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Lysogene is a biopharmaceutical gene therapy company with lead programs in neurodegenerative lysosomal storage disorders that affect children. Founded in 2009 by Karen Aiach, whose own child was affected by a neurodegenerative disease, Lysogene is rooted in a deep and compassionate understanding of the impact these diseases have on patients and families. Karen`s personal experience fuels her determination to deliver real solutions that will improve patient outcomes and enhance quality of life for both patients and caregivers. Over the past 10 years, Lysogene has acquired deep experience in developing gene therapies—from early discovery through the clinical, registration and review phases. Our strong science and execution track record has been validated by our partnership with Sarepta Therapeutics and the support of top-tier investors, with nearly $100m raised since inception.
Affiliated Dermatologists, S.C. is a Brookfield, WI-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Abeona Therapeutics (Nasdaq: ABEO) is a fully-integrated gene and cell therapy company at the forefront of the rapidly-advancing field of genetic medicine. The Company`s multi-platform expertise across the manufacture, delivery, development, and discovery of novel gene and cell therapies has it uniquely positioned for success. Underpinning the Company`s robust pipeline is its fully-operational manufacturing facility producing therapies and vectors for preclinical and clinical studies. Abeona is also developing the AIM™ Vector Platform: 100+ next-generation AAV capsids for delivering gene therapies targeting a wide range of organs and multiple routes of delivery. A robust and diverse pipeline is led by a novel gene-corrected cell therapy poised to enter Phase 3 in mid-2019 and complemented by one-time gene therapy candidates across four lysosomal storage diseases. Several preclinical discoveries are led by an emerging program in cystic fibrosis that uses the AIM vector platform and a capsid that has shown potential across inherited and acquired retinal diseases.
Galenea Corp is a Cambridge, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Code Bio was founded on the belief that more can be done for people living with debilitating genetic diseases. We are driven by the potential of our transformative technology to treat the untreatable and cure the incurable. Code Bio is developing highly targeted genetic medicines with its proprietary, novel, multivalent, synthetic DNA delivery platform, 3DNA®, which has been engineered to overcome many of the challenges inherent with the delivery of genetic medicines. 3DNA offers unparalleled tissue and cell targeting specificity, improved bioavailability, capability to deliver large genetic payloads, potential ability to re-dose, and a scalable, modular, reproducible manufacturing process. Our 3DNA delivery platform is poised to transform the field of genetic medicines, enabling targeted delivery of gene therapy, RNAi and other genetic modalities. Currently we are advancing an internal pipeline of genetic medicines focused on select diseases without a cure (Duchenne`s Muscular Dystrophy and Type 1 Diabetes), as well as establishing partnerships to take forward programs in both rare and prevalent diseases.