2018 Company Presenters
David Kirn, M.D., Chairman, CEO and Co-Founder
Headquarters: Emeryville, CA
Number of Full-Time Employees: 35
Company Website: www.4dmoleculartherapeutics.com
ABOUT 4D MOLECULAR THERAPEUTICS
4D Molecular Therapeutics (4DMT) is focused on the discovery and development of targeted and proprietary AAV gene therapy vectors and therapeutic products. The company’s robust discovery platform, termed Therapeutic Vector Evolution, empowers 4DMT to create customized gene delivery vehicles to deliver genes to any tissue or organ in the body, by optimal clinical routes of administration and with resistance to pre-existing antibodies. These proprietary and targeted products allow the company to treat both rare genetic diseases and complex large market diseases. 4DMT is creating a diverse and deep product pipeline through partnerships, while progressing internal 4DMT products toward clinical trials in parallel. 4D partners include: Pfizer (PFE), Roche (SIX: ROG; OTCQX: RHHBY), uniQure (QURE), AGTC and Benitec. 4DMT’s lead product is for the treatment of choroideremia, an inherited retinal disease.
James Noble, CEO
Headquarters: Abingdon, UK
Number of Full-Time Employees: 350
Company Website: www.adaptimmune.com
January 8, 2018 | Adaptimmune Announces Positive Safety Data from Pilot Studies with MAGE-A10 SPEAR T-cells and First Patient to Receive 1 billion Target Cell Dose
January 8, 2018 | Adaptimmune Announces Two Manufacturing Achievements on Its way to Become the First Fully Integrated TCR T-cell Therapy Company
January 4, 2018 | Paul Stead Joins Adaptimmune as Vice President of Business Development
Adaptimmune, a leader in T-cell therapy, is a multinational, clinical-stage biopharmaceutical company focused on developing novel immunotherapies using its proprietary SPEAR T-cell receptor platform to treat solid tumors, which has the potential to transform the treatment of cancer. Our proprietary SPEAR T-cell platform has generated a strong pipeline of affinity-enhanced T-cell therapies, with multiple INDs open. We use these therapies to harness the body’s own immune system to find and destroy diseased cells. Our SPEAR TCR therapies offer promise to patients that often have no other options. We are working hard to make that promise a reality. We are based in the UK, in Oxford, and in the US, in Philadelphia.
Amber Salzman, Ph.D., President and CEO
Headquarters: Menlo Park, CA
Number of Full-Time Employees: 75
Company Website: www.adverum.com
January 25, 2018 | Adverum Biotechnologies and Editas Medicine Extend Research Collaboration
ABOUT ADVERUM BIOTECHNOLOGIES
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.
Sue Washer, President and CEO
Headquarters: Alachua, FL
Number of Full-Time Employees: 86
Company Website: www.agtc.com
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 retinoschisis (XLRS), X-linked retinitis pigmentosa (XLRP), and achromatopsia (ACHM CNGB3 & ACHM CNGA3). In addition to its clinical trials, AGTC has preclinical programs in optogenetics, adrenoleukodystrophy (ALD), which is a disease of the central nervous system (CNS), and otology. The clinical-stage XLRS and XLRP programs, the discovery program in ALD and two additional ophthalmology programs are being developed in collaboration with Biogen. 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.
Geoff MacKay, President and CEO
Headquarters: Cambridge, MA
Number of Full-Time Employees: 25
Company Website: www.avrobio.com
February 1, 2018 | AVROBIO, Inc. Announces $60 Million Series B Financing to Advance Gene Therapy Pipeline for Lysosomal Storage Disorders and Apply Lentiviral Platform to Other Genetic Diseases
AVROBIO, a leader in lentiviral-based gene therapies, is a clinical stage company developing disruptive therapies that have the potential to transform patients’ lives in a single dose. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON.
Peter Altman, Ph.D., CEO
Headquarters: San Carlos, CA
Number of Full-Time Employees: 20
Company Website: www.biocardia.com
January 29, 2018 | CardiAMP Cell Therapy Receives FDA Approval for Pivotal Trial in Chronic Myocardial Ischemia
January 5, 2018 | BioCardia Submits Clinical Trial to FDA for New Indication of Chronic Myocardial Ischemia for CardiAMP Cell Therapy
BioCardia is a clinical-stage regenerative medicine company developing novel therapeutics for cardiovascular diseases with large unmet medical needs. BioCardia is developing proprietary comprehensive biotherapeutic solutions for cardiovascular disease in its CardiAMP™ and CardiALLO™ therapies.
Jeff Walsh, Chief Financial and Strategy Officer
Headquarters: Cambridge, MA
Number of Full-Time Employees: 524
Company Website: www.bluebirdbio.com
ABOUT BLUEBIRD BIO
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.
Jeff Liter, President and CEO
Headquarters: Minneapolis, MN
Number of Full-Time Employees: 8
Company Website: www.bmogen.com
ABOUT B-MOGEN BIOTECHNOLOGIES
B-MoGen Biotechnologies is a genome engineering company that was 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. We utilize this unique expertise to provide three distinct services that share complexity in gene delivery and gene editing. We offer a non-viral transposon-based gene delivery platform to support the T-Cell immunotherapy industry, we are the first company in the world to demonstrate genome engineering of the mitochondria genome, and we 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.
Chaim Lebovits, President and CEO
Headquarters: Hackensack, NJ
Number of Full-Time Employees: 19
Company Website: www.brainstorm-cell.com
January 3, 2018 | BrainStorm Granted GMP Certification for a Marketing-Level Manufacturing of NurOwn®
ABOUT BRAINSTORM CELL THERAPEUTICS
BrainStorm Cell Therapeutics is a biotechnology company developing innovative, autologous stem cell therapies for highly debilitating neurodegenerative diseases such as Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig’s disease), Multiple Sclerosis (MS) and Parkinson’s Disease (PD).
Linda Marban, Ph.D., CEO
Headquarters: Beverly Hills, CA
Number of Full-Time Employees: 35
Company Website: www.capricor.com
ABOUT CAPRICOR THERAPEUTICS
Capricor Therapeutics is a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class biological therapeutics for the treatment of rare disorders. Capricor’s lead candidate, CAP-1002, is an allogeneic cell therapy that is currently in clinical development for the treatment of Duchenne muscular dystrophy. Capricor has also established itself as one of the leading companies investigating the field of extracellular vesicles and is exploring the potential of CAP-2003, a cell-free, exosome-based candidate, to treat a variety of disorders.
Ajan Reginald, CEO
Headquarters: Stratford Upon Avon, UK
Number of Full-Time Employees: 44
Company Website: www.celixir.com
January 8, 2018 | CTA Approval for Potentially Pivotal HeartcelTM Phase IIb Trial
Celixir is a private British regenerative medicine company that discovers and develops life-saving and life-altering regenerative medicines for patients with the greatest medical need. Celixir, founded in 2009, is made up of a world-class team of scientists and biopharmaceutical executives, led by Nobel Laureate Professor Sir Martin Evans and former Roche Global Head of Emerging Technologies, Ajan Reginald. Celixir’s unique platform technology allows them to adopt an ‘off-the-shelf’ approach to deliver regenerative medicines to patients. We focus on delivering disruptive technologies in areas of high unmet need, We discover cellular therapies both in the field of regenerative medicine, and in related areas such as oncology. Celixir has developed world-leading expertise in the discovery and development of cellular therapies, and has built a management team and a series of collaborations to ensure that its medicines are successfully developed in the most efficient and effective manner.
Ross Macdonald, Ph.D., CEO
Headquarters: Melbourne, Australia
Number of Full-Time Employees: 2
Company Website: www.cynata.com
January 30, 2018 | December 2017 Quarterly Report
January 24, 2018 | Treatment Commences in Second Patient Cohort in Cynata’s World First Clinical Trial
January 23, 2018 | Cynata Executes MoU for Commercial Evaluation with Celularity, Inc.
ABOUT CYNATA THERAPEUTICS
Cynata Therapeutics Limited is an Australian clinical-stage stem cell and regenerative medicine company that is developing a therapeutic allogenic stem cell platform technology, Cymerus™, originating from the University of Wisconsin-Madison. 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 utilises iPSCs to produce MSCs through a proprietary process that is independent of donor limitations. It and provides an “off-the-shelf” stem cell platform for therapeutic product use, with a pharmaceutical product business model and economies of scale. This has the potential to create a new standard in the emergent arena of stem cell therapeutics. A Phase 1 clinical trial of the Company’s first MSC product is underway in graft-versus-host disease. Cynata has a strategic partnership with Fujifilm, the Company’s largest shareholder.
John Maslowski, President and CEO
Headquarters: Exton, PA
Number of Full-Time Employees: 25
Company Website: www.fibrocell.com
January 30, 2018 | Fibrocell Announces FDA Allowance to Initiate Pediatric Enrollment in Phase 1/2 Clinical Trial of FCX-007 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin and connective tissue. Fibrocell’s most advanced product candidate, FCX-007, is the subject of a Phase 1/2 clinical trial for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is in pre-clinical development of FCX-013, its product candidate for the treatment of moderate to severe localized scleroderma. Fibrocell’s gene therapy portfolio is being developed in collaboration with Intrexon Corporation (NYSE: XON), a leader in synthetic biology.
David Lucchino, Co-Founder, President and CEO
Headquarters: Woburn, MA
Number of Full-Time Employees: 23
Company Website: www.frequencytx.com
ABOUT FREQUENCY THERAPEUTICS
Frequency Therapeutics is a clinical-stage company harnessing the body’s innate regenerative biology with its Progenitor Cell Activation (PCA) regenerative platform. Frequency has successfully completed a first-in-human study of FX-322, the company’s lead PCA candidate for hearing restoration. The phase 1 study provided crucial data on safety and pharmacokinetics associated with intratympanic delivery of FX-322 as the company moves forward toward additional clinical trials in the U.S. to monitor clinical response. Frequency expects to initiate Phase 2 clinical work for its hearing regeneration program in 2H 2018. Frequency announced a three-way partnership with the US Army and the Harvard Stem Cell Institute to investigate small molecule regeneration for several disease indications involving muscle. The company also announced a Juvenile Diabetes Research Foundation award for the development of small molecule approach to create insulin and GLP-1 secreting cells. The company has secured $45M in financing.
Jon Lieber, Chief Financial Officer
Headquarters: Waltham, MA
Number of Full-Time Employees: 50
Company Website: www.histogenics.com
January 25, 2018 | Histogenics Corporation Announces Closing of Underwritten Registered Direct Offering of Common Stock and Full Exercise of Option to Purchase Additional Shares
January 22, 2018 | Histogenics Corporation Announces Pricing of Underwritten Registered Direct Offering of 2,340,430 Shares of Common Stock
January 9, 2018 | Histogenics Corporation Announces Receipt of $10 Million Up-front Payment for Japanese Neocart Agreement
Histogenics is a leader in the development of restorative cell therapies that may offer rapid-onset pain relief and restored function. Histogenics’ lead investigational product, NeoCart, is designed to rebuild a patient’s own knee cartilage to treat pain at the source and potentially prevent a patient’s progression to osteoarthritis. NeoCart is one of the most rigorously studied restorative cell therapies for orthopedic use. Histogenics recently completed enrollment of its NeoCart Phase III clinical trial and expects to report top-line, one-year superiority data in the third quarter of 2018. NeoCart is designed to perform like articular hyaline cartilage at the time of treatment, and as a result, may provide patients with more rapid pain relief and accelerated recovery as compared to the current standard of care. Histogenics’ technology platform has the potential to be used for a broad range of additional restorative cell therapy indications.
Maria Fardis, President and CEO
Headquarters: San Carlos, CA
Number of Full-Time Employees: 65
Company Website: www.iovance.com
January 29, 2018 | Iovance Biotherapeutics, Inc. Announces Closing of $172.5 Million Common Stock Public Offering
January 24, 2018 | Iovance Biotherapeutics Announces Preliminary Phase 2 Data for TIL Treatment in Head and Neck and Cervical Cancers
ABOUT IOVANCE BIOTHERAPEUTICS
Iovance Biotherapeutics is a clinical-stage biotechnology company focused on the development of cancer immunotherapy products for the treatment of various cancers. The Company’s lead product candidate is an adoptive cell therapy using tumor-infiltrating lymphocyte (TIL) technology being investigated for the treatment of patients with metastatic melanoma, recurrent and/or metastatic squamous cell carcinoma of the head and neck and recurrent and metastatic or persistent cervical cancer.
Arthur Lahr, CEO
Headquarters: Amsterdam, The Netherlands
Number of Full-Time Employees: 60
Company Website: www.kiadis.com
January 16, 2018 | Kiadis Pharma Proposes Former Cipla CEO Subhanu Saxena as New Supervisory Board Member
ABOUT KIADIS PHARMA
Kiadis’ allodepleted T-cell immunotherapy product can make haploidentical hematopoietic stem cell transplantations safer and more effective. Phase 2 data with lead product ATIR101™ given after a HSCT in patients with blood cancer shows a strong and clinically relevant improvement over literature for the Baltimore protocol, without severe chronic Graft versus Host Disease. The Company submitted an MAA to the EMA in April 2017, for approval of ATIR101™ as an adjunctive treatment in HSCT for malignant disease. Kiadis received Day 120 questions in September 2017 and expects a potential (conditional) approval in 4Q18 and launch in 2H19. Kiadis is conducting a Phase 3 trial across Europe and North America (head to head against the Baltimore protocol). The first patient was enrolled in December 2017. In September 2017 the FDA granted ATIR101™ the Regenerative Medicine Advanced Therapy (RMAT) designation. ATIR101™ has been granted Orphan Drug Designations both in the US and Europe.
Fred Chereau, CEO
Headquarters: Cambridge, MA
Number of Full-Time Employees: 16
Company Website: www.logicbio.com
January 2, 2018 | LogicBio Therapeutics Announces Expansion of Leadership Team through Multiple Key Hires
ABOUT LOGICBIO THERAPEUTICS
Launched in 2016, LogicBio is a leading genome-editing company founded by pioneers in gene therapy from Stanford University, with a mission to develop transformative medicines for life-threatening diseases. LogicBio’s technology includes GeneRide™, a promoterless, nuclease-free genome editing technology, which drives high expression of therapeutic proteins by harnessing homologous recombination—a naturally occurring process—to safely integrate transgenes site-specifically into the genome to provide a durable cure from a one-time treatment; and a proprietary library of adeno-associated viral vectors which have proven highly effective and safe in clinical trials. This combination of next generation genome editing and proven delivery technology allow LogicBio to target a wide range of diseases not addressable today, including early-onset conditions in young patients. With a world-class global team, headquartered in Cambridge, Massachusetts, LogicBio is working to deliver the next wave of innovation in genetic medicine.
Mark Landy, SVP Strategic Initiatives
Headquarters: Marietta, GA
Number of Full-Time Employees: 850
Company Website: www.mimedx.com
January 25, 2018 | MiMedx Adds New Materials To Its Website Exposing Misinformation Disseminated Through Short Sellers
January 18, 2018 | MiMedx Comments on Magistrate Recommendation in the Company’s Lawsuit Against Short Sellers
January 17, 2018 | MiMedx Announces the Enrollment of the First Patients in the Phase 3 Clinical Trial of AmnioFix® Injectable for Achilles Tendonitis
MiMedx® is the leading biopharmaceutical company developing and marketing regenerative and therapeutic biologics utilizing human placental tissue allografts with patent-protected processes for multiple sectors of healthcare. “Innovations in Regenerative Medicine” is the framework behind our mission to give physicians products and tissues to help the body heal itself. We process the human placental tissue utilizing our proprietary PURION® Process among other processes, to produce safe and effective allografts. MiMedx proprietary processing methodology employs aseptic processing techniques in addition to terminal sterilization. MiMedx is the leading supplier of placental tissue, having supplied over 1 million allografts to date for application in the Wound Care, Burn, Surgical, Orthopedic, Spine, Sports Medicine, Ophthalmic and Dental sectors of healthcare.
Jeff Ross, Ph.D., CEO
Headquarters: Eden Prairie, MN
Number of Full-Time Employees: 28
Company Website: www.miromatrix.com
Miromatrix is a leading regenerative medicine company bioengineering transplantable organs including livers, kidneys and hearts based on its patented perfusion decellularization and recellularization technology, with collaborators including The Mayo Clinic, Mount Sinai Hospital, and Texas Heart Institute. In addition to whole organ development, Miromatrix has successfully developed, manufactured and commercialized a series of porcine organ-derived biological products. MIROMESH®, is the only liver-derived biologic mesh for soft tissue reinforcement. Miromatrix released 1-year prospective multicenter-clinical trial data for the treatment of hiatal hernias demonstrating no reintervention. MIRODERM®, is the only liver-derived wound care product and a recent case-series published in WOUNDS® demonstrated its ability to successfully treat diabetic foot ulcers that had previous failed other advanced biologics. A multicenter-clinical study is currently enrolling to further demonstrate its ability to treat this challenging patient population. Miromatrix is leveraging these successes with our recellularization technology to fully address the transplantable organ shortage.
Mark Rothera, President and CEO
Headquarters: London, UK
Number of Full-Time Employees: 71
Company Website: www.orchard-tx.com
January 17, 2018 | Orchard Therapeutics Appoints Frank Thomas as Chief Financial Officer and Chief Business Officer
ABOUT ORCHARD THERAPEUTICS
Orchard Therapeutics is a biotechnology company incorporated in September 2015 and dedicated to transforming the lives of patients with rare disorders through innovative gene therapies. Our programmes will use the potential of ex-vivo autologous haematopoietic stem cell gene therapy to restore normal gene function in severe and life-threatening inherited disorders. We work in partnership with the world’s leading research centres to harness the life-giving potential of gene therapy. Our mission is to transform the lives of patients with rare disorders through innovative gene therapies. Our vision is to become the leader in gene therapy for primary immune deficiencies and inherited metabolic disorders.
John Dawson, CEO
Headquarters: Oxford, UK
Number of Full-Time Employees: 320
Company Website: www.oxfordbiomedica.co.uk
January 17, 2018 | Oxford BioMedica Announces £3 Million Grant Awarded by Innovate UK
January 23, 2018 | Oxford BioMedica Notes the US FDA Priority Review for Kymriah™ for Adults with r/r DLBCL and EMA Accelerated Assessment for Children, Young Adults with r/r B-cell ALL and Adult Patients with r/r DLBCL
ABOUT OXFORD BIOMEDICA
Oxford BioMedica is a leading gene and cell therapy group focused on developing life changing treatments for serious diseases. Oxford BioMedica has built a sector leading lentiviral vector delivery platform (LentiVector®), which we leverage to develop in vivo and ex vivo products ourselves and with Partners. The Company has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oCNS disorders (Parkinson’s), oncology (CAR-T), and ophthalmology. The Group has also entered into a number of partnerships, including with Novartis, Sanofi, GSK, Orchard Therapeutics and Immune Design, through which it has long-term economic interests in other potential gene and cell therapy products. Oxford BioMedica is a fast growing company in Oxfordshire, UK and employs more than 320 people.
Matt Kane, CEO
Headquarters: Durham, NC
Number of Full-Time Employees: 94
Company Website: www.precisionbiosciences.com
ABOUT PRECISION BIOSCIENCES
Precision BioSciences is dedicated to improving life. We develop products designed to cure genetic disease, overcome cancer, and feed the planet. To make this vision a reality, we have brought together the world’s leading minds in genome editing and ARCUS, the first therapeutic grade editing platform. This simple and uniquely powerful genome editing method allows us to create products that solve significant problems in oncology, genetic disease, agriculture, and beyond.
Suzanne Ildstad, M.D., CEO
Headquarters: Louisville, KY
Number of Full-Time Employees: 10
Company Website: www.regenerex.com
Regenerex LLC, an innovative, late clinical stage cellular therapy company, is developing its FCRx platform technology with the goal of eliminating the burden of chronic immunosuppression in select organ transplant and severe autoimmune conditions. In an ongoing Phase 2 study, the Company’s proprietary allogeneic cell therapy product, FCR001, has been shown to induce tolerance in recipients of solid organ transplants from unmatched and unrelated donors, allowing living donor kidney transplant recipients to wean completely off immunosuppression one year after transplant. FCR001 Phase 2 subjects achieved these results utilizing nonmyeloablative conditioning which minimizes toxicities typically associated with a standard Hematopoietic Stem Cells (HSC) transplant. FDA has granted Regenerex clearance to proceed with a pivotal Phase 3 study in Living Donor Kidney Transplant. FDA has also granted FCR001 Orphan Drug designation and Regenerative Medicine Advanced Therapy (RMAT) designation.
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.
Philip Toleikis, Ph.D., President and CEO
(TSX-V: SVA; OTCQB: SEOVF)
Headquarters: London, Ontario, Canada
Number of Full-Time Employees: 8
Company Website: www.sernova.com
ABOUT SERNOVA CORP.
Sernova Corp. is a clinical-stage regenerative medicine company commercializing products which benefit patients with chronic metabolic, neurological, and hematological diseases with first therapeutic applications in diabetes and hemophilia. Sernova’s platform technologies include the Cell Pouch System™, an implantable medical device for the long-term survival and function of therapeutic cells, therapeutic cells (porcine & human donor islets, stem cell processes) and micro-encapsulation-based technologies which provide local immune protection to therapeutic cells enabling potential elimination of antirejection drugs.
David Venables, Ph.D., CEO
Headquarters: Edinburgh, UK
Number of Full-Time Employees: 32
Company Website: www.synpromics.com
January 25, 2018 | Synpromics Announces New Partnership with UCL to Develop Novel Gene Therapies for Blood-based Disorders
January 17, 2018 | Synpromics Appoints Sarah Haecker Meeks, PhD as Vice President of Business Development
Synpromics is the leader in gene control, improving human health by enabling safer, more effective cell and gene medicines through proprietary genomics, bioinformatics and intelligent data driven design. Synpromics enables unprecedented control of gene expression through custom designed synthetic cell-type, or condition, specific promoters. Custom control of gene expression is fundamentally important in the creation of gene and cell therapy products. The technology created by the Synpromics team allows tighter control of gene expression, facilitating the creation of novel promoters tailored to: Tissue-type, e.g., liver, muscle, CNS; Developmental stage, e.g., lineage specificity in stem cells; Cell affliction, e.g., pathogen-infected; Inducible by biological, chemical or environmental stimuli; Eliminate off-target gene expression. By enhancing the specific activity of the promoter it is possible to reduce vector dose, increasing the safety profile of the therapeutic, lowering immunogenicity, reducing off-target side effects.
Usman Azam, M.D., President and CEO
Headquarters: Philadelphia, PA
Number of Full-Time Employees: 2
Company Website: www.tmunity.com
ABOUT TMUNITY THERAPEUTICS
At the heart of Tmunity is a relentless focus on creating proprietary products by integrating T cell discovery, development and production capabilities. Tmunity has established a broad, exclusive research and development collaboration and license agreement with the University of Pennsylvania (UPenn) in the field of T cell therapies in oncology, infectious diseases and autoimmune diseases. Tmunity’s portfolio consists of key T cell therapy platforms designed to produce engineered T cells that activate or suppress immunity. These approaches include: T cell receptors, regulatory T cells, gene transfer, allogeneic T cells and bionic T cells, as well as certain CAR-T programs.
Stephane Boissel, CEO
(EURONEXT PARIS: TXCL)
Headquarters: Valbonne, France
Number of Full-Time Employees: 46
Company Website: www.txcell.com
January 24, 2018 | TxCell Announces 4th Quarter 2017 Financials and Participation at Upcoming Scientific Conferences
January 17, 2018 | TxCell Obtains Government Funding to Advance its Lead CAR-Treg Program
January 8, 2018 | TxCell Announces its Financial Calendar for 2018
TxCell is a biotechnology company that develops platforms for innovative, personalized T cell immunotherapies for the treatment of severe inflammatory and autoimmune diseases with high unmet medical need. TxCell is targeting transplant rejection as well as a range of autoimmune diseases (both T-cell and B-cell-mediated), including multiple sclerosis, lupus nephritis and bullous pemphigoid. TxCell’s cellular immunotherapies are based on regulatory T lymphocytes (Tregs). Tregs are a T cell population discovered in the nineties for which anti-inflammatory properties have been demonstrated. Contrary to conventional approaches based on non-specific polyclonal Tregs, TxCell is exclusively developing engineered antigen-specific Tregs, where the antigen specificity is brought by a Chimeric Antigen Receptor (CAR) (CAR-Treg cells).
Nick Colangelo, President and CEO
Headquarters: Cambridge, MA
Number of Full-Time Employees: 250
Company Website: www.vcel.com
January 2, 2018 | Vericel Initiates Collaboration with Innovative Cellular Therapeutics
Develops, manufactures, and markets autologous cell-based therapies for patients with serious diseases and conditions. The company markets two cell therapy products in the United States. MACI® (autologous cultured chondrocytes on porcine collagen membrane) is a third generation autologous cellularized scaffold product that is indicated for the repair of single or multiple symptomatic, full-thickness cartilage defects of the adult knee, with or without bone involvement. Epicel® (cultured epidermal autografts) is a permanent skin replacement for the treatment of patients with deep dermal or full thickness burns greater than or equal to 30% of total body surface area.