Wellington Management

Atsena Therapeutics is a clinical-stage gene therapy company focused on developing treatments for inherited retinal diseases that can lead to blindness. Their clinical pipeline includes therapies for conditions such as X-linked retinoschisis and GUCY2D-associated Leber congenital amaurosis. Atsena employs an adeno-associated virus (AAV) technology platform, including a novel spreading capsid, to specifically target these retinal disorders. The company aims to prevent vision loss caused by genetic mutations through innovative ocular gene therapy solutions. With a commitment to advancing treatments for some of the most challenging forms of inherited retinal disease, Atsena Therapeutics seeks to improve the lives of patients affected by these conditions.

nChroma Bio is a biotechnology company specializing in targeted genetic medicine. It operates an integrated product engine for safe, accurate, and targeted in vivo administration, aiming to overcome limitations of current genetic medicine techniques. The company focuses on liver-targeted therapies, initially for chronic hepatitis B and D, leveraging epigenetics to enable precise and specific gene regulation. This approach allows for therapies with programmable tissue specificity, potentially offering durable and targeted cures for the liver and other organs.

Element Biosciences, Inc., established in 2017 and headquartered in San Diego, California, specializes in developing innovative genetic analysis tools for research and diagnostic markets. The company's core business revolves around its disruptive DNA sequencing technology, which encompasses surface chemistry, sequencing chemistry, detection methods, and data analysis. Element Biosciences aims to enhance accessibility to next-generation sequencing by offering a modular, high-performing platform that delivers high-quality data and workflow flexibility, thereby reducing run and capital costs.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative treatments for severe hematologic disorders. Founded in 2017, the company specializes in human genetics-based therapeutics that aim to improve red blood cell production in patients suffering from various hematologic diseases. Disc Medicine's pipeline includes therapeutic candidates targeting critical pathways involved in red blood cell biology, such as heme biosynthesis and iron homeostasis. Notable programs include bitopertin, which is being developed for conditions like erythropoietic porphyrias and Diamond-Blackfan anemia. Through its research and development efforts, Disc Medicine seeks to transform the lives of patients affected by these challenging disorders.

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 founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

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 founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics based on CRISPR technology to address genetic disorders. Founded in 2017 and headquartered in Berkeley, California, the company utilizes its proprietary X-Editing (XE) platform, which features highly engineered CRISPR enzymes designed to improve the efficacy, specificity, and deliverability of genome editing. Scribe Therapeutics aims to overcome existing limitations in genetic medicine by creating custom-engineered enzymes and advanced delivery systems. Their approach seeks to establish CRISPR-based therapies as a new standard in clinical care, potentially transforming the treatment landscape for various conditions, including immuno-oncology, degenerative disorders, and enzyme replacement therapy. By prioritizing therapeutic precision and allele-specific targeting, Scribe Therapeutics is committed to expanding access to groundbreaking treatments that can significantly impact patient lives.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

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 focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

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.

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.

Goldfinch Bio, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, dedicated to discovering and developing precision therapies for patients with kidney diseases. The company focuses on conditions such as focal segmental glomerulosclerosis, diabetic nephropathy, and polycystic kidney disease. Key product candidates include GFB-887, a selective inhibitor of the TRPC5 channel, and GFB-024, a cannabinoid receptor 1 inverse agonist. Goldfinch Bio integrates advancements in kidney genetics and biology through its Kidney Genome Atlas, a genomic registry that aids in identifying new disease targets and biomarkers. Additionally, the company employs stem cell science to create human organoid models, which facilitate the validation of novel therapeutic targets. Goldfinch Bio has established a strategic collaboration with Gilead Sciences to further enhance its research and development efforts. Founded in 2016, Goldfinch Bio aims to transform the treatment landscape for progressive kidney disease.

Freeline Therapeutics, established in 2015 and based in Stevenage, UK, is a clinical-stage biotechnology company specializing in the development of systemic adeno-associated virus (AAV) vector-mediated gene therapies. The company focuses on treating inherited, debilitating diseases by delivering functional copies of therapeutic genes into human liver cells, aiming to express functional proteins in the patient's bloodstream. Freeline's advanced product candidates include FLT180a for hemophilia B, FLT190 for Fabry disease, and FLT201 for Gaucher disease Type 1, all in various stages of clinical trials. Additionally, Freeline offers expertise in CMC process development, analytics, and AAV technologies.

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.

Nightstar Therapeutics plc is a clinical-stage gene therapy company based in London, focused on developing innovative one-time treatments for rare inherited retinal diseases. The company’s lead product candidate, NSR-REP1, is currently in phase 3 clinical development for choroideremia, while NSR-RPGR is undergoing phase 2/3 trials for X-linked retinitis pigmentosa. Additionally, Nightstar is developing NSR-BEST1 and NSR-ABCA4, with the latter in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address the unmet medical needs of patients suffering from these conditions.

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 dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Moderna, Inc. is a biotechnology company established in 2010 and headquartered in Cambridge, Massachusetts. The company specializes in developing therapeutics and vaccines using messenger RNA (mRNA) technology, which provides instructions for cells to produce proteins essential for various biological functions. Moderna has a diverse pipeline, with numerous programs targeting infectious diseases, immuno-oncology, rare diseases, and cardiovascular conditions. As of September 2024, it has 40 mRNA development candidates in clinical trials across multiple therapeutic areas. The company's mRNA technology gained significant recognition with the authorization of its COVID-19 vaccine in December 2020. Moderna maintains strategic alliances with various organizations, including major pharmaceutical companies and research institutions, to enhance its research and development efforts.

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.

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.