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Ribon Therapeutics is a biotech company focused on developing first-in-class therapeutics targeting mono(ADP-ribosyl)ating PARPs for cancer.
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.
Prothena Corporation plc (Nasdaq: PRTA) Prothena is a global biotechnology company seeking to fundamentally alter the course of progressive diseases. We are a late-stage clinical company focused on the discovery, development, and commercialization of novel protein immunotherapies for the treatment of diseases that involve protein misfolding and inflammatory cell adhesion disorders. Our team has a recognized track record both for profound scientific discoveries and for the development of therapeutics that have become leading commercial products in their respective therapeutic categories. Prothena`s pipeline focuses on therapeutic monoclonal antibodies directed specifically to disease-causing proteins and our antibody-based product candidates target a number of potential indications, including AL amyloidosis (NEOD001), Parkinson’s disease and other related synucleinopathies (PRX002), ATTR amyloidosis (PRX004), and novel cell adhesion targets involved in psoriasis, psoriatic arthritis and other inflammatory diseases (PRX003). Prior to December 2012, Prothena’s business operated as part of Elan Corporation, plc. Prothena’s business consists of a substantial portion of Elan’s former drug discovery business platform. After the separation from Elan and the related distribution of our ordinary shares to Elan’s stockholders, our ordinary shares began trading on The Nasdaq Global Market under the symbol “PRTA” on December 21, 2012.
Retrophin is a biopharmaceutical company focused on the discovery, acquisition, development and commercialization of drugs for the treatment of debilitating and often life-threatening diseases for which there are currently limited patient options.
StrideBio is a product-driven, fully integrated gene therapy company focused on creating and developing innovative genetic medicines with life-changing or curative potential for patients with devastating conditions, including monogenic rare disease and beyond. We leverage our proprietary structure-inspired adeno-associated virus (AAV) vector engineering platform to create unique and differentiated vectors that improve upon naturally occurring AAV serotypes to overcome current limitations of first-generation gene therapies. Improved characteristics of StrideBio`s AAV vectors include evasion of pre-existing neutralizing antibodies, tissue targeting and de-targeting, enhanced potency, and manufacturability at scale. Combined with our gene construct design expertise and in-house manufacturing capabilities, StrideBio is positioned to generate best-in-class genetic medicines that allow more patients to benefit with maximum efficiency. StrideBio is based in a state-of-the-art 24,000-square-foot facility in Research Triangle Park, N.C, which houses our offices, research labs and in-house AAV manufacturing facilities.