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AveXis is a Dallas-based company developing gene therapy for spinal muscular atrophy, an often progressive and disabling muscle wasting disease.
LaJolla Pharmaceutical is a California biotech focused on endogenous peptides and therapies for rare diseases.
ReGenX is the most broadly licensed AAV capsid technology platform in gene therapy. ReGenX’s own pipeline is focused on metabolic, CNS, and retinal diseases.
Immunocore is a London-based company with an immuno-oncology platform technology called ImmTACs. ImmTACs enable the potent targeting of intracellular cancer antigens with controlled pharmacology.
Solid Biosciences is dedicated to developing therapies for Duchenne Muscular Dystrophy, a disease characterized by progressive muscular weakness in boys.
Armo BioSciences is an immuno-oncology company with a pipeline of novel interleukins for cancer.
Odonate Therapeutics is developing a novel oral taxane for breast cancer.
Orchard Therapeutics is a biotechnology company incorporated in September 2015 and dedicated to bringing transformative gene therapies to patients with serious and life-threatening orphan diseases.
Rocket Pharma brings hope and relief to patients with undertreated rare diseases through the development and commercialization of curative first-in-class gene-modified cell therapies.
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