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Maze Therapeutics is a biotechnology company focused on developing medicines that mimic the effects of rare, protective genetic variants to address unmet medical needs. Utilizing its Compass platform, the company identifies genetic variants associated with diseases and maps them to the biological pathways driving these conditions in specific patient populations.

858 Therapeutics is a biotechnology company specializing in the discovery of small molecule therapeutics for novel targets in oncology and immunology. The company focuses on innovative approaches such as synthetic lethality, innate immunity, and RNA modulation, with its lead program being a PARG inhibitor currently in early clinical development.

Founded in 2020 and headquartered in Boston, Massachusetts, Scorpion Therapeutics is a clinical-stage biotechnology company specializing in precision oncology. It develops targeted small-molecule drugs to treat cancer by focusing on three areas: therapies against known oncogenes, agents for currently undruggable targets, and drugs for new targets.

Applied Therapeutics is a biopharmaceutical company focused on developing transformative drugs targeting fatal and debilitating diseases with high unmet medical needs. Its pipeline includes novel product candidates such as AT-007 for rare metabolic diseases like galactosemia, AT-001 for diabetic cardiomyopathy, and AT-003 for diabetic retinopathy.

Adverum Biotechnologies is a clinical-stage company focused on developing gene therapies to treat ocular diseases. Its pipeline includes ADVM-022 for wet age-related macular degeneration, ADVM-043 for alpha-1 antitrypsin deficiency, and preclinical candidates for other indications.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

Boundless Bio develops innovative cancer therapeutics focused on understanding and treating previously untreatable cancers. It specializes in targeting extrachromosomal DNA (ecDNA) biology, a key driver of aggressive cancers.

Synthego is a biotechnology company specializing in genome engineering. It offers integrated hardware, software, bioinformatics, chemistries, and molecular biology platforms to advance basic research and therapeutic development. Its products include engineered cells, CRISPR kits, and bioinformatics tools for gene knockout, target validation, and disease modeling. Synthego serves scientists globally, with sales through distributors in multiple countries and online.

Parse Biosciences is a biotechnology company specializing in single-cell analysis and sequencing solutions. Founded in 2018 and based in Seattle, Washington, the company focuses on developing innovative technologies that simplify the sequencing process for researchers. By employing combinatorial cDNA barcoding directly within cells, Parse Biosciences eliminates the necessity for complex and costly microfluidic instruments. This technology allows for the fixing, freezing, and storage of samples, enabling researchers to conduct experiments with samples collected on different dates in a single run. This capability enhances sample handling and minimizes errors, ultimately improving the accuracy of results. The company aims to increase gene detection in individual cells while addressing common challenges associated with single-cell sequencing, thereby advancing research in various scientific fields.

Expansion Therapeutics is a drug discovery and development company focused on creating small molecule medicines for RNA-mediated diseases. The company specializes in developing novel RNA-targeted drug candidates that aim to address a wide range of severe medical conditions. By leveraging its innovative technology, Expansion Therapeutics seeks to provide transformative oral treatment options, enhancing the therapeutic landscape for patients suffering from these complex diseases.

858 Therapeutics is a biotechnology company specializing in the discovery of small molecule therapeutics for novel targets in oncology and immunology. The company focuses on innovative approaches such as synthetic lethality, innate immunity, and RNA modulation, with its lead program being a PARG inhibitor currently in early clinical development.

Vanqua Bio is a biotechnology company dedicated to discovering and developing innovative medicines for patients with neurodegenerative diseases. The company's proprietary platform employs human genetics and patient-derived neuronal cells to identify and validate novel disease pathways related to lysosomal dysfunction or aberrant immune system activation, aiming to translate these findings into clinically effective treatments.

RareCyte, Inc. is a life sciences company based in Seattle, Washington, founded in 2009. The company specializes in developing advanced platforms for liquid and tissue biopsy analysis, focusing on the detection and characterization of rare cells, particularly circulating tumor cells (CTCs) and circulating endothelial cells (CECs) in blood samples. RareCyte's AccuCyte system provides an open platform that facilitates the unbiased discovery and isolation of these rare cells, supporting various applications in cancer research, cardiovascular disease studies, and diagnostic development. The company offers a range of products, including sample preparation systems, staining kits, automated fluorescence microscopy instruments, and slide imaging systems. By enabling precision medicine approaches, RareCyte aims to provide new tools for medical professionals and researchers to address complex questions in the fields of cancer and other therapeutic areas.

Founded in 2015 and based in Cambridge, Massachusetts, Obsidian Therapeutics develops next-generation controllable cell and gene therapies designed to extend adoptive immunotherapy to every cancer patient. Its innovative approach uses pharmacologic operating systems to precisely control protein activity in cells, enabling physicians to improve upon current cell therapies using simple, safe, orally-active marketed drugs.

Tango Therapeutics is a biotechnology company focused on developing cancer therapies based on the genetic principle of synthetic lethality. It aims to counteract tumor suppressor gene loss, reverse immune evasion in cancer cells, and identify effective drug combinations.

Element Biosciences develops innovative genetic analysis tools for research and diagnostic markets. Founded in 2017, the company specializes in a disruptive DNA sequencing technology that offers high-quality data, workflow flexibility, and affordable access to next-generation sequencing.

Gennao Bio is a privately held company focused on developing innovative targeted nucleic acid therapeutics, primarily aimed at treating oncology and rare monogenic skeletal muscle diseases. The company utilizes its proprietary gene monoclonal antibody (GMAB) platform technology, which features a novel, cell-penetrating antibody that facilitates the non-covalent binding and delivery of various nucleic acid payloads to specific cells. This non-viral delivery system stands out from traditional gene therapy methods by allowing for multiple types of nucleic acids, enabling repeat dosing, and employing established manufacturing processes. Gennao Bio's approach aims to address significant unmet medical needs and improve patient outcomes in the relevant therapeutic areas.

Boundless Bio develops innovative cancer therapeutics focused on understanding and treating previously untreatable cancers. It specializes in targeting extrachromosomal DNA (ecDNA) biology, a key driver of aggressive cancers.

Omega Therapeutics, Inc. is a development-stage biotechnology company based in Cambridge, Massachusetts, founded in 2016. The company specializes in developing genomic medicines aimed at curing diseases through precision tuning of the human genome. It utilizes its proprietary epigenomic programming platform to engineer a new class of programmable epigenetic medicines known as Omega Epigenomic Controllers. These controllers are designed to selectively regulate genomic activity, allowing for the downregulation or upregulation of genes to achieve therapeutic effects. Omega Therapeutics seeks to transform human medicine by harnessing the natural capacity of the genome to treat and cure various diseases.

Graphite Bio is a clinical-stage gene editing company focused on targeted DNA integration to treat serious diseases. Its platform aims to precisely insert, repair, or replace genetic sequences by integrating therapeutic cargoes at predefined genomic locations, enabling correction of mutations or replacement of disease-causing genes while preserving normal regulatory control. Based in South San Francisco, the company pursues high-efficiency precision gene editing to address underlying causes of disease.

Century Therapeutics is a biotechnology company developing allogeneic cell therapies derived from induced pluripotent stem cells to treat cancer and related diseases. Its platform enables engineering of iPSCs into NK and T cells with master cell banks of modified cells that can be expanded and differentiated into standardized, off-the-shelf therapies. The company combines CRISPR-mediated gene editing, proprietary chimeric antigen receptors, Allo-Evasion technology, and manufacturing capabilities to enable scalable production of homogeneous cell products for hematologic and solid tumors. While focused on oncology, its platform also targets autoimmune and inflammatory diseases with the goal of improving cell therapy performance and reducing host immune rejection.

DTx Pharma, LLC is a biotechnology company headquartered in San Diego, California, specializing in the development and commercialization of fatty acid-conjugated peptide therapeutics. Founded in 2017, the company focuses on delivering RNA-based medicines to treat patients with rare and chronic diseases, including retinitis pigmentosa, Duchenne muscular dystrophy, and various central nervous system disorders. DTx Pharma's innovative delivery technology addresses challenges faced by previous-generation platforms, such as poor pharmacokinetics and limited cellular uptake, thereby enhancing the efficiency of nucleic acid drug delivery across diverse tissues and cell types. The company aims to establish RNA therapeutics as a leading option for personalized treatment in multiple therapeutic areas.

Shoreline Biosciences, Inc. is a biotechnology company based in La Jolla, California, founded in 2020. The company specializes in developing cell-based immunotherapies aimed at treating seriously ill patients. Shoreline's proprietary technology platform leverages advanced methods in induced pluripotent stem cell (iPSC) differentiation and genetic programming, specifically targeting the IL-15/CISH pathway. This approach enhances the metabolic fitness and persistence of engineered natural killer (NK) cells, improving their anti-cancer activity. Shoreline's focus includes creating clinically effective immunotherapies using iPSC-derived NK cells and macrophages, providing innovative and cost-effective treatment options for oncology and other serious health 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 biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

Founded in 2020 and headquartered in Boston, Massachusetts, Scorpion Therapeutics is a clinical-stage biotechnology company specializing in precision oncology. It develops targeted small-molecule drugs to treat cancer by focusing on three areas: therapies against known oncogenes, agents for currently undruggable targets, and drugs for new targets.

Biomea Fusion is a precision medicine company focused on accelerating cancer drug development by targeting specific gene alterations that drive tumor growth. Its lead program aims to disrupt the protein-to-protein interaction between menin and the MLL complex for treating various tumors.

Talaris Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies aimed at eliminating chronic immunosuppression for organ transplant recipients. The company's leading product, FCR001, is designed to enable living donor kidney transplant recipients to discontinue all chronic immunosuppression therapy within twelve months post-transplant. In addition to its applications in organ transplantation, Talaris’s technology facilitates the safe use of hematopoietic stem cells from unmatched and unrelated donors for bone marrow transplants, reducing the associated toxicity and risks of traditional methods. Founded in 1988 and originally named Regenerex, Inc., Talaris Therapeutics rebranded in March 2019. The company is headquartered in Louisville, Kentucky, with an additional office in Wellesley, Massachusetts.

Rain Oncology Inc. is a late-stage precision oncology company focused on developing targeted therapies aimed at oncogenic drivers. The company employs a genetic selection strategy to identify patients who are most likely to benefit from its treatments, utilizing a tumor-agnostic approach that prioritizes the underlying genetics of tumors over traditional histological classifications. Its lead product candidate, milademetan, is a small molecule oral inhibitor of MDM2, which plays a role in various cancers. Additionally, Rain Oncology is advancing a preclinical program aimed at inducing synthetic lethality in cancer cells by inhibiting RAD52, further diversifying its therapeutic pipeline.

Kinnate Biopharma Inc. is a biopharmaceutical company dedicated to the discovery and development of small molecule kinase inhibitors aimed at treating genomically defined cancers. Established in 2018 and headquartered in San Diego, California, the company focuses on precision oncology therapeutics, leveraging its expertise in structure-based drug discovery and translational research. Kinnate's lead product candidate, KIN002787, is a rapidly accelerated fibrosarcoma inhibitor currently in preclinical development for lung cancer, melanoma, and other solid tumors. Additionally, the company is advancing other preclinical candidates, including KIN003, which targets cancer-associated alterations in FGFR2 and FGFR3 genes, and a Cyclin-Dependent Kinase 12 inhibitor in the KIN004 program. Kinnate Biopharma is committed to developing targeted therapeutics for underserved patient populations, aiming to expand the reach of precision medicine in cancer treatment.

Dyne Therapeutics is a clinical-stage biotechnology company developing therapies for genetically driven neuromuscular diseases. It aims to deliver disease-modifying therapeutics to skeletal, cardiac, and smooth muscle, addressing conditions such as muscular dystrophies and other muscle disorders. The company uses the FORCE platform to create targeted therapeutics with payloads designed to address the disease gene, connected via a linker to an antigen-binding fragment, enabling delivery to muscle tissue and the central nervous system. Its pipeline includes DYNE-101, DYNE-251, DYNE-302, and DYNE-401. Headquartered in Waltham, 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).

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

Repare Therapeutics is a precision oncology company that discovers and develops novel therapeutics using its synthetic lethality approach. It employs its proprietary SNIPRx platform to systematically identify and target genomic instability, particularly DNA damage repair pathways in cancer cells. The company's lead product candidate, RP-3500, is an oral small molecule inhibitor for treating solid tumors with specific DNA damage repair-related alterations.

Passage Bio is a gene therapy company dedicated to developing transformative treatments for rare, monogenic central nervous system disorders. It has an integrated pipeline of five AAV-delivered therapeutics and collaborates with the University of Pennsylvania's Gene Therapy Program for preclinical work.