiomyocytes and hepatocytes, to meet small- and large-scale needs. www.fujifilmcdi.com

GENETHON
Frederic Revah, Ph.D., CEO
Evry, France
(Private)
Created in 1990 by the AFM-Telethon and the French Muscular Dystrophy Association, Genethon is a non-profit research and development organization dedicated to the development of gene therapy for orphan genetic diseases from research to clinical validation. Genethon has multiple ongoing programs at clinical, preclinical and research stage, led alone or in partnership with external academic or biotech partners for neuromuscular, blood, immune system, liver, and eye diseases.
www.genethon.fr/en

HEALIOS
David Smith, Chief Commercial Officer
Tokyo, Japan
(JPX 4593.T)
Healios is a biotechnology venture leading the field of developing iPS cell-based products for regenerative medicine. It was founded in 2011 and listed on the stock exchange in 2015. In Japan, the company is developing a product for the treatment of age-related macular degeneration jointly with Suimitomo Dainippon Pharma under the plan of obtaining approval of its manufacture and distribution in 2020. In fields other than ophthalmology, the company has started research and development of products for regenerative medicine capable of creating functional human organs jointly with Yokohama City University. www.healios.co.jp/en

INVITRX THERAPEUTICS
Habib Torfi, CEO
Irvine, CA
(Private)
Invitrx Therapeutics is a global research-based life science company with more than 10 years of experience in the harvesting and isolation of stem cells in order to discover, develop, manufacture, and commercialize innovative products. Although Invitrx Therapeutics is based in California it has subdivisions throughout Asia. The introduction of regenerative medicine to the Asian market has been in development by Invitrx for nearly a decade. Invitrx Therapeutics prime focus is in advancing the field of regenerative medicine through scientific research and development. Invitrx Therapeutics is built upon a team with expertise in each zone of regenerative medicine including scientific, clinical, and regulatory affairs. www.invitrx.com

IOVANCE BIOTHERAPEUTICS
Maria Fardis, Ph.D., President and CEO
San Carlos, CA
(NASDAQ: IOVA)
Iovance Biotherapeutics is working to treat cancer by developing immuno-oncology Tumor Infiltrating Lymphocytes (TIL) therapies that harness a patient’s own immune system. Iovance is focused on the development and commercialization of autologous cellular immunotherapies optimizing personalized TIL for the treatment of solid tumors. The company’s TIL technology is designed to address the various obstacles that reduce the natural anti-tumor immune response in the body. The company is conducting four Phase II clinical trials to assess the efficacy and safety of TILs for treatment of patients with metastatic melanoma, squamous cell carcinoma of the head and heck, metastatic non-small cell lung cancer (NSCLC), and cervical cancer. The company is also studying TIL therapy in ovarian cancer and sarcomas. The company aims to change the cancer treatment paradigm by developing and optimizing TIL therapy to help support the patient’s own immune system in fighting various cancers. www.iovance.com

KIADIS PHARMA
Arthur Lahr, CEO
Amsterdam, Netherlands
(Euronext Amsterdam and Brussels: KDS)
Kiadis is developing its lead product candidate, ATIR101, for use in conjunction with haploidentical hematopoietic stem-cell transplantations for adult blood cancers. Based on the positive results from a Phase II study, the company submitted an MAA to the EMA in 2017. If the product is approved in H1 2019, Kiadis intends to launch ATIR101 in a first EU member state in H2 2019. In December 2017, Kiadis Pharma commenced a Phase III trial against the Post-Transplant Cyclophosphamide protocol. ATIR101 has received RMAT designation from the FDA and been granted orphan drug designations both in the EU and U.S. www.kiadis.com

LOGICBIO
Frederic Chereau, President and CEO
Cambridge, MA
(NASDAQ: LOGC)
LogicBio is a genome editing company that began operations in 2015 to develop medicines to deliver the potential benefits of genetic medicine in the fight against rare diseases in pediatric patients. The company’s scientific co-founders are global leaders in gene therapy and gene editing. It was their research at Stanford University that led to the development of the company’s foundational technology platform GeneRide. GeneRide is designed to precisely and stably integrate corrective genes into a patient’s genome to provide a durable therapeutic benefit from a single treatment. The company’s technology harnesses a natural DNA repair process called homologous recombination, which they believe will enable the insertion of therapeutic genes into specific locations in the chromosome without the use of exogenous nucleases (“molecular scissors” that are engineered to cut DNA), which are used in other gene editing technologies. www.logicbio.com

LONGEVERON
Joshua Hare, M.D., Co-Founder and Chief Scientific Officer
Miami, FL
(Private)
Longeveron is a cellular therapy company developing biological solutions for aging and aging-associated diseases through the use of Allogeneic Human Mesenchymal Stem Cells, harvested and grown at their facility in Miami, Florida, from adult-donor bone marrow. The Longeveron-grown Mesenchymal Stem Cells (LMSCs) are multipotent stem cells. Longeveron has received nearly $8M in grant funding from NIH, Maryland Stem Cell Research Fund and the Alzheimer’s Association in addition to private investment. They are conducting Phase I and Phase IIb clinical trials, and plan to be the first to offer stem cells as a safe, effective, FDA-approved treatment for conditions such as Aging Frailty, Alzheimer’s Disease and Metabolic Syndrome. The company also plans to initiate a trial in Japan in 2019. www.longeveron.com

LONZA PHARMA & BIOTECH
Alberto Santagostino, Business Unit Head of Cell and Gene Technologies
Basel, Switzerland
(SWX: LONN)
Lonza Pharma & Biotech provides contract development and manufacturing services that enable pharma and biotech companies to bring medicines to patients in need. From the building blocks of life to the final drug product, their solutions are created to simplify the outsourcing experience and provide a reliable outcome, at the time it is expected. The company’s extensive track record includes commercialization of pioneering therapies and manufacturing of a wide variety of biological and chemical drugs. They continuously invest to solve not just the current, but also the future challenges. www.lonza.com

MAXCYTE
Debra Bowes, Chief Business Officer
Gaithersburg, MD
(LSE: MXCR)
MaxCyte is a global cell-based medicines and life sciences company applying its proprietary cell engineering technology platform to deliver the advances of cell-based medicine to patients with high unmet medical needs. MaxCyte is developing novel CARMA™ therapies for its own pipeline. CARMA is MaxCyte’s mRNA-based proprietary therapeutic platform for rapid autologous cell therapy for the treatment of solid cancers. In addition, through its core business, MaxCyte leverages its Flow Electroporation® Technology to enable its biopharmaceutical partners to advance the development of innovative medicines, particularly in cell therapy. MaxCyte has placed its flow electroporation instruments worldwide, with all of the top ten global biopharmaceutical companies, has more than 70 partnered program licenses in cell therapy including more than 35 licensed for clinical use. With its robust delivery technology platform, MaxCyte helps its partners to unlock the full potential of their products. www.maxcyte.com

MEIRAGTX
Zandy Forbes, Ph.D., CEO
New York, NY
(NASDAQ: MGTX)
MeiraGTx is a vertically integrated, clinical-stage gene therapy company with four ongoing clinical programs and a broad pipeline of preclinical and research programs. MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team, MeiraGTx has taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTx’s initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, severe forms of xerostomia and neurodegenerative diseases. Though initially focusing on the eye, salivary gland and central nervous system, MeiraGTx intends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases. www.meiragtx.com

MOLMED
Luca Alberici, Ph.D., Chief Business Officer
Milan, Italy
(MLM.MI)
MolMed S.p.A. is an Italian biotechnology cell and gene company, focused on research, development, manufacturing and clinical validation of innovative therapies in oncology and rare diseases. MolMed built an original dual business model, based on research and development of proprietary products, and third party GMP services. MolMed is currently the developer and manufacturer for worldwide leaders in the field of gene therapy for rare diseases and allogenic CAR-T therapies supporting their clinical trials and commercial supply. MolMed, founded in 1996 as an academic spin-off of the San Raffaele Scientific Institute, is listed on the main market (MTA) of the Milan stock exchange and has been managed by Borsa Italiana since March 2008. MolMed is headquartered and based in Milan, at the San Raffaele Biotechnology Department and has an operating unit at OpenZone in Bresso. www.molmed.com

NAVAN TECHNOLOGIES
Michael Nowak, Ph.D., CEO
San Francisco, CA
(Private)
NAVAN Technologies is an enabling technology company commercializing a novel non-viral delivery platform to radically accelerate genetic engineering of human primary cells. The Nanostraw technology, exclusively licensed from Stanford University, provides direct intracellular access into hard-to-transfect primary human cells for efficient delivery of any cargo. By using a physical, non-perturbative mechanism for delivery, the challenges of viral-based cell manufacturing (time, cost, and technical difficulties) are eliminated, resulting in fewer, faster operational steps. NAVAN’s vision is to facilitate cell and gene therapies at the point of care. With the capability to streamline new therapy development and robust performance at scale, Nanostraws will greatly decrease costs, making clinical applications more affordable and accessible. www.navan-tech.com

ORCHARD THERAPEUTICS
Mark Rothera, CEO
Boston, MA
(NASDAQ: ORTX)
Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies. Orchard’s advanced portfolio of autologous ex vivo gene therapies targets serious and life-threatening rare diseases, initially primary immune deficiencies, neurometabolic disorders and hemoglobinopathies. Orchard’s gene therapy pipeline includes Strimvelis®, the first autologous ex vivo gene therapy approved by the EMA in 2016, three programs in advanced registrational studies in MLD (metachromatic leukodystrophy), WAS (Wiskott–Aldrich syndrome), and ADA-SCID (adenosine deaminase severe combined immunodeficiency), two other clinical programs in X-CGD (X-linked chronic granulomatous disease) and beta-thalassemia, as well as an extensive preclinical pipeline. Orchard has developed a unique expertise in the manufacturing, preclinical and clinical development of gene therapies for rare diseases. To date, more than 150 patients have been treated across five different disease areas, with evidence of sustained clinical effects up to 18 years. www.orchard-tx.com

ORGANOVO
Steven Hughes, M.D., Chief Medical Officer
San Diego, CA
(NASDAQ: ONVO)
Organovo is a biotechnology company pioneering a unique set of therapeutic and drug profiling capabilities based on its revolutionary ability to 3D bioprint tissues that emulate human biology and disease. They are developing in vivo liver tissues to treat a range of rare, life-threatening diseases, for which there are few current treatment options other than organ transplantation. Organovo’s program which focuses on a rare disease known as Alpha-1-antitrypsin deficiency received orphan drug designation from the FDA in December 2017 and is targeted for an Initial New Drug Application filing in calendar-year 2020. They are also capitalizing on our foundational ability to isolate highly specialized human cells to build robust, functional human tissues by creating a range of novel preclinical in vitro disease modeling platforms, including a broad set of non-alcoholic fatty liver disease and non-alcoholic steatohepatitis conditions. www.organovo.com

OXFORD BIOMEDICA
Jason Slingsby, Ph.D., Chief Business Officer
Oxford, UK
(LSE:OXB)
Using the company’s unique LentiVector® delivery platform, Oxford BioMedica have created a valuable portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology and CNS disorders. The company strong partnerships with Novartis, Bioverativ (part of the Sanofi group), Immune Design, Boehringer Ingelheim/UK Cystic Fibrosis Gene Therapy Consortium, and Orchard Therapeutics, providing them with access to they company’s intellectual property, state-of-the-art production facilities and expertise, and, in addition, they have licensed products and technology rights to Sanofi and Axovant. These partnerships provide Oxford BioMedica with multiple income streams, consisting of upfront milestone payments, development and production fees and potential royalties on future product sales. They plan to progress their wholly-owned products via spin-outs and out-licensing opportunities, while continuing to invest in their LentiVector® platform. The company plans to continue preclinical research and development to discover new potential products. www.oxb.com

PDC*LINE PHARMA
Eric Halioua, President and CEO
Liege, Belgium
(Private)
Founded in 2014 as a spin-off of the French Blood Bank (EFS), PDC*line Pharma is a Belgian biotech company that develops an innovative class of active immunotherapies for cancers, based on an allogeneic antigen presenting cell line (PDC*line). PDC*line is a new potent and scalable therapeutic cancer vaccine based on a proprietary allogeneic cell line of Plasmacytoid Dendritic Cells. PDC*line is much more potent to prime and boost antitumor antigen, including neoantigens, specific cytotoxic T-cells than conventional vaccines and improves the response to checkpoint inhibitors. The technology can be applied for any type of cancer. Based on a first-in-human Phase I study in melanoma, PDC*line Pharma is focused on the development of a candidate for lung cancer (PDC*lung) and neoantigens (PDC*Neo). The company has a seasoned management team and has raised more than 15M€ in equity and loans. www.pdc-line-pharma.com

POLARITYTE
Denver Lough, M.D., Ph.D., CEO
Salt Lake City, UT
(NASDAQ: PTE)
PolarityTE® is a commercial-stage biotechnology and regenerative biomaterials company focused on transforming the lives of patients by discovering, designing, and developing regenerative tissue products and biomaterials for medicine, biomedical engineering, and material sciences. The PolarityTE platform technology begins with a small piece of the patient’s own, or autologous, healthy tissue rather than artificially manipulated individual cells. From this small piece of healthy autologous tissue, the company creates an easily deployable, dynamic and self-propagating product designed to enhance and stimulate the patient’s own cells to regenerate the target tissues. SkinTE™ is commercially available and uses this technology to regenerate skin using a small piece of the patient’s own skin to treat full-thickness burns, chronic wounds, and large cutaneous defects. Its ease of use and one-time application allows its use in outpatient and resource-limited settings while reducing treatment costs and, most importantly, providing the patient with full-thickness skin. www.polarityte.com

PRECISION BIOSCIENCES
Michael Dombeck, VP, Business Development
Durham, NC
(Private)
Precision BioSciences is a biotechnology company dedicated to improving life with its next generation gene editing technology, ARCUS®, a proprietary, wholly-owned non-CRISPR editing platform with applications in gene therapy, cell therapy, and agriculture. Precision’s allogeneic cell therapy pipeline includes a broad collection of engineered T-cells, with its lead CD19 CAR-T on schedule to initiate clinical testing by 2019. Precision’s gene therapy pipeline includes multiple in vivo liver, eye, and CNS gene editing therapeutic candidates, based on the ability of ARCUS nucleases to efficiently edit desired target sites in vivo, with systemic therapeutic efficacy demonstrated in non-human primates. The value of Precision’s ARCUS editing platform is enhanced by our extensive patent portfolio, established freedom to operate, and significant partnerships with industry leaders, positioning Precision to lead the development and commercialization of advanced gene editing products that solve important problems in oncology, genetic disease, agriculture, and beyond. www.precisionbiosciences.com

PROMETHERA BIOSCIENCES
Etienne Sokal, Ph.D., Chief Scientific and Medical Officer
Mont-Saint-Guibert, Belgium
(Private)
Promethera Biosciences is a global innovator in liver therapeutics whose mission is to enable patients to overcome acute and chronic liver diseases. The company’s lead clinical program, derived from its patented cell technology platform HepaStem, is designed to benefit from its immune-modulatory and anti-fibrotic properties. In addition to the company’s cell-based pipeline, they develop antibody technologies, such as the anti-TNF-R1 antibody Atrosimab, to complement and diversify their therapeutic options. The company is a team of international experts operating out of facilities in Mont-Saint-Guibert, Belgium, Durham, North Carolina, Tokyo, Japan, and Basel, Switzerland. www.promethera.com

REGENXBIO
Ram Palanki, SVP, Commercial Strategy and Operations
Rockville, MD
(NASDAQ: RGNX)
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. The company’s gene therapy product candidates are designed to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins that are intended to impact disease. Through a single administration, the product candidates are designed to provide long-lasting effects, potentially significantly altering the course of disease and delivering improved patient outcomes. The company is currently developing gene therapy product candidates for the treatment of retinal, metabolic, and neurodegenerative diseases. The company’s product candidates all utilize AAV viral vectors from their proprietary gene delivery platform, which is called NAV Technology Platform. In addition to their internal product candidate programs, the company is also selectively licensing its NAV Vectors to other leading biotechnology companies. www.regenxbio.com

RENEURON
Michael Hunt, Chief Financial Officer
Pencoed, South Wales
(LON: RENE)
ReNeuron is a leading, clinical-stage cell therapy development company. Based in the UK, ReNeuron has therapeutic candidates in clinical development for disability as a result of stroke and for the blindness-causing disease, retinitis pigmentosa. ReNeuron is also advancing its proprietary exosome technology platform as a potential delivery system for drugs that would otherwise be unable to reach their site of action. www.reneuron.com

REXGENERO
Joe Dupere, Ph.D., CEO
London, UK
(Private)
Rexgenero is a clinical-stage regenerative medicine company developing innovative cell-based therapies targeting Critical Limb Ischemia (CLI), a serious disease with a high risk of amputation and death. The company’s lead candidate REX-001 is a highly innovative autologous cell therapy that is being studied in two Phase III clinical trials in patients with CLI with diabetes. REX-001 has shown to be effective in Phase II trials, alleviating CLI in the majority of patients, offering the potential to increase the quality of life by reducing pain, alleviating ulcers, increasing mobility, improving sleep and reducing the need for amputation. Rexgenero is planning to develop REX-001 in a range of indications. Rexgenero draws on an exceptional understanding of the fundamental science of cell therapies developed by the Andalusian Health Authority and the Andalusian Initiative of Advanced Therapies. Rexgenero is headquartered in London with research and development, and manufacturing operations in Seville, Spain. www.rexgenero.com

SANGAMO THERAPEUTICS
Stephane Boissel, EVP, Corporate Strategy
Richmond, CA
(NASDAQ: SGMO)
Sangamo Therapeutics is focused on translating ground-breaking science into genomic therapies that transform patients’ lives using the company’s platform technologies in genome editing, gene therapy, gene regulation, and cell therapy. www.sangamo.com

SERAXIS
William Rust, Ph.D., CEO
Germantown, MD
(Private)
Seraxis is a private regenerative medicine company with the mission of bringing cell replacement therapies to patients in need. With several products in development, its lead product, SR-01, is a novel combination product for type 1 diabetes with an IND application anticipated for 2020. SR-01 comprises of lab-grown human pancreatic islets encased within a novel bi-layered encapsulation device that protects the cells from cytotoxic alloimmune response. The Seraxis researchers are able to create functionally mature human insulin-secreting islets at a level and purity never before reported through a combination of a proprietary iPS cell line and a novel cell differentiation protocol. The retrievable encapsulation device employs biocompatible components and enables the cells to have close association to the host vasculature to physiologically regulate blood glucose. These proprietary technologies have been developed using GMP-processes and are characterized for clinical use. www.seraxis.com

SIGILON THERAPEUTICS
Devyn Smith, Ph.D., Chief Strategy Officer and Head of Operations
Cambridge, MA
(Private)
For adaptive, programmable therapeutics to be effective over extended periods, cells and objects placed in the body must avoid fibrosis caused by the foreign body response of the immune system. The Afibromer™ biomaterials of the Sigilon platform avoid the fibrotic response, allowing engineered cells implanted in the body to deliver therapeutic molecules for extended periods of time, acting as living therapeutics. These engineered cells can either deliver molecules at a constant rate or be programmed using the techniques of synthetic biology to respond to changing conditions, such as intermittent flare-ups of disease symptoms or fluctuating blood levels of specific factors like glucose. Synthetic biology tools and programmable genetic circuits create new possibilities for the treatment of complex diseases. Sigilon therapeutics can be programmed to sense changing conditions and respond appropriately. This is unlike gene therapies or RNA interference, which cannot be adjusted once they are introduced into the body. www.sigilon.com

SPARK THERAPEUTICS
Dan Faga, Chief Business Officer
Philadelphia, PA
(NASDAQ: ONCE)
Spark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, challenges the inevitability of genetic diseases, including blindness, hemophilia, lysosomal storage disorders, and neurodegenerative diseases. The company has successfully applied its technology in the first FDA-approved gene therapy in the U.S. and EU for a genetic disease, and currently has three programs in clinical trials, including product candidates that have shown promising early results in patients with hemophilia. Spark sees the path to a world where no life is limited by genetic disease. www.sparktx.com

TERUMO BCT
Antoinette Gawin, CEO
Lakewood, CO
(Private)
Terumo BCT, a global leader in blood component, therapeutic apheresis, and cellular technologies, believes in the potential of blood and cells to do even more for patients than they do today. Terumo BCT’s Cell Therapy Technologies business enables researchers, developers, and manufacturers to create next-generation cell and gene therapies. The company does this through flexible, automated solutions that help meet your evolving needs for reproducibility, quality, and scale through the phases of development, from translational research to current good manufacturing practice (cGMP) for commercial manufacturing. Terumo BCT is also looking further downstream in the process to provide innovative solutions, like the FINIA Fill and Finish System, to support your needs into the future. www.terumobct.com

TIKOMED
Adam Bruce, Executive Board Member
Viken, Sweden
(Private)
TikoMed AB is an investment/holding company that develops projects through daughter companies. The company’s lead product candidate, IBsolvMIR, prevents IBMIR and promotes engraftment of transplanted cells. Consequently, IBsolvMIR enables and ensures efficacy of the therapy given, minimizes COGS and improves the medical outcome. IBsolvMIR has undergone a Phase II study in Clinical Islet transplantation a follow-up study will start in early 2019 to document a higher dose. www.tikomed.com

TMUNITY THERAPEUTICS
Usman Azam, M.D., President and CEO
Philadelphia, PA
(Private)
Tmunity Therapeutics is a privately-held clinical stage biotherapeutics company delivering the potential of next-generation T-cell immunotherapies to patients with cancer. Tmunity is a unique and differentiated company, by virtue of a unique model that incorporates cutting edge technology from the University of Pennsylvania and coupling with groundbreaking scientific, clinical, and manufacturing expertise. Tmunity’s scientific founders and management have demonstrated a track record of having developed first-in-class T-cell technologies and platforms. The company is developing a diversified portfolio of novel treatments that exhibit best-in-class control over T-cell activation and direction in the body. These personalized immunotherapies for cancer are advancing rapidly in the clinic. With headquarters in Philadelphia, Tmunity also has laboratories dedicated cGMP manufacturing facility in Norristown, PA, in support of clinical development of T-cell therapies. www.tmunity.com

UNIQURE
Jon Garen, Chief Business Officer
Lexington, MA
(NASDAQ: QURE)
uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. The company has developed a modular technology platform to rapidly bring new disease-modifying therapies to patients with severe genetic diseases. They are advancing a focused pipeline of innovative gene therapies and have established clinical proof-of-concept in the company’s lead indication, hemophilia B, and preclinical proof-of-concept in Huntington’s disease. uniQure’s pipeline of adeno-associated virus (AAV)-based gene therapies has been developed using an innovative technology platform, supported by industry-leading proprietary commercial-grade manufacturing capabilities. Through recent collaborations and a strategic partnership with Bristol-Myers Squibb to develop gene therapies for cardiovascular diseases, the company has taken the next steps toward developing gene therapies targeting chronic and degenerative diseases that affect larger populations. www.uniqure.com

VERIGRAFT
Petter Bjorquist, Ph.D., CEO
Gothenburg, Sweden
(Private)
VERIGRAFT is a small, innovative biotech company located in Gothenburg, Sweden. VERIGRAFT is developing personalized tissue-engineered transplants for use in regenerative medicine, avoiding transplant rejection and the need for life-long immunosuppression. A donated, allogenic tissue is patient-individualized by a combination of decellularization and reconditioning / recellularization processes. The resulting tissues have the capacity to be a game-changer for treatment of severe, chronic diseases. VERIGRAFT’s personalized tissue-engineered vein (P-TEV) has gone through careful preclinical development at the company. Safety data from large animals has been generated, a GMP-compliant manufacturing process has been validated, and the company is currently waiting for approval of an application for clinical trial authorization (CTA) in order to start a first-in-man Phase I safety trial. VERIGRAFT’s goal is to deliver a life-changing impact to patients and to provide major benefits for hospitals, healthcare providers and the society. www.verigraft.com

VIVET THERAPEUTICS
Jean-Philippe Combal, Ph.D., Co-Founder and CEO
Paris, France
(Private)
Vivet Therapeutics is a gene therapy biotech company dedicated to the research, development, and future commercialization of gene therapy products for inherited liver disorders with high medical need. VTX-801, Vivet’s lead gene therapy program, targets Wilson’s Disease, and the company has four additional gene therapy programs under preclinical development. In addition, Vivet is working on new technologies to address key challenges of AAV-based gene therapy products related to sustainable expression in young patients and immunomodulation for potential retreatment. www.vivet-therapeutics.com/en

ZELLUNA IMMUNOTHERAPY
Miguel Forte, M.D., Ph.D., CEO
Oslo, Norway
(Private)
Zelluna Immunotherapy is developing innovative T-cell receptor (TCR) based cancer therapies for patients with a high unmet medical need. The company’s core asset and unique competitive advantage is a large portfolio of TCRs isolated from long term surviving patients from peptide vaccine trials. Our TCRs are directed against epitopes from the known tumor associated antigens TGFβRII, hTERT and K-Ras and have the potential to be used in the treatment of a broad range of solid tumors. Zelluna has a long-term collaborative research and development agreement with the Department of Cell Therapy at the Oslo University Hospital and is in the process of setting up cell manufacturing capabilities in collaboration with MaSThercell. Zelluna aims to start its first clinical trial in 2019-2020. www.zelluna.com

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