Adage Capital Management

Tectonic Therapeutic is a biotechnology company dedicated to the discovery and development of innovative drugs targeting G protein-coupled receptors (GPCRs). By utilizing its proprietary technology platform, GEODe, Tectonic aims to create therapeutic proteins and antibodies that effectively modulate GPCR activity. The company seeks to address the complexities associated with GPCR-targeted drug discovery, focusing on developing biologic medicines that have the potential to alter disease progression. Founded by experts in biochemistry and molecular pharmacology, Tectonic is committed to advancing the therapeutic utility of some of the most challenging receptors in this class.

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.

LEXEO Therapeutics is a biotechnology company that specializes in the development of gene therapies, particularly those utilizing adeno-associated virus (AAV) technology. The company’s pipeline includes both rare and non-rare monogenic diseases, with a strong emphasis on hereditary and acquired conditions that present significant unmet medical needs. LEXEO's lead programs and preclinical candidates are primarily developed in collaboration with Weill Cornell Medicine’s Department of Genetic Medicine. The company aims to advance its clinical programs toward commercialization while maintaining an ongoing research partnership with Weill Cornell to enhance its preclinical efforts. Through its innovative approach, LEXEO Therapeutics seeks to transform the treatment landscape for diseases that impose a substantial burden on patients and society.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

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.

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.

Syros Pharmaceuticals is a life sciences company that is focused on treating disease by mapping gene regulatory circuits and modulating the factors that regulate gene expression. Syros has pioneered world-leading gene control research and drug discovery capabilities with a proven ability to integrate disease biology and genomic data--a proficiency that is not well represented in pharmaceutical R&D. Central to the Syros approach is a proprietary platform of carefully integrated assay technologies, bioinformatics, and biologic insights developed by members of Syros' senior leadership. While this scientific approach has applications in many therapeutic areas, Syros has demonstrated success initially in oncology, where it may help address numerous unmet medical needs.

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.

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.

Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases. Prevail was launched in 2017 by The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, and is headquartered in New York, NY.

Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases. Prevail was launched in 2017 by The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, and is headquartered in New York, NY.

PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of orally administered small-molecule therapeutics for the treatment of rare disorders and cancer. The company offers Translarna, used for nonsense mutation Duchenne muscular dystrophy in ambulatory patients, and Emflaza, a corticosteroid for the same condition. PTC Therapeutics is advancing several candidates in clinical development, including Translarna for other rare genetic conditions, RG7916 and RO7034067 for spinal muscular atrophy, and PTC596 and PTC299, which target cancer through dihydrooratate dehydrogenase inhibition. Additionally, the company is developing a gene therapy candidate, PTC-AADC, for Aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics collaborates with various organizations, including F. Hoffman-La Roche Ltd and Massachusetts General Hospital, to further its research and commercialization efforts. Founded in 1998, the company is headquartered in South Plainfield, New Jersey.