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Garuda Therapeutics develops off-the-shelf hematopoietic stem cell therapies to treat life-threatening diseases. It seeks to eliminate dependency on donor or patient cells for blood stem cell transplants, leveraging a platform technology that generates off-the-shelf, self-renewing blood stem cells that are HLA-compatible and transgene-free, providing patients with rapid and broad access to durable therapies.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

GondolaBio is a biopharmaceutical company dedicated to researching, developing, and manufacturing pharmaceutical products. Its primary focus is on creating therapeutics for patients with genetic diseases, addressing high unmet medical needs across various therapeutic areas and stages of development. The company leverages biological research to drive its mission, aiming to enable the healthcare industry to effectively treat and manage rare genetic conditions.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

Fore Biotherapeutics is a precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.

Garuda Therapeutics develops off-the-shelf hematopoietic stem cell therapies to treat life-threatening diseases. It seeks to eliminate dependency on donor or patient cells for blood stem cell transplants, leveraging a platform technology that generates off-the-shelf, self-renewing blood stem cells that are HLA-compatible and transgene-free, providing patients with rapid and broad access to durable therapies.

Ensoma is a biotechnology company focused on advancing genomic medicine through its innovative in vivo approach, utilizing proprietary Engenious vectors. These vectors are engineered to deliver a wide array of gene modification technologies directly to blood and immune cells, including T cells, B cells, and myeloid cells, without the necessity for stem cell collection or prior myeloablative conditioning, which can pose risks to patients. This unique method allows for the administration of therapies via a single injection, enhancing accessibility and convenience in various healthcare settings, even where resources may be limited. Ensoma aims to expand the curative potential of genomic medicine, making it more accessible to patients in need.

Orionis Biosciences is a biotechnology company focused on early-stage drug discovery and development. It specializes in innovative medicines for diseases with high unmet medical needs, initially concentrating on oncology and immunotherapies. The company operates research facilities in Waltham, MA, USA, and Ghent, Belgium, led by an experienced team of entrepreneurs and scientists.

Tessera Therapeutics is an early-stage life sciences company focused on pioneering Gene Writing technology, which allows for the precise insertion of therapeutic messages into the human genome. This innovative approach aims to address diseases at their source by enabling both small and large genetic alterations. By building on recent advancements in gene therapy and gene editing, Tessera Therapeutics seeks to overcome existing limitations in these fields, enhancing their efficacy and reach. Founded by Flagship Pioneering, the company aspires to establish a new category in genetic medicine that can significantly improve patient outcomes and transform healthcare.

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.

Garuda Therapeutics develops off-the-shelf hematopoietic stem cell therapies to treat life-threatening diseases. It seeks to eliminate dependency on donor or patient cells for blood stem cell transplants, leveraging a platform technology that generates off-the-shelf, self-renewing blood stem cells that are HLA-compatible and transgene-free, providing patients with rapid and broad access to durable therapies.

Prime Medicine is a biotechnology company focused on developing genetic therapies using its innovative Prime Editing technology. This next-generation gene editing method allows for precise "search and replace" modifications in the genome, aimed at restoring normal genetic function. The company targets a wide range of diseases with significant unmet medical needs, leveraging the efficiency and versatility of its gene editing capabilities to address the underlying causes of these conditions. Through its commitment to advancing gene editing, Prime Medicine seeks to transform the landscape of genetic medicine.

Monte Rosa Therapeutics is a biotechnology company focused on cancer therapeutics that target protein degradation pathways. It specializes in developing molecular glue degraders, a class of small molecules that direct disease-relevant proteins to degradation by the cell's natural machinery. The company uses its QuEEN platform, incorporating artificial intelligence and proprietary experimental tools, to identify target proteins for degradation by molecular glue degraders. It maintains a diverse library of more than 50,000 molecules and advancing its pipeline, including MRT-2359, a candidate that targets the translation termination factor GSPT1 for potential use in MYC-driven tumors.

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.

Ensoma is a biotechnology company focused on advancing genomic medicine through its innovative in vivo approach, utilizing proprietary Engenious vectors. These vectors are engineered to deliver a wide array of gene modification technologies directly to blood and immune cells, including T cells, B cells, and myeloid cells, without the necessity for stem cell collection or prior myeloablative conditioning, which can pose risks to patients. This unique method allows for the administration of therapies via a single injection, enhancing accessibility and convenience in various healthcare settings, even where resources may be limited. Ensoma aims to expand the curative potential of genomic medicine, making it more accessible to patients in need.

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.

Beam Therapeutics is a biotechnology company developing precision genetic medicines using its proprietary base editing technology. Its focus includes therapies for hematological and genetic diseases, with ongoing programs targeting sickle cell disease, alpha-1 antitrypsin deficiency, and other serious conditions.

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.

Immuneering Corporation is a biotechnology company based in Cambridge, Massachusetts, with an additional office in New York, New York. Founded in 2008, it specializes in genetic, genomic, and proteomic data analysis for pharmaceutical clients, aiming to enhance the clinical and commercial success of medicines. The company utilizes advanced techniques such as gene expression analysis, proteomics, and next-generation sequencing to uncover biological insights that inform drug development. Immuneering's expertise in translational bioinformatics is applied throughout the drug development process, particularly for oncologic and neurologic diseases, and is integral to its proprietary computational platform, known as Disease Cancelling Technology. This platform supports drug discovery initiatives and provides computational biology services to both pharmaceutical and biotechnology companies, contributing to the creation of therapies for serious illnesses, including cancer and autoimmune diseases.

Neurogene is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. It leverages its proprietary transgene regulation platform, EXACT, to create gene therapies with the aim of addressing high-need conditions. The company’s pipeline includes NGN-401 for Rett syndrome and NGN-101 for CLN5 Batten disease. Founded in 2018, Neurogene is headquartered in New York and concentrates on advancing therapies that can improve outcomes for patients with limited treatment options.

Monte Rosa Therapeutics is a biotechnology company focused on cancer therapeutics that target protein degradation pathways. It specializes in developing molecular glue degraders, a class of small molecules that direct disease-relevant proteins to degradation by the cell's natural machinery. The company uses its QuEEN platform, incorporating artificial intelligence and proprietary experimental tools, to identify target proteins for degradation by molecular glue degraders. It maintains a diverse library of more than 50,000 molecules and advancing its pipeline, including MRT-2359, a candidate that targets the translation termination factor GSPT1 for potential use in MYC-driven tumors.

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 precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

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.

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.

Aligos Therapeutics is a biopharmaceutical company dedicated to developing innovative therapeutics targeting viral infections and liver diseases. Founded in 2018, the company focuses on chronic hepatitis B (CHB) and non-alcoholic steatohepatitis (NASH), leveraging its expertise in liver disease drug development.

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.

Beam Therapeutics is a biotechnology company developing precision genetic medicines using its proprietary base editing technology. Its focus includes therapies for hematological and genetic diseases, with ongoing programs targeting sickle cell disease, alpha-1 antitrypsin deficiency, and other serious conditions.

Neurogene is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. It leverages its proprietary transgene regulation platform, EXACT, to create gene therapies with the aim of addressing high-need conditions. The company’s pipeline includes NGN-401 for Rett syndrome and NGN-101 for CLN5 Batten disease. Founded in 2018, Neurogene is headquartered in New York and concentrates on advancing therapies that can improve outcomes for patients with limited treatment options.

Stoke Therapeutics is a biopharmaceutical company developing novel antisense oligonucleotide medicines. These target RNA splicing to increase gene expression, aiming to treat severe genetic diseases. Its lead product candidate, STK-001, focuses on Dravet syndrome, a progressive genetic epilepsy.

Cibus is a biotechnology company specializing in precision gene editing for agriculture, aimed at making farming more sustainable and profitable. The company has developed patented breeding technologies that facilitate precise and predictable modifications in plants without the integration of foreign genetic material. Its core innovation, the Rapid Trait Development System, combines crop-specific cell biology platforms with advanced gene editing techniques. Cibus focuses on creating plant traits that address specific agricultural challenges, including those related to productivity, disease resistance, insect control, weed management, nutrient use, and climate adaptability. Through these efforts, Cibus seeks to enhance the efficiency and sustainability of agricultural practices.

Avrobio is a clinical-stage biotechnology company focused on developing ex vivo lentiviral-based gene therapies to treat rare diseases with a single dose. Its pipeline includes AVR-RD-01 for Fabry disease, AVR-RD-02 for type 1 Gaucher disease, and AVR-RD-03 for Pompe disease.

Dicerna Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development of RNA interference (RNAi)-based therapeutics. The company focuses on innovative treatments for rare inherited liver diseases, viral infections, chronic liver diseases, and cardiometabolic conditions. Utilizing its proprietary GalXC RNAi technology platform, Dicerna is advancing several key product candidates, including nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B, and programs targeting genetic liver diseases and cardiovascular conditions. Founded in 2007 and headquartered in Lexington, Massachusetts, Dicerna has established strategic collaborations with major pharmaceutical firms to enhance its research and development efforts.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a 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. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Proteostasis Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule therapies that regulate the body's protein homeostasis network to treat cystic fibrosis and other genetic or degenerative diseases. It develops Proteostasis Regulators intended to restore proteostasis balance and address conditions such as cystic fibrosis, emphysema, diabetes, and neurodegenerative disorders. In CF, its lead candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. The company collaborates with the Cystic Fibrosis Foundation to research, develop, and commercialize CF-related products, and has licensing relationships with Genentech. It was founded in 2006, formerly known as Proteoguard, and was renamed Proteostasis Therapeutics in 2007, based in Boston. It was acquired by Yumanity Therapeutics in 2020 in a reverse merger.

Wave Life Sciences is a clinical-stage biotechnology company focused on RNA medicines, designing, optimizing, and producing stereopure oligonucleotides through its PRISM platform. The company aims to address genetically defined diseases by reducing harmful protein expression or correcting dysfunctional proteins, employing modalities such as RNA editing, antisense silencing, and RNA interference. Its programs span neurological and neuromuscular conditions, reflecting an emphasis on central nervous system targets. Wave collaborates with Pfizer and Takeda on research, development, and commercialization of stereopure oligonucleotide therapeutics and antisense therapies. Founded in 2012 and based in Singapore, the company seeks to unlock the broader potential of RNA medicines to improve patient outcomes.

Fore Biotherapeutics is a precision oncology company focused on developing targeted cancer therapies guided by functional genomics. It aims to create a pipeline of treatments for patients with currently unaddressed mutations across established oncology targets.