Atlas Venture

Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.

Trace Neuroscience is a biopharmaceutical company dedicated to advancing genetic medicine for patients with neurodegenerative diseases. It specializes in developing targeted antisense oligonucleotide therapies, with its lead product aimed at restoring UNC13A protein function in individuals with Amyotrophic Lateral Sclerosis (ALS) caused by TDP-43 mislocalization. This therapy corrects UNC13A mRNA splicing, potentially improving muscle function and extending survival for these patients.

Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.

Judo Bio is a biotechnology company focused on developing precision therapeutics that target specific cell populations to treat genetic diseases. Utilizing an innovative receptor targeting approach, Judo Bio is building a pipeline of therapies designed to benefit patients suffering from both common and rare conditions. Currently, the company is operating in stealth mode, indicating a focus on research and development without public visibility.

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.

GRO Biosciences is focused on developing advanced protein therapeutics by utilizing innovative technologies in computational protein design and synthetic biology. The company has created several genomically recoded strains of bacteria that enable the incorporation of non-standard amino acids into proteins. This approach enhances the potency, stability, and targeted delivery of these therapeutics. GRO Biosciences aims to apply its technology to improve treatments for a range of conditions, including diabetes, growth disorders, and autoimmunity, thereby addressing significant unmet medical needs.

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

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.

Judo Bio is a biotechnology company focused on developing precision therapeutics that target specific cell populations to treat genetic diseases. Utilizing an innovative receptor targeting approach, Judo Bio is building a pipeline of therapies designed to benefit patients suffering from both common and rare conditions. Currently, the company is operating in stealth mode, indicating a focus on research and development without public visibility.

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.

Aiolos Bio is a biotechnology company specializing in the development of therapies for respiratory diseases and immune conditions. Its primary focus is on advancing AIO-001, a monoclonal antibody therapy for moderate-to-severe asthma, designed for dosing every 6 months. The company, founded by industry veterans, aims to address unmet treatment needs and improve patient outcomes by providing more efficacious and less burdensome therapies.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

K36 Therapeutics is engaged in the development of small molecule therapeutics aimed at treating cancer. The company focuses on translating epigenetic modulation of oncogenic pathways into effective therapies, utilizing technology to create orally bioavailable small molecules and selective inhibitors. This innovative approach enables healthcare professionals to enhance treatment options for cancer patients, addressing the complexities of oncogenic pathways and improving therapeutic outcomes.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

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

Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.

Triana Biomedicines is focused on discovering and developing molecular glues, a novel therapeutic approach aimed at regulating challenging disease targets. The company employs a sophisticated drug discovery engine that combines high-resolution structural insights, advanced in silico tools, and custom chemical libraries. This platform is designed to stabilize existing protein interactions or create new ones, thereby influencing the functionality of disease targets. Triana has successfully validated its technology and is currently advancing multiple research programs across various disease areas, positioning itself to contribute significantly to therapeutic innovation.

Arkuda Therapeutics is a biotechnology company based in Cambridge, Massachusetts, founded in 2018. The company focuses on developing innovative medicines for patients suffering from neurological diseases, particularly targeting neurodegenerative conditions. Arkuda leverages insights into progranulin and lysosomal biology to create therapies aimed at addressing progranulin deficiency and lysosomal dysfunction, specifically in genetically-defined frontotemporal dementia related to mutations in the GRN gene. Its lead compounds are designed to improve cellular health in the brain, with the goal of delaying disease progression and the onset of symptoms.

K36 Therapeutics is engaged in the development of small molecule therapeutics aimed at treating cancer. The company focuses on translating epigenetic modulation of oncogenic pathways into effective therapies, utilizing technology to create orally bioavailable small molecules and selective inhibitors. This innovative approach enables healthcare professionals to enhance treatment options for cancer patients, addressing the complexities of oncogenic pathways and improving therapeutic outcomes.

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

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

Vedere Bio, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on developing gene therapy products aimed at restoring functional vision for patients who have experienced vision loss due to Inherited Retinal Degenerations (IRDs) and other causes of vision impairment, both genetic and non-genetic. Unlike many ocular gene therapies that primarily slow the progression of vision loss, Vedere Bio seeks to restore lost vision, regardless of the patient's genetic background or stage of their condition. As of September 2020, Vedere Bio operates as a subsidiary of Novartis AG.

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.

Ikena Oncology, Inc. is a biotechnology company focused on discovering and developing biomarker-driven therapies for cancer treatment. It specializes in precision oncology by targeting pathways essential for cancer growth and therapeutic resistance, particularly within the Hippo and RAS signaling networks. The company's portfolio includes several preclinical and discovery-stage programs, such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-412, a kynurenine-degrading enzyme. Additionally, Ikena is developing IK-930, an oral small-molecule inhibitor of the TEAD transcription factor involved in the Hippo pathway. Ikena Oncology, originally founded as Kyn Therapeutics in 2016, rebranded in December 2019 and is headquartered in Boston, Massachusetts. The company aims to address significant medical needs in oncology through innovative drug development.

Scorpion Therapeutics, Inc. is a biotechnology company based in Boston, Massachusetts, focused on developing next-generation precision oncology technologies for cancer treatment. Founded in 2020, the company specializes in creating targeted small-molecule drugs that interact with validated cancer targets. Its research encompasses therapies aimed at known oncogenes, previously undruggable cancer targets, and novel targets. By integrating technologies across target discovery, medicinal chemistry, and translational medicine, Scorpion Therapeutics aims to advance a comprehensive pipeline of optimized compounds. The company's goal is to expand the reach of precision medicine, enabling healthcare professionals to offer innovative treatments to a broader range of cancer patients.

Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.

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.

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).

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

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.

IFM Therapeutics specializes in developing small molecule medicines that target the innate immune system to address complex challenges in treating inflammatory and autoimmune disorders, as well as cancer. The company collaborates with academic partners to leverage expertise in innate immunity alongside experienced drug discovery professionals. Its subsidiary, IFM Due, focuses on discovering and developing antagonists of the cGAS/STING pathway, aiming to treat conditions such as inflammation, neuroinflammation, autoimmunity, and cancer. Additionally, IFM Discovery, a newly established incubator within the company, is engaged in advancing a portfolio of genetically validated targets for next-generation therapies addressing inflammation, neuroinflammation, autoimmunity, and fibrosis. Through these initiatives, IFM Therapeutics seeks to enhance immune response and improve the quality of life for patients facing serious health challenges.

Arkuda Therapeutics is a biotechnology company based in Cambridge, Massachusetts, founded in 2018. The company focuses on developing innovative medicines for patients suffering from neurological diseases, particularly targeting neurodegenerative conditions. Arkuda leverages insights into progranulin and lysosomal biology to create therapies aimed at addressing progranulin deficiency and lysosomal dysfunction, specifically in genetically-defined frontotemporal dementia related to mutations in the GRN gene. Its lead compounds are designed to improve cellular health in the brain, with the goal of delaying disease progression and the onset of symptoms.

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

Vedere Bio, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on developing gene therapy products aimed at restoring functional vision for patients who have experienced vision loss due to Inherited Retinal Degenerations (IRDs) and other causes of vision impairment, both genetic and non-genetic. Unlike many ocular gene therapies that primarily slow the progression of vision loss, Vedere Bio seeks to restore lost vision, regardless of the patient's genetic background or stage of their condition. As of September 2020, Vedere Bio operates as a subsidiary of Novartis AG.

Akero Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, that specializes in developing treatments for serious metabolic diseases, particularly nonalcoholic steatohepatitis (NASH). Founded in 2017 and formerly known as Pippin Pharmaceuticals, the company is dedicated to addressing the significant unmet medical needs associated with NASH, a condition characterized by liver inflammation and damage, which can lead to severe complications such as fibrosis and liver failure. Akero's lead product candidate, AKR-001, is a long-acting Fc fusion modified FGF21 protein designed to restore metabolic balance by targeting both liver and adipose tissues, thereby reducing liver fat and suppressing inflammation and fibrosis. This innovative approach builds upon two decades of research into FGF21 biology, positioning Akero as a frontrunner in the quest for effective NASH therapies.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

Akero Therapeutics is a clinical-stage biotechnology company based in South San Francisco, California, that specializes in developing treatments for serious metabolic diseases, particularly nonalcoholic steatohepatitis (NASH). Founded in 2017 and formerly known as Pippin Pharmaceuticals, the company is dedicated to addressing the significant unmet medical needs associated with NASH, a condition characterized by liver inflammation and damage, which can lead to severe complications such as fibrosis and liver failure. Akero's lead product candidate, AKR-001, is a long-acting Fc fusion modified FGF21 protein designed to restore metabolic balance by targeting both liver and adipose tissues, thereby reducing liver fat and suppressing inflammation and fibrosis. This innovative approach builds upon two decades of research into FGF21 biology, positioning Akero as a frontrunner in the quest for effective NASH therapies.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

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

Harbour BioMed is a biotechnology company focused on discovering and developing therapeutics for cancer and inflammatory diseases. Established in 2016 with operations in Shanghai, Cambridge, and Rotterdam, the company leverages its proprietary transgenic mouse platforms to generate fully human monoclonal antibodies (H2L2) and heavy chain only antibodies (HCAb), enabling the development of differentiated compounds such as bi-specific and multi-specific antibodies. Harbour BioMed collaborates strategically with various institutions and companies, including The Wistar Institute, WuXi Biologics, and Vir Biotechnology, to advance its pipeline of therapeutics targeting autoimmune diseases, dry eye disease, COVID-19, and other indications.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

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.

Ikena Oncology, Inc. is a biotechnology company focused on discovering and developing biomarker-driven therapies for cancer treatment. It specializes in precision oncology by targeting pathways essential for cancer growth and therapeutic resistance, particularly within the Hippo and RAS signaling networks. The company's portfolio includes several preclinical and discovery-stage programs, such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-412, a kynurenine-degrading enzyme. Additionally, Ikena is developing IK-930, an oral small-molecule inhibitor of the TEAD transcription factor involved in the Hippo pathway. Ikena Oncology, originally founded as Kyn Therapeutics in 2016, rebranded in December 2019 and is headquartered in Boston, Massachusetts. The company aims to address significant medical needs in oncology through innovative drug development.

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.

Disarm Therapeutics, Inc. is a biotechnology company focused on developing therapeutics for patients suffering from neurological diseases. Founded in 2016 and headquartered in Cambridge, Massachusetts, the company aims to create disease-modifying drugs that prevent axonal degeneration, which is a key factor in various neurological disorders. Disarm's portfolio addresses conditions such as multiple sclerosis, amyotrophic lateral sclerosis, glaucoma, and peripheral neuropathies, providing potential treatments for acute diseases affecting the central, ocular, and peripheral nervous systems. As of late 2020, Disarm Therapeutics operates as a subsidiary of Eli Lilly and Company.

Replimune Group, Inc., established in 2015 and based in Woburn, Massachusetts, is a biotechnology company specializing in the development of oncolytic immune-gene therapies for cancer treatment. The company's proprietary Immulytic platform enables the design of product candidates that directly attack cancer cells and stimulate the immune system. Replimune's lead candidate, RP1, is a modified herpes simplex virus currently in Phase I/II trials for various solid tumors and Phase II trials for cutaneous squamous cell carcinoma. Additionally, RP2 and RP3 are in early-stage clinical trials, targeting immune checkpoint inhibition and T-cell stimulation, respectively. Replimune aims to rapidly advance these therapies through clinical trials and explore combinations with other immuno-oncology products.

Indalo Therapeutics is a preclinical-stage biotechnology company focused on developing innovative therapies for patients afflicted by fibrotic diseases. Founded on significant scientific advancements, the company leverages deep insights into the molecular mechanisms that drive abnormal scarring. By assembling a skilled team of pharmaceutical scientists and industry executives, Indalo aims to address critical unmet medical needs in the field of fibrosis. Its therapeutic approach seeks to not only halt but also potentially reverse the progression of fibrotic conditions, ultimately improving the quality of life for affected individuals.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

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

IFM Therapeutics specializes in developing small molecule medicines that target the innate immune system to address complex challenges in treating inflammatory and autoimmune disorders, as well as cancer. The company collaborates with academic partners to leverage expertise in innate immunity alongside experienced drug discovery professionals. Its subsidiary, IFM Due, focuses on discovering and developing antagonists of the cGAS/STING pathway, aiming to treat conditions such as inflammation, neuroinflammation, autoimmunity, and cancer. Additionally, IFM Discovery, a newly established incubator within the company, is engaged in advancing a portfolio of genetically validated targets for next-generation therapies addressing inflammation, neuroinflammation, autoimmunity, and fibrosis. Through these initiatives, IFM Therapeutics seeks to enhance immune response and improve the quality of life for patients facing serious health challenges.

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.

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

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

Navitor Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing small molecule drugs that target mTORC1, a key regulator of cellular metabolism and renewal, to treat age-related diseases. Utilizing a proprietary platform, Navitor's therapies can selectively inhibit or activate mTORC1, which plays a crucial role in cellular processes. One of its notable products, NV-5138, is an orally bioavailable small molecule designed to activate mTORC1, particularly addressing its suppression in the brain associated with depression. Additionally, the company's N-Valog program focuses on selective inhibition of mTORC1 for chronic kidney disease, with implications for other age-related conditions. Founded in 2009 and headquartered in Cambridge, Massachusetts, Navitor Pharmaceuticals aims to unlock the therapeutic potential of the mTOR pathway by modulating nutrient-sensing proteins, thereby addressing various metabolic, neurodegenerative, autoimmune, and musculoskeletal diseases.

Replimune Group, Inc., established in 2015 and based in Woburn, Massachusetts, is a biotechnology company specializing in the development of oncolytic immune-gene therapies for cancer treatment. The company's proprietary Immulytic platform enables the design of product candidates that directly attack cancer cells and stimulate the immune system. Replimune's lead candidate, RP1, is a modified herpes simplex virus currently in Phase I/II trials for various solid tumors and Phase II trials for cutaneous squamous cell carcinoma. Additionally, RP2 and RP3 are in early-stage clinical trials, targeting immune checkpoint inhibition and T-cell stimulation, respectively. Replimune aims to rapidly advance these therapies through clinical trials and explore combinations with other immuno-oncology products.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

Lysosomal Therapeutics, Inc. is a biotechnology company focused on discovering and developing small-molecule therapies for severe neurological diseases. Founded in 2011 and based in Cambridge, Massachusetts, the company targets disorders such as Gaucher-related neurodegeneration, Parkinson’s disease, and other synucleinopathies. Lysosomal Therapeutics employs a unique molecular platform informed by the clinically validated connection between lysosomal disorders and neurodegeneration. This approach facilitates the development of innovative treatment options aimed at improving the health and quality of life for patients affected by these debilitating conditions.

Padlock Therapeutics is a biotechnology company focused on developing innovative medicines that target protein-arginine deiminases (PADs), enzymes implicated in the generation of autoantigens and the inflammatory processes associated with autoimmune diseases. Founded by scientists from The Scripps Research Institute and members of the Atlas Venture Life Sciences Team, Padlock aims to explore the biological and therapeutic potential of protein deimination by PADs. The company operates as a seed-stage entity based in Cambridge, Massachusetts, and is backed by notable investors, including Atlas Venture, Johnson & Johnson, and MS Ventures.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

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).

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

Navitor Pharmaceuticals, Inc. is a biopharmaceutical company focused on developing small molecule drugs that target mTORC1, a key regulator of cellular metabolism and renewal, to treat age-related diseases. Utilizing a proprietary platform, Navitor's therapies can selectively inhibit or activate mTORC1, which plays a crucial role in cellular processes. One of its notable products, NV-5138, is an orally bioavailable small molecule designed to activate mTORC1, particularly addressing its suppression in the brain associated with depression. Additionally, the company's N-Valog program focuses on selective inhibition of mTORC1 for chronic kidney disease, with implications for other age-related conditions. Founded in 2009 and headquartered in Cambridge, Massachusetts, Navitor Pharmaceuticals aims to unlock the therapeutic potential of the mTOR pathway by modulating nutrient-sensing proteins, thereby addressing various metabolic, neurodegenerative, autoimmune, and musculoskeletal diseases.

Numerate Inc., established in 2007 and headquartered in San Bruno, California, is a biotechnology company specializing in small molecule therapeutics development. It offers a drug design platform that integrates data science, machine learning, cloud-scale analytics, and medicinal chemistry to predict how potential drugs will behave in lab and body settings. This platform enables Numerate to explore vast chemical spaces efficiently, accelerating drug discovery for diseases like obesity, heart failure, Alzheimer's, and Huntington's disease. By combining computational power with traditional medicinal chemistry, Numerate aims to make the drug development process more cost-effective, faster, and successful than conventional methods.

Lysosomal Therapeutics, Inc. is a biotechnology company focused on discovering and developing small-molecule therapies for severe neurological diseases. Founded in 2011 and based in Cambridge, Massachusetts, the company targets disorders such as Gaucher-related neurodegeneration, Parkinson’s disease, and other synucleinopathies. Lysosomal Therapeutics employs a unique molecular platform informed by the clinically validated connection between lysosomal disorders and neurodegeneration. This approach facilitates the development of innovative treatment options aimed at improving the health and quality of life for patients affected by these debilitating conditions.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

Ataxion is a discovery-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapies for rare and underserved neurologic diseases. Founded in April 2013 by Dr. Josh Resnick and David Grayzel, MD, through the Atlas Venture seed program, Ataxion specializes in small-molecule therapeutics that target hereditary ataxia and other degenerative neurological disorders such as multiple sclerosis and Huntington's disease. By leveraging ion-channel modulators, the company aims to provide effective treatments for patients suffering from these debilitating conditions.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

Harbour Antibodies BV, established in 2006 and based in Rotterdam, Netherlands, specializes in the production of antibodies and VH domains for therapeutic applications. The company utilizes advanced transgenic mouse technology to engineer mice capable of generating high affinity human antibodies. It has developed two key types of human immunoglobulin gene transgenic mice: those that produce conventional tetrameric antibodies, known as H2L2 antibodies, which consist of two heavy and two light chains, and those that generate heavy chain-only antibodies (HCAbs), which lack light chains. The availability of HCAbs allows for the generation of soluble human VH domains, facilitating the creation of innovative multi-functional molecules. Harbour Antibodies' transgenic mouse lines are accessible to licensing partners, enhancing research and development in the field of antibody therapeutics. The company operates as a subsidiary of Harbour BioMed.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

Avila Therapeutics is focused on designing and developing covalent drugs aimed at treating viral infections, cancers, and autoimmune diseases. One of its key products is AVL-181, a small molecule that acts as a protease inhibitor for hepatitis C virus. Founded in 2006 and based in Waltham, Massachusetts, Avila Therapeutics operates as a subsidiary of Celgene Corporation, following its acquisition in 2012. The company has also established a strategic alliance with Sanofi-Aventis to enhance its research and development efforts.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

Aureon Laboratories is a life sciences company dedicated to enabling the advancement of predictive and personalized cancer treatment. Aureon provides objective, actionable information allowing patients and physicians to make more-informed treatment decisions through its prostate diagnostic tools.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

Aureon Laboratories is a life sciences company dedicated to enabling the advancement of predictive and personalized cancer treatment. Aureon provides objective, actionable information allowing patients and physicians to make more-informed treatment decisions through its prostate diagnostic tools.

Molecular Biometrics, Inc. is a metabolomics company based in Norwood, Massachusetts, focused on developing non-invasive methodologies for assessing biological functions in health and disease, as well as for drug discovery and development. Established in 2005, the company leverages innovative metabolomic technologies to create molecular gastronomy kits. Its applications span various fields, including reproductive health, neurodegenerative diseases such as Alzheimer's and Parkinson's, maternal-fetal medicine, pulmonary metabolism and edema, and lactate metabolism. By applying its proprietary technology, Molecular Biometrics aims to enhance the understanding and treatment of complex health issues.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

Helicos BioSciences Corporation (Helicos) is a life sciences company, which has developed technology focused on the research, drug discovery and clinical diagnostics markets. Its True Single Molecule Sequencing (tSMS) technology enables rapid analysis of large quantities of genetic material by directly sequencing single molecules of deoxyribonucleic acid (DNA) or single DNA copies of ribonucleic acid (RNA) (cDNA) and its approach of direct sequencing of RNA. Its Helicos Genetic Analysis Platform is designed to obtain sequencing information by repetitively performing a cycle of biochemical reactions on individual DNA or RNA molecules and imaging the results after each cycle. The platform consists of an instrument called the HeliScope Single Molecule Sequencer, an image analysis computer tower called the HeliScope Analysis Engine, associated reagents, which are chemicals used in the sequencing process and disposable supplies.

Avila Therapeutics is focused on designing and developing covalent drugs aimed at treating viral infections, cancers, and autoimmune diseases. One of its key products is AVL-181, a small molecule that acts as a protease inhibitor for hepatitis C virus. Founded in 2006 and based in Waltham, Massachusetts, Avila Therapeutics operates as a subsidiary of Celgene Corporation, following its acquisition in 2012. The company has also established a strategic alliance with Sanofi-Aventis to enhance its research and development efforts.

Adnexus, a Bristol-Myers Squibb R&D Company, specializes in the discovery and development of Adnectins, a unique class of targeted biologics. These proteins are engineered to block or stimulate therapeutic targets to combat various diseases, addressing significant medical needs in oncology, immunology, and cardiovascular health. Utilizing a proprietary protein engineering system known as PROfusion™, Adnexus can generate a vast library of over 10 trillion potential Adnectins. This technology enhances the efficiency of identifying candidates with desirable therapeutic properties. Among its product candidates is Angiocept/CT-322, an anti-angiogenesis agent aimed at treating cancer by inhibiting the VEGFR-2 pathway. Since its acquisition by Bristol-Myers Squibb in 2007, Adnexus has further advanced its capabilities in developing innovative medicines to improve patient outcomes.

ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases. ARCA’s principal focus is to develop personalized therapies for the treatment of cardiovascular disease through the use of genetics. The Company’s business focus combines expertise in cardiovascular pathophysiology, molecular genetics and clinical development.

Novexel is a pharmaceutical company dedicated to the research and development of innovative anti-infective therapies for severe bacterial and fungal infections. With a robust portfolio of antibacterial and anti-fungal compounds, the company addresses the challenges posed by multi-resistant organisms. Novexel's expertise spans various scientific disciplines, including biology, medicinal chemistry, pharmacology, microbiology, and clinical development. This multidisciplinary approach positions the company at the forefront of discovering and developing new treatments for infectious diseases, aiming to provide effective solutions against microbial resistance.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

Adnexus, a Bristol-Myers Squibb R&D Company, specializes in the discovery and development of Adnectins, a unique class of targeted biologics. These proteins are engineered to block or stimulate therapeutic targets to combat various diseases, addressing significant medical needs in oncology, immunology, and cardiovascular health. Utilizing a proprietary protein engineering system known as PROfusion™, Adnexus can generate a vast library of over 10 trillion potential Adnectins. This technology enhances the efficiency of identifying candidates with desirable therapeutic properties. Among its product candidates is Angiocept/CT-322, an anti-angiogenesis agent aimed at treating cancer by inhibiting the VEGFR-2 pathway. Since its acquisition by Bristol-Myers Squibb in 2007, Adnexus has further advanced its capabilities in developing innovative medicines to improve patient outcomes.

U3 Pharma GmbH, based in Martinsried near Munich, Germany, is a leader in targeted cancer drug development. Established in July 2001, the company has produced a pipeline of novel targeted therapeutics based on the ground-breaking discoveries made by its founder - Professor Axel Ullrich. Since May 2008 U3 Pharma belongs to DAIICHI Sankyo Co. Ltd., one of the top 20 leading pharmaceutical companies worldwide and no. 3 in Japan. Being part of this global environment, U3 Pharma is striving to provide patients around the world with innovative antibody-based therapeutics.

ARCA biopharma, Inc. is a Colorado-based biopharmaceutical company developing genetically-targeted therapies for cardiovascular diseases. ARCA’s principal focus is to develop personalized therapies for the treatment of cardiovascular disease through the use of genetics. The Company’s business focus combines expertise in cardiovascular pathophysiology, molecular genetics and clinical development.

Xytis is a biopharmaceutical company focusing on the discovery and development of innovative CNS drug candidates. The company is privately held and was created in Jan 2006 by the merger of Xytis Pharmaceuticals Ltd. (UK) and Remergent, Inc. (USA). The company received funding from Sanderling Ventures, Atlas Venture, CDC Entreprises Innovation, and Ventech. Xytis Inc. is assembling a pipeline of innovative compounds with validated mechanisms of action, addressing major unmet clinical needs in CNS fields such as Schizophrenia, Traumatic Brain Injury, Depression, Anxiety, or Insomnia. The company has two clinical stage compounds in development and operates from the USA, UK and Switzerland.

Aureon Laboratories is a life sciences company dedicated to enabling the advancement of predictive and personalized cancer treatment. Aureon provides objective, actionable information allowing patients and physicians to make more-informed treatment decisions through its prostate diagnostic tools.

Novexel is a pharmaceutical company dedicated to the research and development of innovative anti-infective therapies for severe bacterial and fungal infections. With a robust portfolio of antibacterial and anti-fungal compounds, the company addresses the challenges posed by multi-resistant organisms. Novexel's expertise spans various scientific disciplines, including biology, medicinal chemistry, pharmacology, microbiology, and clinical development. This multidisciplinary approach positions the company at the forefront of discovering and developing new treatments for infectious diseases, aiming to provide effective solutions against microbial resistance.

U3 Pharma GmbH, based in Martinsried near Munich, Germany, is a leader in targeted cancer drug development. Established in July 2001, the company has produced a pipeline of novel targeted therapeutics based on the ground-breaking discoveries made by its founder - Professor Axel Ullrich. Since May 2008 U3 Pharma belongs to DAIICHI Sankyo Co. Ltd., one of the top 20 leading pharmaceutical companies worldwide and no. 3 in Japan. Being part of this global environment, U3 Pharma is striving to provide patients around the world with innovative antibody-based therapeutics.

Arrow Therapeutics was founded in 1998 and is already making a significant contribution to anti-infective drug discovery. Microbial resistance to existing drugs is an ever increasing threat that is leading to serious unmet medical need and incurring a huge financial burden on society. Despite the huge challenges of this situation it also presents excellent opportunities to combine scientific research and business. As more and more big pharma groups concentrate their efforts on drug development, greater chances are open to smaller companies such as ours to show their excellence in research.