Company Presentations

Initial Slate of Presenting Companies

See the initial slate of 40+ leading companies presenting at the Cell & Gene Meeting on the Mediterranean!

This meeting features presentations by leading public and private companies, highlighting technical and clinical achievements over the past 12 months in the areas of cell therapy, gene therapy, gene editing, tissue engineering and broader regenerative medicine technologies.

2020 Company Presenters




ADAPTIMMUNE
Kimberly Freeman, VP, Commercial Planning
Philadelphia, PA
(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. The foundation of the company’s TCR therapies is with NY-ESO SPEAR T-cells, which transitioned to GSK in 2018, showing responses in two solid tumors and treating more than 90 patients in six different indications. Adaptimmune has generated a strong pipeline of affinity-enhanced T-cell therapies, with multiple INDs open. The company uses these therapies to harness the body’s own immune system to find and destroy diseased cells. The company’s SPEAR T-cell therapies offer promise to patients that often have no other options. Adaptimmune is working hard to make that promise a reality. www.adaptimmune.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

 

 


AMBYS MEDICINES

Jeff Jonker, CEO
San Fransisco, CA
(Private)
Ambys Medicines is working to fundamentally transform the lives of people suffering from severe liver diseases by creating breakthrough therapies capable of restoring liver function, preventing the progression of liver disease, and treating the devastating complications of liver failure. The company is pioneering multiple novel technologies — including liver cell therapy, liver reprogramming gene therapies, and liver-targeted drug therapies — to meet the urgent need for treatments that reverse disease progression across multiple liver diseases that are untreatable or poorly treated today. www.ambys.com

ASKBIO
David Venables, Ph.D., President, AskBio Europe
Raleigh, NC
(Private)
Asklepios BioPharmaceutical (AskBio) is a privately held AAV gene therapy company founded in 2001 to advance the work of Dr. Richard Jude Samulski from the lab into the clinic. Dr. Samulski is the former Director of the Gene Therapy Center at the University of North Carolina (UNC) and was the first to clone AAV for therapeutic purposes. Since AskBio’s inception almost two decades ago, Dr. Samulski’s efforts have joined those of a group of widely acclaimed researchers and clinicians. The company has the following competitive advantages relative to others in the AAV space: next generation AAV technology; Synpromics gene control technology; a broad patents and intellectual property portfolio; scaled-up manufacturing; and a broad portfolio of therapeutic programs. www.askbio.com

ASPECT BIOSYSTEMS
Tamer Mohamed, CEO
Vancouver, BC
(Private)
Aspect Biosystems is a privately held biotechnology company operating at the leading edge of 3D bioprinting and tissue engineering. The company’s primary focus is to apply its proprietary microfluidic 3D-printing technology to address significant unmet medical need through the generation of implantable therapeutic tissues that have the potential to change clinical practice. In addition to its internal regenerative medicine programs, Aspect strategically partners with biopharma companies, as well as academic researchers, to create physiologically relevant tissues to model disease, identify novel targets, and perform phenotypic screening with disease-relevant endpoints, thus enable the discovery and development of new drugs and therapies. www.aspectbiosystems.com

ATARA BIOTHERAPEUTICS
Pascal Touchon, President and CEO
San Francisco, CA
(NASDAQ: ATRA)
Atara Biotherapeutics is a leading off-the-shelf, allogeneic T-cell immunotherapy company developing novel treatments for patients with cancer, autoimmune, and viral diseases. Atara’s technology platform leverages research collaborations with leading academic institutions with the company’s scientific, clinical, regulatory, and manufacturing expertise. Atara’s pipeline includes tab-cel® (tabelecleucel), which is in Phase III development for patients with Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV+ PTLD), as well as in earlier stage development for other EBV-associated hematologic malignancies and solid tumors, including nasopharyngeal carcinoma (NPC); T-cell immunotherapies targeting EBV antigens believed to be important for the potential treatment of multiple sclerosis; and next-generation chimeric antigen receptor T-cell (CAR T) immunotherapies. The company was founded in 2012 and is co-located in South San Francisco and Southern California. Atara’s Southern California hub is anchored by the state-of-the-art Atara T-cell Operations and Manufacturing (ATOM) facility in Thousand Oaks. www.atarabio.com

AUTOLUS
Chris Vann, Chief Operating Officer
London, UK
(NASDAQ: AUTL)
Autolus is a clinical-stage biopharmaceutical company developing next-generation, programmed T cell therapies for the treatment of cancer. Using a broad suite of proprietary and modular T cell programming technologies, the company is engineering precisely targeted, controlled, and highly active T cell therapies that are designed to better recognize cancer cells, break down their defense mechanisms, and eliminate these cells. Autolus has a pipeline of product candidates in development for the treatment of hematological malignancies and solid tumors. www.autolus.com

AVECTAS
Michael Maguire, Ph.D., CEO
Maynooth, Ireland
(Private)
Avectas is a cell engineering technology business enabling the manufacture of cell therapies. Avectas’ technology, Solupore®, delivers advanced molecules such as DNA, mRNA, proteins, and gene-editing tools to a range of primary cell types including T-cells for immunoncology and gene editing applications. www.avectas.com

 

 

 

AVROBIO
Geoff MacKay, President and CEO
Cambridge, MA
(NASDAQ: AVRO)
AVROBIO is a leading, Phase II gene therapy company focused on the development of its investigational gene therapy, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis, and Pompe disease. The company’s plato™ platform includes a proprietary vector system, automated cell manufacturing solution, and a personalized conditioning regimen deploying state-of-the-art precision dosing. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. www.avrobio.com

AXOVANT GENE THERAPIES
Parag Meswani, Pharm.D, SVP, Commercial Strategy and Operations
New York, NY
(NASDAQ: AXGT)
Axovant is accelerating the development of innovative gene therapy products that address serious neurological and neuromuscular diseases with significant unmet medical needs. The company is currently studying gene therapy candidates for GM1 gangliosidosis, Tay-Sachs and Sandhoff diseases, and Parkinson’s disease in clinical trials, and are working quickly to bring gene therapy candidates for other indications to patients in the clinic. www.axovant.com

BLUEBIRD BIO
Alison Finger, Chief Commercial Officer
Cambridge, MA
(NASDAQ: BLUE)
bluebird bio is pioneering gene therapy with purpose. The company is developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond the lab, bluebird bio is working to positively disrupt the healthcare system to create access, transparency, and education so that gene therapy can become available to all those who can benefit. bluebird bio is a human company powered by human stories. bluebird bio is putting care and expertise to work across a spectrum of disorders including cerebral adrenoleukodystrophy, sickle cell disease, β-thalassemia, and multiple myeloma using three gene therapy technologies: gene addition, cell therapy, and (megaTAL-enabled) gene editing. In addition to its headquarters, bluebird bio has additional nests in Seattle, WA; Durham, NC; and Zug, Switzerland. www.bluebirdbio.com

BONE THERAPEUTICS
Thomas Lienard, CEO
Gosselies, Belgium
(Euronext Brussels and Paris: BOTHE)
Bone Therapeutics is a leading biotech company focused on the development of innovative products to address high unmet needs in orthopedics and bone diseases. The company has a broad, diversified portfolio of bone cell therapy and an innovative biological product in later-stage clinical development across a number of disease areas. Bone Therapeutics’ core technology is based on its allogeneic cell therapy platform (ALLOB) which uses a unique, proprietary approach to bone regeneration, which turns undifferentiated stem cells from healthy donors into bone-forming cells. The ALLOB product pipeline includes a cell therapy product candidate that is expected to enter Phase II/III clinical development for the treatment of delayed-union fractures, and a Phase II asset in patients undergoing a spinal fusion procedure. The company is also developing an off-the-shelf protein solution, JTA-004, which is expected to enter Phase III development for the treatment of pain in knee osteoarthritis. www.bonetherapeutics.com

CABALETTA BIO

Cabaletta Bio is a clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for B cell-mediated autoimmune diseases. Our proprietary technology utilizes chimeric autoantibody receptor (CAAR) T cells designed to selectively bind and eliminate B cells that produce disease-causing autoantibodies while sparing normal B cells. Our lead CAAR T cell product candidate was designed based on chimeric antigen receptor (CAR) T cell technology that was successfully developed and is marketed for the treatment of B cell cancers. We have an exclusive license with Penn for CAAR T products and a partnership to manufacture our lead product candidate leveraging the process developed at Penn for early clinical trials of CART19, which became known commercially as Kymriah. We believe our technology, in combination with our proprietary Cabaletta Approach for selective B cell Ablation (CABA) platform has potential applicability across more than two dozen B cell-mediated autoimmune diseases. https://cabalettabio.com/

CARIBOU BIOSCIENCES
Rachel Haurwitz, Ph.D., President and CEO
Berkeley, CA
(Private)
Caribou Biosciences is a leading company in CRISPR genome editing founded by pioneers of CRISPR-Cas9 biology. The company is using next generation gene editing technologies to advance an internal pipeline of off-the-shelf CAR T-cell therapies to treat multiple types of cancers including non-Hodgkin lymphoma and multiple myeloma. Caribou uses its gene editing capabilities to boost the persistence of allogeneic cell therapy products. The company’s discovery pipeline includes NK cell therapies derived from gene-edited induced pluripotent stem cells and engineered gut microbes. www.cariboubio.com

CELAVIE BIOSCIENCES
Sandy Solmon, President and CEO
Oxnard, CA
(Private)
Celavie Biosciences is a privately held company whose mission is to improve lives and restore hope by advancing regenerative stem cell therapies for the treatment of Parkinson’s disease and other disorders of the central nervous system. The company develops undifferentiated, unmodified allogeneic pluripotent stem cell-based therapies, holds a strong IP portfolio, including 18 issued patents, and has an experienced management team blending expertise in concept and cell technology, product scalability, and entrepreneurship. Celavet, a subsidiary, applies the same proprietary technologies for the treatment and prevention of serious veterinary diseases. www.celavie.com

CELLATOZ THERAPEUTICS
Jaeseung Lim, Ph.D., CEO and Chief Scientific Officer
Gyeonggi, Republic of Korea
(Private)
Cellatoz Therapeutics is a biotech company dedicated to developing innovative cell therapies for the treatment of intractable diseases which there are no effective therapies for. Cellatoz has learned from failures of existing cell therapies using primary cells and/or mesenchymal stem cells showing marginal efficacy to treat various disease conditions. The company has sought meaningful ways to use certain cells as regenerative medicine therapy, and obtained unique proprietary cells which can open a new era of Cell Therapy 2.0 using differentiated allogenic cells defined with featured cell-specific markers and the ability to regenerate damaged cells or tissues. The company has been carrying out preclinical development programs for neurological, endocrine, and musucloskeletal disorders. www.cellatozrx.com/en

CELLECT BIOTECHNOLOGY
Cellect Biotechnology (NASDAQ: APOP), founded in 2011 after 10 years of academic research, is developing a first-in-class and potentially transformative cell selection technology platform, the ApoGraftTM; based on a revolutionary approach of exposure of stem cell-containing cell populations to an apoptosis-inducing environment, causing selective cell death in specific populations of differentiated cells, while not harming and even expanding stem cells.  The technology, protected by 8 patent families, has broad potential application, enabling the stem-cell based industry to produce safe, robust and affordable stem cells for any usage and indication.  ApoGraftTM first application in clinical development is the prevention of GvHD in bone marrow transplantation recipients. Early phase I/II study results show good engraftment, perfect safety score and no ApoGraftTM-related toxicity. Future potential applications include curing of autoimmune diseases and inducing immune tolerance in solid organ transplantation. Cellect’s pipeline also includes purification and expansion of Mesenchymal Stem Cells and CAR-T production. http://Cellect.co

CEVEC PHARMACEUTICALS
Nicole Faust, Ph.D., CEO and Chief Scientific Officer
Cologne, Germany
(Private)
CEVEC is a center of expertise for the production of biopharmaceuticals using a unique human cell-based expression system. The company offers a fully scalable manufacturing platform for viral vector production. CEVEC has successfully developed CAP-GT suspension cell-derived viral packaging cell lines, including a stable, helper virus-free AAV production platform, which enables better scale-up and competitive production costs when compared to adherent cell culture systems. CAP-GT suspension cell lines grow to high cell densities and show excellent productivity for a broad range of viruses. Gene therapy vectors such as lentivirus (LV), adenovirus (AV), and adeno-associated virus (AAV) can be produced at industrial scale. www.cevec.com

CYNATA THERAPEUTICS
Ross Macdonald, Ph.D., CEO
Melbourne, Australia
(ASX: CYP)
Cynata Therapeutics is a clinical-stage stem cell and regenerative medicine company that is developing a therapeutic stem cell platform technology, Cymerus™, originating from the University of Wisconsin-Madison, a world leader in stem cell research. The proprietary Cymerus technology addresses a critical shortcoming in existing methods of production of multi-donor derived mesenchymal stem cells (MSCs) for therapeutic use, which is the ability to achieve consistent economic manufacture at commercial scale. Cymerus utilizes induced pluripotent stem cells (iPSCs) to produce MSCs through a proprietary process that is independent of donor limitations providing an off-the-shelf stem cell platform for therapeutic product manufacture. A successful world-first clinical trial in GvHD was recently completed, meeting all safety and efficacy endpoints. The company expects to commence three Phase II trials during 2019/20. Cynata has a strategic partnership with FUJIFILM, which is one of the company’s larger shareholders. www.cynata.com

FLEXION THERAPEUTICS
Adam Muzikant, Ph.D., SVP, Business Development
Burlington, MA
(NASDAQ: FLXN)
Flexion Therapeutics is a biopharmaceutical company focused on the development and commercialization of novel, local therapies for the treatment of patients with musculoskeletal conditions, beginning with osteoarthritis (OA). The company’s first product, ZILRETTA® (triamcinolone acetonide extended-release injectable suspension), is the first and only FDA-approved extended-release, intra-articular (in the joint) injection for OA knee pain. Flexion’s pipeline includes FX201, an intra-articular gene therapy candidate being developed for symptomatic relief and disease modification in patients with knee OA. FX201 is designed to durably stimulate the production of an anti-inflammatory protein, IL-1Ra, whenever inflammation is present in the joint. An IND filing for FX201 has been cleared by FDA and enrollment in a Phase I study is expected to begin in 2019. The company is also developing FX301, a locally delivered NaV1.7 inhibitor formulated for extended release from a thermosensitive hydrogel for control of post-surgical pain. FX301 is expected to enter clinical trials in 2021. www.flexiontherapeutics.com

FRAUNHOFER IZI
Thomas Tradler, Head, Executive Department Business Development
Leipzig, Germany
(Private)
Fraunhofer IZI operates along the entire value chain of cell and gene therapy and drug and diagnostics R&D. Fraunhofer develops, tests, and manufactures cell-based therapies, advanced therapy medicinal products (ATMPs), biopharmaceuticals, small molecules, vaccines, and diagnostics for various human and veterinary medicine indications having a particular focus on immuno-oncology, inflammatory, and infectious as well as neurodegenerative diseases. Besides innovative drug and diagnostics product candidates and technologies, Fraunhofer offers a multitude of services including small and large animal models, as well as ATMP and biopharmaceuticals, GMP-manufacturing, and GLP drug testing (efficacy and toxicology). www.izi.fraunhofer.de/en

GENETHON
Genethon is a non-profit R&D institute and a leader in gene therapy for rare diseases. Genethon was created by the AFM-Telethon in 1990. Our mission is to design gene therapy products for rare diseases, and to ensure their pre-clinical and clinical development in order to provide patients with access to these innovative treatments. Genethon is developing therapies mainly for rare neuromuscular and liver diseases with an additional deep expertise in immune system & blood disorders Our translational research programs are supported by a first-rate technological platform and nearly 200 people who have joined forces in order to develop these innovative treatments. We have partnered with worldwide gene therapy leaders like Sarepta, Orchard Therapeutics, Avexis (Novartis), Audentes (Astellas) and Spark (Roche). https://www.genethon.fr/en/

GENSIGHT BIOLOGICS
Magali Taiel, M.D., Chief Medical Officer
Paris, France
(XPAR: SIGHT)
GenSight Biologics is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics’ lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.. www.gensight-biologics.com

HEALIOS
David Smith, Chief Commercial Officer
Tokyo, Japan
(JPX 4593.T)
Healios is a Tokyo-based, clinical-stage world leader in regenerative medicine and cell therapy. After its founding in 2011, Healios listed on the Tokyo Stock Exchange in 2015 and currently has more than 130 people based in its Japan and US offices. Healios leverages the attractive Japanese regulatory framework for regenerative medicine to rapidly and efficiently deliver results for patients, its partners, and stakeholders. It is currently running clinical trials for ischemic stroke (Phase 2/3) and acute respiratory distress syndrome (Phase 2) using bone marrow-derived allogeneic adult stem cells. At the same time, Healios is developing a next generation gene edited iPSC platform to remove the need for immunosuppressants, improve scalability, and enhance effectiveness. Its iPSC programs seek to address multiple areas of unmet medical need in the ophthalmology, liver disease, and immuno-oncology fields, and are supported by manufacturing platform development undertaken by Healios and its industry and academic partners. www.healios.co.jp/en

IOVANCE BIOTHERAPEUTICS
Maria Fardis, Ph.D., President and CEO
San Carlos, CA
(NASDAQ: IOVA)
Iovance Biotherapeutics intends to commercialize autologous cell therapy products for solid tumors and blood cancers. Tumor infiltrating lymphocyte (TIL) therapy uses a patient’s own cancer-fighting immune cells to attack solid tumors. TIL are extracted from tumor cells, and once expanded through a proprietary process are infused back into the patient. After infusion, TIL enter tumor tissue, where they recognize, attack, and destroy the tumor. The company is currently conducting pivotal studies in patients with metastatic melanoma and advanced cervical cancer. In addition, the company’s TIL therapies are being investigated for the treatment of patients with locally advanced, recurrent or metastatic cancers including head and neck and non-small cell lung cancer. Iovance has also developed a T cell therapy for blood cancers called peripheral blood lymphocyte (PBL) and intends to bring that product to clinic to investigate utility of PBL in chronic lymphocytic leukemia. www.iovance.com

KIADIS PHARMA
Robert Friesen, Ph.D., Chief Operating Officer
Amsterdam, Netherlands
(Euronext Amsterdam and Brussels: KDS)
Kiadis’ NK-cell program consists of off-the-shelf and haplo-donor cell therapy products for the treatment of liquid and solid tumors as adjunctive and stand-alone therapies. The company’s NK-cell PM21 particle technology enables improved ex-vivo expansion and activation of anti-cancer cytotoxic NK-cells supporting multiple high-dose infusions. Kiadis’ proprietary off-the-shelf NK-cell platform is based on NK-cells from a unique universal donor. The Kiadis off-the-shelf K-NK platform can make NK-cell therapy product rapidly and more economically available for a broad patient population across a potentially wide range of indications. Administered as an adjunctive immunotherapeutic on top of HSCT, K-NK002 provides lymphocyte infusions with functional, mature and potent cytotoxic NK cells from a haploidentical family member. In addition, Kiadis is developing K-NK003 for the treatment of relapse/refractory AML and has pre-clinical programs evaluating NK-cell therapy for the treatment of solid tumors. www.kiadis.com

KYTOPEN
Paulo Garcia, Ph.D., CEO and Co-Founder
Cambridge, MA
(Private)
Kytopen is a spin out of MIT developing proprietary platforms for non-viral delivery of payloads for ex vivo cell therapy discovery and manufacturing. The scalable solution combines microfluidics and automation to make transfection easier, faster, and more cost-effective than the state-of-the art solutions. Kytopen is integrating their platforms with automated liquid handling systems for processing small volumes and also developing a large volume platform for therapeutic applications. The non-viral solution will accelerate the time-to-market of next-generation cell and gene therapies by enabling discovery of therapeutic targets and reducing the cell therapy manufacturing costs. Kytopen recently closed a $3.6M seed funding round and is the recipient of a Phase II SBIR grant from the National Science Foundation (NSF). www.kytopen.com

LOGICBIO
Frederic Chereau, CEO
Cambridge, MA
(NASDAQ: LOGC)
LogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide™, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free and promoterless approach by relying on the native process of homologous recombination to drive potential lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families. www.logicbio.com

MEIRAGTX
Stuart Naylor, Ph.D., Chief Development Officer
London, UK
(NASDAQ: MGTX)
MeiraGTx is a clinical-stage gene therapy company focused on developing potentially curative treatments for patients living with serious diseases. We currently have six programs in clinical development including four ocular indications, a salivary gland condition, and a Parkinson’s disease program. Additionally, we are developing proprietary technology to potentially enable innovative gene therapy treatments whose expression can be turned on and off with an easily administered small molecule. Headquartered in New York and London, our global footprint provides us the opportunity to partner with leading institutions around the world, allowing us to deepen our understanding of diseases and their progression. Our state-of-the-art manufacturing facility, completed in 2018, was designed to meet global regulatory requirements, including cGMP required by the MHRA in the UK and the FDA. The 29,000-square foot facility has the flexibility and capacity to produce sufficient product for all our clinical trials and will scale to commercial capacity. www.meiragtx.com

MINERVA BIOTECHNOLOGIES
Cynthia Bamdad, Ph.D., CEO
Waltham, MA
(Private)
Minerva Biotechnologies discovered that the growth and pluripotency of naïve stem cells is mediated by the same growth factor receptor, MUC1*, and growth factor, NME7AB, that mediate growth and metastatic potential of cancer cells. We elucidated the molecular mechanisms that limit stem cell self-replication but allow cancer cells to self-replicate indefinitely. We learned how to override the pluripotency/differentiation switch to make naïve stem cell proliferate, without spontaneous differentiation, until we trigger the switch. Conversely, we have developed therapeutic agents that block MUC1* and others that block NME7AB, which block cancer growth. Minerva makes NME7AB-containing AlphaSTEM naïve stem cell media and a synthetic peptide that flips the switch to initiate differentiation. Minerva is currently conducting a Phase I/II 1st-in-human trial for a CAR T that targets MUC1* for the treatment of metastatic breast cancers. No therapeutic that targets MUC1* has ever been tested in humans. www.minervabio.com

MOLMED
Luca Alberici, Ph.D., Chief Business Officer
Milan, Italy
(MLM.MI)
MolMed is a biotech company focused on innovative cell and gene therapies that can meet the therapeutic needs in the treatment of tumors and rare diseases, by combining scientific and research excellence with a clear and strong industrial projects. MolMed is now structured on a dual business model, combining the development of onco-haematology proprietary pipeline with contract develop and manufacturing organization (CDMO), offering world class services and technologies to our clients. With two approved manufacturing facilities (about 4.800 SQM) and top level expertise, MolMed offers platforms of lentiviral and retroviral vector manufacturing with different scales (48L up to 200L) and a consolidated processes in cells engineering (Hematopoietic Stem cells and T-cells). Our different portfolio of top costumers includes GSK, Cellectis, Orchard Therapeutics, Telethon, Genenta, Rocket Pharma, Boston Children Hospital. www.molmed.com

NOVADIP BIOSCIENCES
Jeff Abbey, CEO
Mount-Saint-Guibert, Brussels
(Private)
Novadip Biosciences is a clinical-stage biopharmaceutical company using its unique tissue regeneration technology platform to generate multiple product classes to address hard and soft tissue reconstruction for patients who have limited or no treatment options. The company’s proprietary platform is a scaffold-free, 3-dimensional, extracellular matrix that utilizes stem cells to mimic the physiology of natural healing and creates a range of products that address specific challenges in tissue regeneration. Novadip’s initial focus is on orphan indications for critical size hard and soft tissue reconstruction and its lead program is in development for a rare pediatric orthopedic disease. The company is also applying its platform to develop truly novel off-the-shelf/allogeneic therapies to address more prevalent indications. www.novadip.com

ORCHARD THERAPEUTICS
Mark Rothera, CEO
London, UK
(NASDAQ: ORTX)
Orchard Therapeutics is a leading global fully integrated commercial-stage company dedicated to transforming the lives of patients with rare diseases through innovative gene therapies. Orchard’s portfolio of ex vivo autologous gene therapy programs has demonstrated sustained clinical benefit in over 150 patients across five disease areas. These programs include Strimvelis®, the first ex vivo autologous gene therapy approved by the EMA in 2016, three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. The company is partnered with world-leading institutions in gene therapy, including University College London, Great Ormond Street Hospital, the University of Manchester and Central Manchester University Hospitals, the University of California Los Angeles and Boston Children’s Hospital, and Telethon Institute of Gene Therapy/Ospedale San Raffaele. www.orchard-tx.com

OXFORD BIOMEDICA
John Dawson, CEO
Oxford, UK
(LSE:OXB)
Oxford Biomedica is developing life-changing gene therapies for patients with high unmet medical needs. Our unique LentiVector® platform has enabled the successful development of breakthrough gene and cell-based medicines, including the first approved CAR T therapy in the US and Europe. We have strong partnerships with leaders in the field, including Novartis, Orchard Therapeutics, Sanofi, Axovant, Santen, Boehringer Ingelheim, the UK Cystic Fibrosis Gene Therapy Consortium and Imperial Innovations. Oxford Biomedica has a long clinical and commercial track record in the gene therapy field spanning over 22 years. We were the first to administer lentiviral vector gene therapy directly to patients and the first FDA, EMA and PDMA-approved commercial supplier of lentiviral vectors in the world. Several hundreds of patients have safely received ex vivo and in vivo treatment with therapies that use our vectors. Our own portfolio of products includes candidates in the fields on oncology, ophthalmology, and CNS disorders. www.oxb.com

PDC*LINE PHARMA
Eric Halioua, President and CEO
Liege, Belgium
(Private)
PDC*line Pharma was founded in April 2014 as a spin-off of the French Blood Bank. PDC*line Pharma is a biotech company developing a novel class of off-the-shelf cancer immunotherapies based on a proprietary Plasmacytoid Dendritic Cell line (PDC*line) pre-loaded with peptides that are derived from target tumor antigens. Based on a robust preclinical package and a first-in-human phase Ib feasibility study in melanoma, PDC*line Pharma is focusing on Non-Small Cell Lung Cancer with a new candidate in phase 1b/2 clinical trial (PDC*lung) and neoantigens (PDC*Neo). In 2019, the company get authorization to launch phase I/II trial with PDC*lung in France and Belgium. PDC*line Pharma has raised nearly €17M in equity and non-dilutive funding. In 2019, we granted exclusive license in South Korea and exclusive option in other Asian countries to LG Chem, for the development and commercialization of PDC*lung cancer vaccine. Total deal value is 123M$ plus tiered royalties on net sales. www.pdc-line-pharma.com

PRECISION BIOSCIENCES
Michael Dombeck, SVP, Corporate Development
Durham, NC
(NASDAQ: DTIL)
Precision BioSciences is dedicated to improving life (DTIL) through the application of its pioneering, proprietary genome editing platform, ARCUS. Precision leverages ARCUS to develop product candidates in allogeneic (off-the-shelf) CAR T immunotherapy, in vivo gene correction, and food. Its lead clinical programs are currently in the allogeneic CAR T space with the most advanced product candidate being PBCAR0191, an allogeneic anti-CD19 CAR T therapy candidate currently being evaluated in collaboration with Servier in adult patients with relapsed or refractory NHL or B-ALL. Precision initiated dosing of subjects in a Phase 1/2a clinical trial of PBCAR0191 in April 2019, which continues to progress as planned. In September 2019, Precision announced FDA clearance of its IND for PBCAR20A, the company’s second and wholly-owned allogeneic CAR T candidate targeting CD20. Precision expects to file an IND for its wholly-owned BCMA targeted allogeneic CAR T candidate in 2020. precisionbiosciences.com

PROMETHERA BIOSCIENCES
Etienne Sokal, Ph.D., Group Chief Medical Officer
Mont-Saint-Guibert, Belgium
(Private)
Promethera Biosciences is pioneering the development of cutting-edge cell-based therapies for the treatment of liver diseases. We aim to bring patients life-saving treatments to reduce the need for liver transplantation. HepaStem™ is our lead therapeutic platform and has the potential to deliver novel treatment options for acquired chronic and acute liver diseases, currently in phase 2 clinical trials for both ACLF and NASH. A broad intellectual property portfolio, which includes a proprietary antibody therapy, human organ and cell sourcing and manufacturing capabilities, positions the Promethera Group as a global leader in regenerative medicine and cell therapy for liver diseases. www.promethera.com

PTC THERAPEUTICS
Marcio Souza, Chief Operating Officer
South Plainfield, NJ
(NASDAQ: PTCT)
PTC Therapeutics is a leader in the discovery, development and commercialization of medicines for the treatment of rare disorders. For 20+ years, PTC has been pursuing innovative technologies and therapies to develop and provide access to clinically-differentiated medicines for patients. PTC believes that by using proven scientific technologies, they can find innovative ways to treat rare disorders to create more shared moments and opportunities for patients and their families. Patients are at the center of everything PTC does – they work closely with patient advocacy groups, patients and caregivers to develop treatments and programs to address the needs of the rare disorder community. Currently, PTC is at the forefront of a new and transformative era of gene therapy, developing a targeted gene therapy platform to treat these devastating rare disorders and will be submitting a BLA for the first gene therapy to target AADC deficiency at the end of 2019. www.ptcbio.com

RECOMBINETICS
Daniel Carlson, Ph.D., Chief Scientific Officer
St. Paul, MN
(Private)
Recombinetics (RCI) aims to provide gene-editing solutions for some of the world’s most intractable challenges, from improving human health to sustainably feeding an ever-growing population. Founded in 2008, RCI is widely recognized as the global thought leader in livestock gene-editing and applied technology. RCI has developed proprietary toolkits and intellectual property for deployment in agriculture, regenerative medicine, and therapeutic development, commercialized through its subsidiaries Acceligen, Regenevida, and Surrogen. Regenevida and Surrogen are wholly owned subsidiaries of RCI and directly align with the mission of the Alliance for Regenerative Medicine. Regenevida is a therapeutic company that leverages RCI’s swine gene-editing production systems for cultivating human cells, tissues, and organs within living pigs for human therapeutic applications. Surrogen, leverages RCI’s expertise, to develop custom-tailored pig models that simulate the onset and progression of human disease. These models enable more effective therapeutic development, including cell and gene therapies, that will accelerate translation. www.recombinetics.com

REGENXBIO
Curran Simpson, SVP, Product Development and Chief Technology Officer
Rockville, MD
(NASDAQ: RGNX)
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO’s NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas. www.regenxbio.com

RENEURON
Michael Hunt, Chief Financial Officer
Pencoed, UK
(LON: RENE)
ReNeuron is a leading, clinical-stage cell therapy development company. Based in the UK, ReNeuron has therapeutic candidates in clinical development for motor disability as a result of stroke, for critical limb ischaemia and for the blindness-causing disease, retinitis pigmentosa. ReNeuron is also advancing its proprietary exosome technology platform as a potential new nanomedicine targeting cancer and as a potential delivery system for gene therapy treatments. ReNeuron’s shares are traded on the London AIM market under the symbol RENE.L. 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 serious diseases with unmet medical needs. The Company’s lead candidate, REX-001, is an innovative autologous cell therapy currently being studied in a Phase III clinical programme in patients with chronic limb-threatening ischemia, a poorly treated disease with a high risk of amputation and death. REX-001 was shown to be effective in Phase I/II and II trials in the majority of patients, reducing pain, alleviating ulcers, increasing mobility and reducing the need for amputation. Rexgenero intends to launch and market this specialty product in major territories. Rexgenero draws on an exceptional understanding of cell therapies developed by the Andalusian Health Authority and Andalusian Initiative of Advanced Therapies. The Company was founded in 2015 and is headquartered in London (UK) with R&D/manufacturing operations in Seville (Spain). www.rexgenero.com

SANGAMO THERAPEUTICS
Stephane Boissel, EVP, Corporate Strategy
Brisbane, CA
(NASDAQ: SGMO)
Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients’ lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. www.sangamo.com

SMARTPHARM THERAPEUTICS
Jose Trevejo, M.D., Ph.D., President and CEO
Cambridge, MA
(Private)
SmartPharm Therapeutics is a privately-held, development-stage pharmaceutical company focused on developing next-generation, non-viral gene therapies for the treatment of serious or rare diseases with the vision of creating “biologics from within.” The Company is currently developing a novel pipeline of non-viral, gene-encoded proteins for the treatment of conditions that require enzyme replacement therapy and tissue restoration, with an initial focus on Gaucher Disease. SmartPharm’s vision is to transform the nature of biologics therapy to improve cost effectiveness, enhance patient quality of life and extend the global reach of such treatments by shifting the source of production of therapeutic biologics from large pharmaceutical manufacturing facilities to the body of the patient. By developing gene-based drugs that induce target cells to make the necessary amounts of the desired biologic therapy of interest, the scale of clinical production can be greatly reduced, and often the treatment can be given as a simple injection. www.smartpharmtx.com

STANDARDS COORDINATING BODY FOR REGENERATIVE MEDICINE
Peter Reczek, Ph.D., Executive Director
Gaithersburg, MD
(Private)
The Standards Coordinating Body (SCB) is a global, unbiased, non-profit organization that accelerates standards development for the manufacture and distribution of advanced therapeutics. SCB’s mission is to coordinate standards across the regenerative medicine community; to engage subject matter experts in the identification, prioritization, and advancement of these standards and to educate the regenerative medicine community about the standards process and their implementation. www.standardscoordinatingbody.org

THERADAPTIVE
Luis Alvarez, Ph.D., CEO
Boston, MA
(Private)
Theradaptive is revolutionizing the field of therapeutic delivery with a pipeline of first-in-class and best-in-class products that address an $80B global market. There is a critical need for targeted tissue regeneration across a multitude of clinical indications. Our computational platform converts any recombinant protein into a material-binding variant that behaves like a paint and can be used to coat implants, devices, and injectable carriers to enable long-term local delivery with very low off-target effects. Every new therapeutic we generate with our method is novel composition of matter. We are going after previously out-of-reach clinical indications in vascular, soft tissue, and orthopedic repair as well as targeted chemotherapeutics. We have demonstrated superiority over standard of care in all preclinical studies conducted to date. www.theradaptive.com

TMUNITY THERAPEUTICS
Usman Azam, M.D., President and CEO
Philadelphia, PA
(Private)
Tmunity Therapeutics is a private clinical-stage biotherapeutics company focused on saving and improving lives by delivering the full potential of next-generation T-cell immunotherapy to patients with devastating diseases. Integrating a foundational collaboration with the University of Pennsylvania (Penn) with the groundbreaking scientific, clinical, and manufacturing expertise, and the demonstrated track record of its founders, Tmunity represents a new center of gravity in translational T-cell medicine. With headquarters in Philadelphia, Tmunity utilizes laboratories and production facilities at Penn and its own dedicated cGMP manufacturing facility in East Norriton, PA, to pursue process improvement and production scale-up in support of clinical development of cell therapies. www.tmunity.com

THERMOGENESIS
Chris Xu, Ph.D., CEO
Rancho Cordova, CA
(NYSE: TMO)
ThermoGenesis Holdings, Inc., formerly known as Cesca Therapeutics Inc., develops, commercializes and markets a range of automated technologies for CAR T and other cell-based therapies. The company currently markets a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology, including its semi-automated, functionally closed CAR TXpress™ platform, which streamlines the manufacturing process for the emerging CAR T immunotherapy market. www.thermogenesis.com

ULTRAGENYX PHARMACEUTICAL
Sam Wadsworth, Ph.D., Chief Scientific Officer
Novato, CA
(NASDAQ: RARE)
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are no approved therapies. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. www.ultragenyx.com

VERIGRAFT
Petter Bjorquist, Ph.D., CEO
Gothenburg, Sweden
(Private)
VERIGRAFT is a Swedish biotechnology company with 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 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 segment 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). CVI is a multi-billion USD market and these patients do not receive any curative treatment today. www.verigraft.com

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