See the Slate of Presenting Companies!
See the slate of advanced therapy companies selected to present at this year’s conference. These cell therapy, gene therapy and tissue engineering companies will have direct access to 200+ institutional, corporate and venture investors as well as other top executives in the sector. Indicate your interest in attending today to meet with these leading companies.
2017 Company Presenters
Erik Richardson, Research and Development Project Leader
Headquarters: Seattle, US
Number of Full-Time Employees: 43
Company Website: www.acucela.com
Acucela is a Seattle-based, Kubota Pharmaceutical Group company committed to translating innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide. Acucela currently develops a retina focused pipeline. It includes technologies for Age Related Macular Degeneration (AMD), Diabetic Retinopathy and the orphan indications, Retinitis Pigmentosa (RP) and Stargardt Disease (STGD). The RP program is an optogenetic adeno-associated virus (AAV) gene therapy vector that targets human rhodopsin to the ON bipolar cells of the RP patient’s retina to restore visual signal.
Jan Nilsson, CEO
Headquarters: Lund, Sweden
Number of Full-Time Employees: 3
Company Website: www.combigene.com
CombiGene is combining modern neuroscience with recent advances in gene delivery, and has developed a method shown to suppress epileptic seizures in preclinical studies. The company’s current focus is on continuing to develop this method into an effective and safe therapy for epilepsy patients, but the method may also have development potential as a means of treating other neurological disorders. Founded on the basis of scientific discoveries made at Lund University and the University of Copenhagen, CombiGene has offices at Medicon Village in Lund, Sweden. The company is public and listed on the Swedish marketplace AktieTorget.
Arthur Lahr, CEO
Headquarters: Amsterdam, The Netherlands
Number of Full-Time Employees: 25
Company Website: www.kiadis.com
ABOUT KIADIS PHARMA
Kiadis Pharma is focused on cell-based immunotherapy products, as an adjunctive to a haploidentical hematopoietic stem cell transplantation (HSCT), for the treatment of blood cancers and inherited blood disorders. The company’s product candidates have the potential to make allogeneic HSCT safer and more effective for patients. Based on positive results from a single dose Phase II trial with lead product ATIR101™ in patients with blood cancer, Kiadis submitted a marketing authorisation application to the European Medicines Agency (EMA) in April 2017 for approval of ATIR101™ across the EU as an adjunctive treatment in HSCT for malignant disease. In addition, Kiadis has received regulatory approval in various countries to start dosing patients in a Phase III trial with ATIR101™ that will be performed across Europe and North America. ATIR101™ has been granted Orphan Drug Designations both in the US and EU.
Michael Schuster, Co-Founder and Head of Pharma Partnering
(NASDAQ: MESO; ASX: MSB)
Headquarters: Melbourne, Australia
Number of Full-Time Employees: 110
Company Website: www.mesoblast.com
Mesoblast is a world leader in developing innovative cellular medicines. The company has established the industry’s most clinically advanced and diverse portfolio of cell-based products with three programs in active Phase III clinical studies. Mesoblast has leveraged its proprietary technology platform, which is based on specialized cells known as mesenchymal lineage adult stem cells (MLCs), to establish a broad portfolio of late-stage product candidates. These allogeneic, off-the-shelf cell product candidates target advanced stages of diseases with high, unmet medical needs including cardiovascular diseases, immune-mediated and inflammatory conditions, spine orthopedic disorders and oncology and hematology diseases. The company’s licensee in Japan has launched its mesenchymal stem cell-based product, which is the first allogeneic cell-based product to receive full approval in Japan.
Mark Rothera, President and CEO
Headquarters: London, UK
Number of Full-Time Employees: 45
Company Website: www.orchard-tx.com
ABOUT ORCHARD THERAPEUTICS
Orchard Therapeutics is a clinical-stage biotechnology company dedicated to bringing transformative gene therapies to patients with rare diseases of high unmet medical need. The company’s development programmes exploit the potential of autologous ex-vivo haematopoietic stem cell gene therapy to restore normal gene function. OTL-101, Orchard’s lead programme for severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA-SCID), is in pre-registration with very strong efficacy and safety results accrued to date. Orchard is also developing a franchise in inherited metabolic disorders, with a lead program targeting mucopolysaccharidosis type IIIA (MPS-IIIA or Sanfilippo syndrome type A), expected in the clinic in 2018. Orchard Therapeutics has been named by FierceBiotech as one of 2016’s “Fierce 15” biotechnology companies.
Mike Robbat, VP of Business Development, Regenerative Medicine and General Counsel
Headquarters: Boston, US
Number of Full-Time Employees: 45
Company Website: www.orig3n.com
Orig3n is pursuing iPSC-derived, allogeneic cell therapy programs in several indications. The company has assembled a crowdsourced, fully-consented cell bank for this purpose, and have identified cell lines that are matched to the immune profiles of 90% of the US population. The company is developing complimentary cell manufacturing capabilities. Orig3n also offers a large suite of direct-to-consumer genetic tests. These genetic tests are currently sold in North America, Asia and Latin America. The company’s founders are industry veterans who have successfully exited three companies. Their last company was sold to a $5.4B market cap publicly traded company at 78% IRR and a $24:1 return for investors. Orig3n’s investor syndicate includes Hatteras Venture Partners, LabCorp, Mountain Group, Alexandria Venture Investments, Spectrum Health Ventures, Syno Capital, KTB Ventures, Defta Partners, Venture Capital Multiplier Fund, other institutions and the company’s founders.
Peter Nolan, Chief Business Officer
Headquarters: Oxford, UK
Number of Full-Time Employees: 300
Company Website: www.oxfordbiomedica.co.uk
ABOUT OXFORD BIOMEDICA
Oxford BioMedica has 20 years of experience in the field of gene and cell therapy and was the first organization to treat humans in vivo with lentiviral-based vectors. Today, Oxford BioMedica has built the company’s LentiVector® platform of exclusive cutting-edge technologies and capabilities with which the company designs, develops and produces gene and cell-based medicines for Oxford BioMedica itself and for partners. The company already has product-related partnerships with Novartis, Immune Design, Orchard Therapeutics, licensed products and technology rights to Sanofi and GlaxoSmithKline and a research and development collaboration with Green Cross LabCell. In addition, Oxford BioMedica has its own proprietary pipeline of gene and cell therapy products addressing neurodegenerative and ocular diseases and a range of cancers, for which there are either no treatments or where therapy remains inadequate.
Eric Halioua, President and CEO
Headquarters: Liège, Belgium
Number of Full-Time Employees: 14
Company Website: www.pdc-line-pharma.com
ABOUT PDC*LINE PHARMA
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 focuses on the development of a clinical candidate for lung cancer. The company regroups a team of 15 people with a seasonal management team and raised close to €7 million in equity and loans.
Frank Hazevoets, Chief Financial Officer
Headquarters: Mont-Saint-Guibert, Belgium
Number of Full-Time Employees: 60
Company Website: www.promethera.com
ABOUT PROMETHERA BIOSCIENCES
Promethera Biosciences is a global innovator in liver cell-based medicines whose mission is to help patients overcome acute and chronic liver diseases. The company’s lead clinical program, derived from Promethera’s patented cell technology platform HepaStem, is designed to benefit from its immune-modulatory and anti-fibrotic properties. Promethera is a team of international experts operating out of research and development and GMP facilities in Mont-Saint-Guibert, Belgium and Durham, North Carolina.
Olav Hellebo, CEO
Headquarters: Pencoed, Wales
Number of Full-Time Employees: 66
Company Website: www.reneuron.com
ReNeuron is a leading, clinical-stage cell therapy development company. 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.
Claudia D’Augusta, CFO
Headquarters: Leuven, Belgium
Number of Full-Time Employees: 80
Company Website: www.tigenix.com
TiGenix is an advanced biopharmaceutical company focused on exploiting the anti-inflammatory properties of stem cells to develop novel therapies for serious medical conditions. The company’s current therapeutic focus is inflammatory, autoimmune and cardiac diseases, which TiGenix targets through products developed from two proprietary allogeneic platforms of adipose-derived (eASC) and cardiac stem cells (CSC). Three product candidates are currently under clinical development. Cx601 is a local injectable eASC therapy for the treatment of complex perianal fistulas. Cx601 is on track for European approval by the European Medicines Agency in 2017. A global Phase III clinical trial to support a Biologics License Application (BLA) is expected to start in 2017. Takeda Pharmaceuticals holds development and commercialization rights to Cx601 outside of the US. Cx611 is an intravenous administration of eASCs being evaluated in a Phase I/II trial in severe sepsis. AlloCSC-01 is an intracoronary administration of a suspension of allogeneic CSCs for the treatment of acute ischemic heart disease.
Stephane Boissel, CEO
(EURONEXT PARIS: TXCL)
Headquarters: Valbonne, France
Number of Full-Time Employees: 43
Company Website: www.txcell.com
TxCell is a biotechnology company that develops innovative cell immunotherapies based on regulatory T lymphocytes (Tregs). TxCell is targeting a range of severe inflammatory and autoimmune diseases with high unmet medical needs, including Crohn’s disease, lupus nephritis, bullous pemphigoid and multiple sclerosis, as well as transplant rejection. TxCell is the only clinical-stage cellular therapy company fully dedicated to the science of Tregs. Tregs are a recently discovered T-cell population for which anti-inflammatory properties have been demonstrated. Contrary to conventional approaches based on non-specific polyclonal Tregs, TxCell is exclusively developing antigen-specific Tregs. This antigen specificity may either come from genetic modifications with Chimeric Antigen Receptor (CAR) or from pre-existing Treg T-Cell Receptor (TCR). TxCell is developing two proprietary technology platforms, ENTrIA, which is composed of genetically engineered Tregs, and ASTrIA, which is composed of non-modified naturally antigen-specific Tregs.
Paul Laikind, Ph.D., President and CEO
Headquarters: San Diego, US
Number of Full-Time Employees: 50
Company Website: www.viacyte.com
ViaCyte is a privately-held regenerative medicine company developing novel cell replacement therapies as potential long-term diabetes treatments to reduce the risk of hypoglycemia and diabetes-related complications. ViaCyte has two products in clinical development. The PEC-Direct™ product candidate delivers stem cell-derived PEC-01 pancreatic progenitor cells in a non-immunoprotective device and is being developed for type 1 diabetes patients who have hypoglycemia unawareness, extreme glycemic lability and/or recurrent severe hypoglycemic episodes. The PEC-Encap™ (also known as VC-01) product candidate delivers the same pancreatic progenitor cells in an immunoprotective device and is being developed for all patients with diabetes, type 1 and type 2, who use insulin. ViaCyte is headquartered in San Diego, California. The company is funded in part by the California Institute for Regenerative Medicine (CIRM) and JDRF.