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At Spark Therapeutics, we don`t follow footsteps. We create the path. We were born of innovation, springing from the curiosity, imagination and dedication of remarkable scientists and healthcare visionaries. Our mission is seemingly impossible to others, but not to us: Challenge the inevitability of genetic disease by discovering, developing and delivering treatments in ways unimaginable – until now. Since our founding, we have been committed to bringing a wide range of expertise to build a fully integrated gene therapy company focused on inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver, such as hemophilia. We are seeking talented individuals with diverse experiences, abilities and interests who have the curiosity, courage and drive to reimagine a new health care paradigm. Join us on a journey through uncharted territory – seeking to bring gene therapy for genetic diseases to people worldwide. The resilience of the people we serve is our inspiration to break barriers, as we strive to turn genes into medicine for those with inherited diseases. Spark is a member of the Roche Group.
Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Our most advanced product candidate, apitegromab (SRK-015), a selective first-in-class inhibitor of the activation of the growth factor myostatin in skeletal muscle, is currently in development for the treatment of spinal muscular atrophy, or SMA. We are currently evaluating apitegromab in our TOPAZ Phase 2 proof-of-concept trial in patients with later-onset (Type 2 and Type 3) SMA. Positive six-month interim analysis results were announced in October 2020 and top-line data for the full 12-month treatment period are anticipated in the second quarter of 2021. In addition, we are conducting our DRAGON Phase 1 proof-of-concept trial for SRK-181, a highly specific inhibitor of latent TGFβ1 activation, in patients with locally advanced and metastatic solid tumors. We believe SRK-181 has the potential to overcome resistance and meaningfully increase the number of patients who may benefit from checkpoint inhibitors, such as anti-PD-1 or anti-PD-L1 therapies. Utilizing our proprietary platform, we are creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia.
PolarityTE, is the owner of a novel regenerative medicine and tissue engineering platform developed and patented by Denver Lough MD, PhD. This radical and proprietary technology employs a patients` own cells for the healing of full-thickness functionally-polarized tissues. If clinically successful, the PolarityTE platform will be able to provide medical professionals with a truly new paradigm in wound healing and reconstructive surgery by utilizing a patient`s own tissue substrates for the regeneration of skin, bone, muscle, cartilage, fat, blood vessels and nerves. It is because PolarityTE uses a natural and biologically sound platform technology, which is readily adaptable to a wide spectrum of organ and tissue systems, that the company and its world-renowned clinical advisory board, are poised to drastically change the field and future of translational regenerative medicine.
Founded in 2017 by Flagship Pioneering, Cygnal Therapeutics is the first company to build a platform to develop drugs in the field of exoneural biology. Exoneural biology is broadly implicated in human health and disease. Fueled by our dynamic Exoneural Medicine Platform™, we are creating new medicines across multiple therapeutic areas, starting with cancer and inflammatory diseases.
Ikaria is one of the leading companies in the Healthcare, Pharmaceuticals, and Biotech sector.