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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.
Neuron23™ Inc. is an early stage biotechnology company focused on developing precision medicines for genetically defined neurological and immunological diseases.
Advaxis, Inc., a clinical development stage biotechnology company, focuses on developing immunotherapies for cancer and infectious diseases. Its Lm -LLO immunotherapy platform technology stimulates the immune system to induce antigen-specific anti-tumor immune responses involving innate and adaptive arms of the immune system by inhibiting the T-cells, Tregs, myeloid-derived suppressor cells, and MDSC to promote immunologic tolerance of cancer cells in the tumor. The company’s platform technology also facilitates the immune response by altering the tumor microenvironment to reduce immunologic tolerance in the tumors that makes the tumor more susceptible to immune attack. It has approximately 15 distinct immunotherapies in various stages of development, directly developed by the company and through strategic collaborations. The company’s products in clinical development stage comprise ADXS-HPV that is being evaluated in various clinical trials for cervical cancer, head and neck cancer, and anal cancer; ADXS-PSA for the treatment of prostate cancer; and ADXS-cHER2 for the treatment of overexpressing cancers, such as breast, gastric, and other cancers in humans, as well as for osteosarcoma in canines. Advaxis, Inc. was founded in 2002 and is based in Princeton, New Jersey.
Response Genetics is a Los Angeles, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Bamboo was formed to advance the work of Dr. Richard Jude Samulski, the Director of the Gene Therapy Center at the University of North Carolina (UNC), from the laboratory into human clinical trials where it could meet the requirements for drug approval. Dr. Samulski is a pioneer in the field of gene therapy. Over thirty (30) years ago, Dr. Samulski was the first person to realize the potential to use the adeno-associated virus (AAV) as a vehicle to replace a defective gene with a healthy gene. Since that time, he has re-engineered the naturally occurring virus to target delivery to certain tissues, de-target other tissues, and improve its safety. This has resulted in over twenty (20) patents related to the use of AAV for therapeutic uses. Dr. Samulski continues to lead innovation in the field of vector optimization and AAV re-engineering.