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Garuda Therapeutics is a biotechnology company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening blood, bone marrow, immune, and metabolic diseases. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants. Utilizing advanced technologies, including zebrafish, mice, and human-induced pluripotent stem cells, Garuda Therapeutics conducts research to clone and analyze novel genes and mechanisms critical for blood formation. This innovative approach is designed to provide patients with consistent, durable, and HLA-compatible transgene-free blood stem cell therapies, offering rapid and broad access to life-saving treatments.

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 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.

Garuda Therapeutics is a biotechnology company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening blood, bone marrow, immune, and metabolic diseases. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants. Utilizing advanced technologies, including zebrafish, mice, and human-induced pluripotent stem cells, Garuda Therapeutics conducts research to clone and analyze novel genes and mechanisms critical for blood formation. This innovative approach is designed to provide patients with consistent, durable, and HLA-compatible transgene-free blood stem cell therapies, offering rapid and broad access to life-saving treatments.

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 an early-stage biotechnology company dedicated to drug discovery and development, primarily targeting oncology and immunotherapies for diseases with significant unmet medical needs. Based in Waltham, Massachusetts, with additional research facilities in Ghent, Belgium, the company utilizes innovative technologies to create conditionally active drug modalities. Orionis employs mechanisms of molecular proximity and cooperativity to enhance drug potency and precision, leading to a diverse pipeline of drug candidates. These efforts include the development of small molecules that engage both adaptive and innate immune systems, paving the way for effective single-agent therapies for cancer and other life-threatening conditions. The company is supported by a team of experienced professionals and maintains strategic collaborations with leading research institutions, such as VIB in Belgium.

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 is a biotechnology company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening blood, bone marrow, immune, and metabolic diseases. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants. Utilizing advanced technologies, including zebrafish, mice, and human-induced pluripotent stem cells, Garuda Therapeutics conducts research to clone and analyze novel genes and mechanisms critical for blood formation. This innovative approach is designed to provide patients with consistent, durable, and HLA-compatible transgene-free blood stem cell therapies, offering rapid and broad access to life-saving treatments.

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 dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

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 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.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, specializing in the development of precision genetic medicines utilizing its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients suffering from serious diseases by targeting specific bases in the genome without inducing double-stranded breaks in DNA. Beam Therapeutics is actively developing therapies for a range of conditions, including sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, glycogen storage disorder type 1A, as well as ocular and central nervous system disorders. The company’s diverse pipeline includes several programs such as BEAM-101, BEAM-201, BEAM-301, BEAM-302, and ESCAPE, reflecting its commitment to advancing gene correction, gene modification, gene activation, gene silencing, and multiplex editing technologies.

Biomea Fusion is a privately held biopharmaceutical company focused on developing precision medicines for cancer patients. The company targets specific gene alterations that drive tumor growth, aiming to create more effective therapies. Its lead program focuses on inhibiting the interaction between menin and the MLL complex, which is crucial in various tumors. Biomea Fusion's goal is to expedite the development process of these potentially groundbreaking medicines to deliver impactful treatments for patients with genetically defined cancers.

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 Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

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.

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, 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.

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.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, specializing in the development of precision genetic medicines utilizing its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients suffering from serious diseases by targeting specific bases in the genome without inducing double-stranded breaks in DNA. Beam Therapeutics is actively developing therapies for a range of conditions, including sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, glycogen storage disorder type 1A, as well as ocular and central nervous system disorders. The company’s diverse pipeline includes several programs such as BEAM-101, BEAM-201, BEAM-301, BEAM-302, and ESCAPE, reflecting its commitment to advancing gene correction, gene modification, gene activation, gene silencing, and multiplex editing technologies.

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company focused on developing novel antisense oligonucleotide medicines aimed at addressing the underlying causes of severe genetic diseases. Founded in 2014 and headquartered in Bedford, Massachusetts, the company utilizes its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to enhance gene expression through RNA splicing. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke Therapeutics has also partnered with Invitae Corporation to offer complementary genetic testing services.

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, Inc. is a clinical-stage gene therapy company based in Cambridge, Massachusetts, focused on developing ex vivo lentiviral-based therapies aimed at treating rare diseases with a single-dose regimen. The company utilizes hematopoietic stem cells collected from patients, which are modified using a lentiviral vector to introduce functional copies of defective genes associated with specific diseases. AVROBIO's lead product candidate, AVR-RD-01, is currently undergoing Phase II clinical trials for Fabry disease, while AVR-RD-02 and AVR-RD-04 have completed pre-clinical trials for type 1 Gaucher disease and cystinosis, respectively. Additionally, AVR-RD-03 is in preclinical development for Pompe disease. The company has also established a research collaboration with Magenta Therapeutics to explore targeted antibody-drug conjugates as conditioning regimens for its lentiviral gene therapies. Founded in 2015, AVROBIO aims to accelerate advancements in cell and gene therapies to address serious and debilitating conditions.

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, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics aimed at treating cystic fibrosis (CF) and other genetic and degenerative disorders. The company specializes in discovering small molecule therapeutics that regulate the Proteostasis Network, which is essential for maintaining the balance of proteins in the body. Its lead product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis Therapeutics employs a theratyping approach to tailor treatments based on individual responses, regardless of specific CFTR mutations. The company has established collaboration agreements with organizations such as the Cystic Fibrosis Foundation to advance research and commercialization efforts for CF and other pulmonary diseases. Founded in 2006 and headquartered in Boston, Massachusetts, Proteostasis Therapeutics was previously known as Proteoguard, Inc. before rebranding in 2007.

WaVe Life Sciences Ltd. is a clinical-stage genetic medicine company based in Singapore, focused on developing innovative treatments for genetically defined diseases using its proprietary PRISM platform. This platform enables the precise design, optimization, and production of stereopure oligonucleotides, which are designed to target genetic defects by either reducing harmful protein expression or converting dysfunctional proteins into functional ones. The company's primary focus is on neurology, particularly within the central and neuromuscular systems. WaVe Life Sciences collaborates with major pharmaceutical companies, including Pfizer and Takeda, to advance the research and development of its oligonucleotide therapeutics. Founded in 2012, the company aims to deliver transformative therapies to improve patient outcomes in both rare and prevalent disorders.

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.