Suvretta Capital Management

Benitec Biopharma is using patented ‘gene-silencing’ technology to develop drugs for chronic and life-threatening human diseases. Their transformational technology, ddRNAi, targets and ‘turns off’ specific genes related to disease, providing lasting treatment and potentially cure with a single dose. Compared to traditional approaches to RNA intereference, ddRNAi is easier to deliver, safer to us, more targeted and more efficient. Benitec has a pipeline of in-house and partnered drug development programs based on ddRNAI, including Hepatitis C and B, cancer-associated pain and drug-resistant lung cancer. Our Hepatitis C program is nearing clinical trials in 2013. ddRNAi has the potential to silence genes related to thousands of human diseases and is protected by over 40 global patents. The technology is available from Benitec under a variety of licence options.

Abeona Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing gene and cell therapies for severe, life-threatening rare genetic diseases. The company's leading programs include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, and ABO-102 and ABO-101, which are AAV-based gene therapies targeting Sanfilippo syndrome types A and B, respectively. Additional therapies under development include ABO-201 and ABO-202 for Batten disease, ABO-401 for cystic fibrosis, and ABO-5OX for genetic eye disorders. Abeona also operates the AIM Vector Platform, which focuses on advancing gene therapy delivery methods using next-generation AAV capsids. The company is engaged in a licensing agreement with REGENXBIO for gene therapies addressing multiple genetic disorders. Founded in 1974 and headquartered in New York, Abeona has a fully operational manufacturing facility that supports its preclinical and clinical research efforts.

Benitec Biopharma is using patented ‘gene-silencing’ technology to develop drugs for chronic and life-threatening human diseases. Their transformational technology, ddRNAi, targets and ‘turns off’ specific genes related to disease, providing lasting treatment and potentially cure with a single dose. Compared to traditional approaches to RNA intereference, ddRNAi is easier to deliver, safer to us, more targeted and more efficient. Benitec has a pipeline of in-house and partnered drug development programs based on ddRNAI, including Hepatitis C and B, cancer-associated pain and drug-resistant lung cancer. Our Hepatitis C program is nearing clinical trials in 2013. ddRNAi has the potential to silence genes related to thousands of human diseases and is protected by over 40 global patents. The technology is available from Benitec under a variety of licence options.

Fate Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing programmed cellular immunotherapies for cancer and immune disorders. The company’s pipeline includes various NK- and T-cell immuno-oncology programs, such as FT516 for acute myeloid leukemia and B-cell lymphoma, FT596 for B-cell lymphoma and chronic lymphocytic leukemia, and FT538 for multiple myeloma. Additional programs include FT500, FT576, FT819, and FT-ONO1, targeting solid tumors and hematologic malignancies. Fate Therapeutics is also collaborating with Ono Pharmaceutical Co. Ltd. and Janssen Biotech, Inc. to advance its off-the-shelf iPSC-derived CAR T-cell therapies and other innovative treatments. Founded in 2007, the company leverages insights from leading scientists to create and modulate stem cells for therapeutic applications, aiming to restore health through advanced cellular therapies.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

At Kura Oncology, They are committed to realizing the promise of precision medicines for cancer. The genomics revolution is transforming how they treat cancer. They now understand that how a patient responds to treatment depends in part on the genetic makeup of the cancer and, importantly, we have the knowledge and tools to create targeted treatments and companion diagnostics to identify those patients most likely to benefit. This new era offers the potential for innovative treatments that are safer and more effective for patients with particular cancers. They are leveraging our insights into cancer genetics as well as our core strength of translating novel science into life-saving medicines to advance a pipeline of precision medicines. Their development programs target cancers with high unmet need, including lung, colorectal, thyroid and pancreatic cancers as well as blood cancers such as lymphoma and leukemias.

Benitec Biopharma is using patented ‘gene-silencing’ technology to develop drugs for chronic and life-threatening human diseases. Their transformational technology, ddRNAi, targets and ‘turns off’ specific genes related to disease, providing lasting treatment and potentially cure with a single dose. Compared to traditional approaches to RNA intereference, ddRNAi is easier to deliver, safer to us, more targeted and more efficient. Benitec has a pipeline of in-house and partnered drug development programs based on ddRNAI, including Hepatitis C and B, cancer-associated pain and drug-resistant lung cancer. Our Hepatitis C program is nearing clinical trials in 2013. ddRNAi has the potential to silence genes related to thousands of human diseases and is protected by over 40 global patents. The technology is available from Benitec under a variety of licence options.

HemoShear Therapeutics, LLC is dedicated to discovering novel biological targets and drug treatments for rare metabolic disorders, particularly focusing on inherited conditions such as Organic Acidemias and Nonalcoholic Steatohepatitis, a chronic liver disease. The company employs its proprietary REVEAL-Tx platform to replicate human disease biology, enabling the development and validation of new therapies. By creating human-relevant systems, HemoShear generates significant human response data that aids pharmaceutical and biotechnology partners in making informed decisions and reducing the risk of costly failures in drug development. Founded in 2008 and based in Charlottesville, Virginia, HemoShear Therapeutics aims to improve the quality of life for patients suffering from metabolic defects that disrupt essential biochemical processes.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.