Wellington Management

Atsena Therapeutics is a clinical-stage gene therapy company dedicated to developing innovative treatments for inherited retinal diseases. The company focuses on therapies for conditions such as X-linked retinoschisis and GUCY2D-associated Leber congenital amaurosis. Utilizing an advanced adeno-associated virus (AAV) technology platform, including a novel spreading capsid, Atsena aims to target specific retinal conditions to prevent blindness resulting from genetic mutations. Their research is centered on providing effective ocular gene therapies that address significant challenges associated with inherited retinal diseases, with the goal of reversing or halting vision loss.

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.

Avidity Biosciences, Inc. is a biopharmaceutical company based in La Jolla, California, specializing in the development of oligonucleotide-based therapies, particularly through its innovative Antibody Oligonucleotide Conjugates (AOC) platform. This technology aims to address the limitations of traditional oligonucleotide therapies by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide treatments. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disease. In addition to this, the company is advancing multiple programs focused on muscle-related conditions, including muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Furthermore, Avidity is expanding its research to include immune and other cell types, positioning itself at the forefront of biopharmaceutical innovation for serious diseases.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing 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 that specializes in accelerating life science research and development to enhance human health. Established in 2012 and headquartered in Redwood City, California, Synthego utilizes an innovative platform that integrates hardware, software, bioinformatics, chemistries, and molecular biology. This full-stack approach provides researchers with tools for gene editing, including engineered cells, CRISPR kits, and bioinformatics for guide RNA optimization and analysis. The products support various applications in basic research, therapeutic development, disease modeling, and diagnostics. Synthego serves a diverse range of customers, including biotechnology firms, universities, and therapeutic developers worldwide, distributing its offerings online and through partners in multiple countries. By leveraging automation and machine learning, Synthego enables genetic engineers and medical professionals to conduct research that is both rapid and cost-effective.

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 therapies utilizing engineered CRISPR technologies to address genetic disorders. Founded in 2018 and based in Berkeley, California, Scribe Therapeutics is known for its proprietary X-Editing (XE) molecules, which are advanced CRISPR enzymes designed to enhance efficacy, specificity, and deliverability compared to existing genome editing methods. The company's platform aims to overcome the limitations of current technologies by creating custom-engineered enzymes and delivery systems that facilitate precise genetic modifications. Scribe Therapeutics is committed to establishing CRISPR-based therapies as a new standard of clinical care, with the potential to significantly impact the treatment landscape for a variety of diseases, including those related to immuno-oncology, degenerative disorders, and enzyme replacement therapy.

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 headquartered in Solana Beach, California, focused on developing innovative therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company specializes in a novel class of small-molecule gene-targeted chimera therapeutics known as GeneTACTM molecules, which aim to modify disease progression by addressing the root causes of these conditions. Its lead program targets Friedreich’s ataxia, while additional efforts are directed toward conditions such as Fuchs endothelial corneal dystrophy and other degenerative diseases like Fragile X syndrome and myotonic dystrophy. Since its incorporation in 2017, Design Therapeutics has been dedicated to advancing its therapeutic candidates to potentially transform treatment options for patients suffering from these inherited disorders.

Verve Therapeutics is a genetic medicines company based in Cambridge, Massachusetts, focused on innovative therapies for cardiovascular disease. Founded in 2018, the company aims to transform the management of cardiovascular conditions from chronic treatments to single-course gene editing medicines. Verve Therapeutics employs advanced gene-editing technology and human genetic analysis to develop targeted therapies that reduce the risk of coronary artery diseases. Its initial programs focus on the PCSK9 and ANGPTL3 genes, which are crucial for lowering harmful blood lipids like low-density lipoprotein cholesterol. The company collaborates with strategic partners, including Beam Therapeutics for delivery technologies and Verily for gene editing delivery vehicles, enhancing its capabilities in the biotechnology and healthcare sectors.

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 for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing 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 that specializes in accelerating life science research and development to enhance human health. Established in 2012 and headquartered in Redwood City, California, Synthego utilizes an innovative platform that integrates hardware, software, bioinformatics, chemistries, and molecular biology. This full-stack approach provides researchers with tools for gene editing, including engineered cells, CRISPR kits, and bioinformatics for guide RNA optimization and analysis. The products support various applications in basic research, therapeutic development, disease modeling, and diagnostics. Synthego serves a diverse range of customers, including biotechnology firms, universities, and therapeutic developers worldwide, distributing its offerings online and through partners in multiple countries. By leveraging automation and machine learning, Synthego enables genetic engineers and medical professionals to conduct research that is both rapid and cost-effective.

Dyne Therapeutics, Inc. 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 muscular dystrophy, as well as rare skeletal, cardiac, and metabolic muscle diseases. Utilizing its proprietary FORCE platform, Dyne Therapeutics delivers nucleic acids and other therapeutic molecules to muscle tissues with high precision, aiming to restore muscle strength and improve patients' quality of life. The company is currently advancing its product candidates, DYNE-101 and DYNE-251, which are in phase 1/2 clinical trials.

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, enabling the production of functional proteins in the bloodstream. Freeline's pipeline includes FLT180a for hemophilia B, FLT190 for Fabry disease, and FLT201 for Gaucher disease Type 1, all in various stages of clinical trials. Additionally, the company offers expertise in CMC process development, analytics, and AAV technologies.

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.

Verve Therapeutics is a genetic medicines company based in Cambridge, Massachusetts, focused on innovative therapies for cardiovascular disease. Founded in 2018, the company aims to transform the management of cardiovascular conditions from chronic treatments to single-course gene editing medicines. Verve Therapeutics employs advanced gene-editing technology and human genetic analysis to develop targeted therapies that reduce the risk of coronary artery diseases. Its initial programs focus on the PCSK9 and ANGPTL3 genes, which are crucial for lowering harmful blood lipids like low-density lipoprotein cholesterol. The company collaborates with strategic partners, including Beam Therapeutics for delivery technologies and Verily for gene editing delivery vehicles, enhancing its capabilities in the biotechnology and healthcare sectors.

Generation Bio Co. is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies aimed at treating both rare and prevalent diseases. Founded in 2016 and formerly known as Torus Therapeutics, the company focuses on creating durable and redosable treatments for conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs a proprietary platform that utilizes closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP) to achieve targeted delivery of genetic materials across various tissues. This approach is designed to enhance treatment efficacy while expanding access to therapies for rare diseases and addressing more common conditions through scalable manufacturing processes.

Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company founded in 2018 and based in South San Francisco, California. The company is dedicated to developing innovative therapeutics to meet unmet medical needs in viral and liver diseases. Its primary focus is on chronic hepatitis B (CHB), with a robust pipeline that includes several drug candidates: ALG-010133, a synthetic oligonucleotide currently in Phase I clinical trials; ALG-000184, a capsid assembly modulator; ALG-020572, another oligonucleotide; and ALG-125097, an siRNA drug candidate. Additionally, Aligos is advancing ALG-055009, a small molecule THR-ß agonist aimed at treating non-alcoholic steatohepatitis (NASH). Aligos aims to leverage its expertise in liver diseases and viral infections to develop targeted antiviral therapies and establish itself as a leader in this therapeutic area.

Nightstar Therapeutics is a clinical-stage gene therapy company based in London, United Kingdom, specializing in the development of innovative one-time treatments for rare inherited retinal diseases. The company is advancing several product candidates, including its lead candidate NSR-REP1, which is in phase 3 clinical development for choroideremia. Additionally, Nightstar is working on NSR-RPGR, currently in phase 2/3 trials for X-linked retinitis pigmentosa, as well as NSR-BEST1. The company is also developing NSR-ABCA4, which is in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address significant unmet medical needs in the field of retinal disorders.

Allena Pharmaceuticals, Inc. is a late-stage clinical biopharmaceutical company based in Newton, Massachusetts, focused on the development and commercialization of innovative oral enzyme therapeutics for rare and severe metabolic and kidney disorders. Founded in 2011, the company’s lead product candidate, reloxaliase, is currently undergoing a pivotal Phase 3 clinical program aimed at treating enteric hyperoxaluria, a condition characterized by elevated urinary oxalate levels that can lead to kidney stones and chronic kidney disease. Additionally, Allena is developing ALLN-346, a novel enzyme intended to address hyperuricemia in patients with moderate to severe chronic kidney diseases. By targeting metabolic disorders associated with the accumulation of harmful metabolites, Allena Pharmaceuticals aims to provide effective treatments for patients facing serious health challenges.

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).

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 for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing therapies.