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Spotlight Therapeutics is a venture funded early-stage biotechnology company with a mission to develop a new class of therapeutics by enabling direct in vivo gene editing. Founded by Alex Marson (UCSF), Jacob Corn (UC Berkeley), and Patrick Hsu (Salk Institute) in December of 2017, Spotlight is building its core technology platform to achieve precise in vivo gene editing in targeted cell populations. With a therapeutic focus centered on oncology, our engineered nucleases edit the genes in targeted disease organs thereby inactivating or altering the gene product. We are hiring rapidly to build out our cutting-edge research and development team. This team is responsible for preclinical development ranging from protein engineering, gene editing to immuno-oncology and in vivo safety studies. Join us and work on the next generation of gene editing therapeutics in a fast-paced and exciting environment.
Carmot Therapeutics, Inc. is a biotech company focused on the discovery of innovative drugs for the treatment of metabolic diseases, cancer, and inflammation. Carmot`s vision is to become a leader in drug discovery by unlocking novel therapeutic target space not currently accessible to conventional small molecule technologies.
Blue Sky Biotech Inc is a Worcester, MA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
A pioneer in immuno-modulation by cell specific delivery of oligonucleotide drugs to antigen presenting cells completing IND enabling studies for a groundbreaking anti-GVHD drug for hematopoietic stem cell transplantation in hematological cancers.
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.