2016 Company Presenters
Timothy Miller, Ph.D., President, CEO and Director
Headquarters: Texas, USA
Number of Full-Time Employees: 18
Company Website: www.abeonatherapeutics.com
ABOUT ABEONA THERAPEUTICS
Abeona Therapeutics is a clinical-stage company developing gene therapy and plasma-based therapies for severe and life-threatening rare genetic diseases. Abeona’s lead programs are ABO-102 (AAV-SGSH) and ABO-101 (AAV-NAGLU), adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome (MPS IIIA and IIIB), respectively. The company is also developing EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa, ABO-201 (AAV-CLN3) gene therapy for juvenile Batten disease; and ABO-301 (AAV-FANCC) for Fanconi anemia disorder using a novel CRISPR/Cas9-based gene editing approach to gene therapy for rare blood diseases. In addition, Abeona is developing plasma protein therapies, including SDF Alpha™ (alpha-1 protease inhibitor) for inherited COPD, using the company’s proprietary SDF™ (Salt Diafiltration) ethanol-free process.
James Noble, Co-Founder and CEO
Headquarters: Abingdon, UK
Number of Full-Time Employees: 279
Company Website: www.adaptimmune.com
Adaptimmune is a clinical-stage biotechnology company with multiple trials ongoing in both solid tumours and hematologic cancer types, and in cancers where survival rates for patients can be very limited. The company’s T-cell therapies have already shown preliminary evidence of tumor reduction in patients and also show a promising risk/benefit profile. Developed over the last 15 years, Adaptimmune’s proprietary T-cell engineering platform has generated a strong pipeline of affinity enhanced T-cell therapies. The company uses these therapies to harness the body’s own immune system to find and destroy diseased cells. Adaptimmune can develop T-cell therapies for a broad spectrum of cancer types and patients and have multiple T-cell therapies in development or in clinical trials. The company’s TCR therapies offer promise to patients that often have no other options. Adaptimmune is working hard to make that promise a reality.
Mike Rice, President and CEO
Headquarters: Washington, USA
Number of Full-Time Employees: 45
Company Website: www.biolifesolutions.com
ABOUT BIOLIFE SOLUTIONS
BioLife Solutions develops, manufactures and markets biopreservation media products and smart shipping containers connected to a cloud hosted cold chain management app to improve viability and delivery logistics for cells, tissues and organs. BioLife products are embedded in more than 220 customer validations and clinical trials of cell-based therapies.
Linda Marban, Ph.D., Co-Founder and CEO
Headquarters: California, USA
Number of Full-Time Employees: 36
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 therapeutics. Capricor has two product candidates under clinical investigation: CAP-1002, a cardiac cell therapy, and Cenderitide, a dual natriuretic peptide receptor agonist. CAP-1002 is in development for the treatment of post myocardial infarction, advanced heart failure and Duchenne muscular dystrophy-associated cardiomyopathy. Cenderitide is in development for the outpatient treatment of heart failure as well as other potential indications. In addition, Capricor is evaluating its exosomes platform technology for cardiac diseases and other therapeutic areas.
Jean-Philippe Veillard, Chief Financial Officer
Headquarters: Mulhouse, France
Number of Full-Time Employees: 19
Company Website: www.cellprothera.com
Established in 2008, CellProthera is a pioneer in regenerative medicine developing simultaneously therapeutic solutions and the required technologies. As of today, CellProthera has validated an innovative, proprietary technology platform to industrialize and disseminate its current and future stem cell therapies. CellProthera is initially focusing on the cardiac field following a very successful proof of principle study showing outstanding clinical benefits and excellent safety. CellProthera is bringing to market an ATMP/MTMM, ProtheraCytes®, allowing the regeneration of myocardial tissue to treat acute myocardial infarction (AMI) and prevent secondary congestive heart failure. With a significant patient population and few, if any alternative treatments, ProtheraCytes could become a blockbuster. It is now in a Phase I/IIb trial in France and the UK, and another trial will be launched in Singapore later this year. CellProthera has already received €20 million of both public and private funds and the company is now seeking €25 million of capital to finance its next phases.
Manfred Rüdiger, Ph.D., CEO
Headquarters: Amsterdam, The Netherlands
Number of Full-Time Employees: 32
Company Website: www.kiadis.com
ABOUT KIADIS PHARMA
Kiadis Pharma is a clinical stage (Phase II/III) company developing therapies for patients with late-stage blood cancers and non-malignant blood disorders. ATIR101™ is a cell-based medicinal product manufactured from mismatched family members to allow for T cell infusions after stem cell transplantation to strengthen the patient’s immune system without eliciting life threatening acute or severe chronic graft versus host disease.
Douglas Doerfler, President and CEO
Headquarters: Maryland, USA
Number of Full-Time Employees: 30
Company Website: www.maxcyte.com
MaxCyte is an established and revenue generating US-based developer and supplier of cell engineering technology to biotechnology and pharmaceutical firms engaged in cell therapy, drug discovery and development, biomanufacturing, gene editing and immuno-oncology. Its patented flow electroporation technology enables its products to deliver fast, reliable and scalable cell engineering to drive the research and clinical development of a new generation of cell-based medicines. MaxCyte’s high performance platform allows transfection with any molecule or multiple molecules and is compatible with nearly all cell types. It also provides a high degree of consistency and minimal cell disturbance, thereby facilitating rapid, large scale, cell engineering in a non-viral system and with low toxicity concerns. MaxCyte is developing CARMA, its proprietary platform in immuno-oncology, to deliver a validated non-viral approach to CAR therapies in a number of cancer indications, including solid tumors where existing CAR-T approaches face significant challenges.
John Dawson, CEO
Headquarters: Oxford, UK
Number of Full-Time Employees: 230
Company Website: www.oxfordbiomedica.co.uk
ABOUT OXFORD BIOMEDICA
Oxford BioMedica is a leading gene and cell therapy company focused on developing life changing treatments for serious diseases. Oxford BioMedica and its subsidiaries (the “Group”) have built a sector leading lentiviral vector delivery platform (LentiVector®) through which the Group develops in vivo and ex vivo products both in-house and with partners. The Group has created a valuable proprietary portfolio of gene and cell therapy product candidates in the areas of oncology, ophthalmology and CNS disorders. The Group has also entered into a number of partnerships, including with Novartis, Sanofi, GSK and Immune Design, through which it has long-term economic interests in other potential gene and cell therapy products. Oxford BioMedica is based across several locations in Oxfordshire, UK and employs more than 230 people.
Olav Hellebo, CEO
Headquarters: Guildford, UK
Number of Full-Time Employees: 40
Company Website: www.reneuron.com
ReNeuron is a leading, clinical-stage cell therapy development business. Based in the UK, its primary objective is the development of novel cell-based therapies targeting areas of significant unmet or poorly met medical need. ReNeuron has used its unique stem cell technologies to develop cell-based therapies for significant diseases where the cells can be readily administered “off-the-shelf” to any eligible patient without the need for additional immunosuppressive drug treatments. The company’s therapeutic candidates for stroke disability and critical limb ischemia are in clinical development and its cell-based treatment for the blindness-causing disease, retinitis pigmentosa, is about to enter the clinic in the US. ReNeuron is also advancing a proprietary platform technology to exploit nanoparticles (exosomes) secreted by stem cells as potential new drug candidates targeting a range of cancers.
David Venables, Ph.D., CEO
Headquarters: Edinburgh, UK
Number of Full-Time Employees: 15
Company Website: www.synpromics.com
Synpromics is the leading developer of synthetic promoters using proprietary technology and cutting edge techniques to custom regulate gene expression to desired levels and the required specificity. Synpromics enables unprecedented control of gene expression through its ability to custom design synthetic cell-type, or condition, specific promoters. The company achieves this by using proprietary technology and cutting-edge techniques including sophisticated bioinformatics and screening approaches.
Stephane Boissel, CEO
Headquarters: Valbonne, France
Number of Full-Time Employees: 50
Company Website: www.txcell.com
TxCell is a biotechnology company that develops platforms for innovative, personalized T cell immunotherapies for the treatment of severe chronic inflammatory and autoimmune diseases with high unmet medical need. TxCell is the only clinical-stage cellular therapy company dedicated to the science of regulatory T lymphocytes (Tregs). Tregs are a recently discovered T cell population for which anti-inflammatory properties have been demonstrated. TxCell is developing two proprietary technology platforms, ASTrIA and ENTrIA. ASTrIA is composed of autologous antigen-specific Type 1 Tregs. Ovasave®, TxCell’s lead drug-candidate originating from the ASTrIA platform, is currently in a Phase IIb clinical trial in refractory Crohn’s disease patients. ENTrIA is composed of Chimeric Antigen Receptor engineered FoxP3+ regulatory T cells (CAR-Treg). In this area, TxCell is pursuing two CAR-Treg development programs in collaboration with leading European research institutions: one targeting Lupus Nephritis with Ospedale San Raffaele in Milan and the other targeting Bullous Pemphigoid.