Wellington Management

nChroma Bio's integrated product engine allows for safe, accurate, and targeted in vivo administration, addressing major shortcomings of current genetic medicine techniques.

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.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

Chroma Medicine is a genomic medicine company based in Cambridge, Massachusetts, founded in 2021. The company focuses on epigenetic editing, aiming to revolutionize the treatment of genetically driven diseases. By utilizing epigenetics, which is nature's inherent mechanism for regulating genes, Chroma Medicine develops programmable epigenetic editors that target specific genes and control chromatin conformation. This innovative approach combines a DNA binding domain with epigenetic effector domains, providing medical practitioners with a new class of therapeutics that offers enhanced control over gene expression.

Synthego Corporation is a genome engineering company focused on advancing life science research and therapeutic development to improve human health. Founded in 2012 and headquartered in Redwood City, California, Synthego offers a comprehensive platform that integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology. The company's product portfolio includes engineered cells, CRISPR kits, and bioinformatics tools designed for various applications such as pathway analysis, target validation, disease modeling, and diagnostics. By leveraging machine learning and automation, Synthego enables researchers in biotechnology and academia to conduct efficient and precise gene editing, facilitating rapid advancements in drug discovery and development. The company serves a global customer base, distributing its products through various channels, including online sales and partnerships in multiple countries.

Chroma Medicine is a genomic medicine company based in Cambridge, Massachusetts, founded in 2021. The company focuses on epigenetic editing, aiming to revolutionize the treatment of genetically driven diseases. By utilizing epigenetics, which is nature's inherent mechanism for regulating genes, Chroma Medicine develops programmable epigenetic editors that target specific genes and control chromatin conformation. This innovative approach combines a DNA binding domain with epigenetic effector domains, providing medical practitioners with a new class of therapeutics that offers enhanced control over gene expression.

Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing innovative therapies for degenerative disorders linked to nucleotide repeat expansions. Founded in 2017 and headquartered in Solana Beach, California, the company is advancing its lead program aimed at treating Friedreich’s ataxia. Additionally, Design Therapeutics is exploring treatments for other conditions such as Fragile X syndrome and myotonic dystrophy, utilizing its proprietary GeneTACTM technology, which involves small-molecule gene-targeted therapeutics designed to modify the underlying causes of these inherited diseases. The company is committed to addressing serious monogenic disorders driven by nucleotide repeat mutations through its ongoing research and development efforts.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

Novellus is a biotechnology company working to develop engineered cellular medicines. The company uses its non-immunogenic mRNA molecules to reprogram and gene-edit cells safely, and with high efficiency. They engineer precision medicines for patient segments and select from its technology toolbox for in vivo, ex vivo, allogeneic and autologous delivery. Novellus platform enables the high-level expression of gene-editing and cell-reprogramming proteins for high-specificity gene repair and both autologous and allogeneic cell therapies. The company is advancing its lead program, a breakthrough therapy for a rare dermatologic disease, through pre-clinical studies. Novellus was founded in 2015 and is headquartered in Cambridge, Massachusetts, United States.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

Escape Bio is a clinical-stage biopharmaceutical company that develops small molecule drugs intended to treat neurodegenerative diseases. The company is developing therapeutics for the novel, precisely therapies for genetic neurodegenerative diseases. Escape Bio's pipeline includes candidates targeting known genetic drivers of multiple diseases, including ESB1609, a small molecule S1P5 receptor agonist for the treatment of CNS lysosomal storage disorders and ESB5070, a small molecule kinase inhibitor for Parkinson’s disease patients who have an LRRK2 G2019S variant and an Alzheimer's disease program targeting ApoE4. E-scape Bio was founded in 2015 and is headquartered in San Francisco, California, United States.

Dyne Therapeutics is a biotechnology company based in Waltham, Massachusetts, dedicated to developing targeted therapies for genetically driven muscle diseases. Founded in 2017, the company focuses on advancing treatments for conditions such as myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral dystrophy, among others. Utilizing its innovative FORCE platform, Dyne Therapeutics aims to deliver disease-modifying therapies with enhanced precision to skeletal, cardiac, and smooth muscle tissues. The company's lead product candidates, DYNE-101 and DYNE-251, are currently undergoing phase 1/2 clinical trials, reflecting its commitment to addressing the unmet medical needs of patients with serious muscle disorders.

Freeline Therapeutics is a clinical-stage biotechnology company focused on developing innovative adeno-associated virus (AAV) vector-mediated gene therapies aimed at treating inherited systemic diseases. The company employs engineered AAV vectors and capsids, alongside novel promoters and transgenes, to introduce functional copies of therapeutic genes into human liver cells. This process allows for the production of essential proteins in the bloodstream, addressing deficiencies caused by genetic disorders. Freeline's key product candidates include FLT180a for hemophilia B, FLT190 for Fabry disease, and FLT201 for Gaucher disease Type 1. Their ongoing development programs encompass various clinical trials to advance these gene therapies and provide transformative solutions for patients suffering from debilitating conditions.

Goldfinch is a clinical-stage biotech company focused on discovering and developing precision therapies for patients with kidney disease. It is integrating breakthroughs in kidney genetics and biology to identify new therapeutic targets and advance first-in-class drug candidates to treat patients with kidney disease. Its Product Engine will industrialize the integration of genetics and kidney biology and confer a differentiated ability to identify, validate, and pursue novel therapeutic targets to treat progressive kidney disease. The company was founded in 2016 and is based in Cambridge, Massachusetts.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

NightstaRx Ltd. is a U.K. startup company. Gene therapy is the use of a gene to treat disease. The approach could enable treatments for a several rare conditions caused by mutations in a single gene.

Allena Pharmaceuticals is a late-stage clinical biopharmaceutical company based in Newton, Massachusetts, that specializes in developing and commercializing oral enzyme therapeutics for rare and severe metabolic and kidney disorders. The company's primary focus is on conditions that involve the excessive accumulation of metabolites such as oxalate and urate, which can lead to complications like kidney stones and chronic kidney disease. Allena's lead product candidate, reloxaliase, is currently undergoing a pivotal Phase 3 clinical program aimed at treating enteric hyperoxaluria, a disorder linked to significantly high urinary oxalate levels. Additionally, the company is developing ALLN-346, a novel enzyme designed to address hyperuricemia in patients with moderate to severe chronic kidney diseases. Allena Pharmaceuticals aims to provide innovative treatments that address unmet medical needs in these patient populations.

Rhythm Metabolic is the subsidiary of Rhythm Pharmaceuticals that develops setmelanotide (RM-493), a novel peptide MC4 agonist for the treatment of obesity caused by genetic deficiencies in the MC4 pathway. It was established in 2013 and is headquartered in Boston, Massachusetts.

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.

Moderna, Inc. is a biotechnology company based in Cambridge, Massachusetts, that specializes in developing therapeutics and vaccines using messenger RNA technology. Founded in 2010, the company focuses on various therapeutic areas, including infectious diseases, oncology, rare genetic disorders, and cardiovascular diseases. As of September 2024, Moderna has 40 mRNA development candidates in clinical development, demonstrating its commitment to advancing medical science. The company's mRNA platform has gained significant recognition, particularly with the authorization of its COVID-19 vaccine in December 2020. Moderna has established strategic collaborations with notable organizations such as AstraZeneca, Merck & Co., and the Bill & Melinda Gates Foundation. Additionally, it collaborates with Lonza Ltd. and Catalent for the manufacturing of its COVID-19 vaccine. Through its innovative approach, Moderna aims to provide effective solutions for a range of health challenges.

Invitae is a genetic information company focused on integrating genetic insights into everyday medical practice to enhance healthcare quality for a large population. The company specializes in genetic diagnostics for hereditary disorders, offering a comprehensive service that consolidates multiple genetic tests into a single platform. Invitae's offerings include assessments for genes linked to hereditary cancer, neurological disorders, cardiovascular conditions, pediatric issues, metabolic disorders, and other hereditary ailments. By providing high-quality testing with quicker turnaround times and competitive pricing, Invitae aims to make genetic testing more accessible and effective for patients and healthcare providers alike.

Spark Therapeutics, Inc. is a biotechnology company focused on developing gene therapy products aimed at treating debilitating genetic diseases. Headquartered in Philadelphia, Pennsylvania, the company was founded in 2013 and has established itself as a leader in the field of gene therapy, with a robust pipeline that includes LUXTURNA for retinal dystrophy, as well as several candidates for hemophilia and choroideremia. Spark is also exploring therapies for neurodegenerative diseases and inherited retinal diseases. The company holds a collaboration agreement with Pfizer for the development of hemophilia B treatments and has partnered with Novartis for the commercialization of its lead product outside the United States. Spark Therapeutics leverages a proprietary manufacturing platform and a team with extensive expertise in research, clinical development, and regulatory affairs, building on a legacy of innovation in gene therapy from its origins at The Children’s Hospital of Philadelphia. As of December 2019, Spark operates as a subsidiary of Roche Holding AG.