Atlas Venture

Kailera Therapeutics is a clinical-stage biotechnology company that provides revolutionary therapies for treating obesity and related diseases. Kailera Therapeutics is advancing a range of injectable and oral GLP-1 (Glucagon-like peptide-1) therapies for obesity and related diseases.

Pheon Therapeutics is an Antibody-Drug Conjugate (ADC) specialist that creates ADCs for a variety of difficult-to-treat cancers. Its lead program is a first-in-class ADC against a novel target that is highly expressed in solid tumors across a broad range of hard-to-treat cancer types. The company was founded in 2022 and is based in London, England.

Comanche Biopharma is a biopharmaceutical company focused on developing an investigational small interfering RNA (siRNA) medicine aimed at treating preterm preeclampsia. The company is committed to ensuring that all women and their babies have access to safe and effective therapies for life-threatening complications during pregnancy. Comanche Biopharma's mission emphasizes the importance of evidence-based and affordable solutions, enabling women to receive cost-effective treatments that address the root causes of preeclampsia.

Nvelop Therapeutics is engaged in the development of advanced genetic medicines aimed at addressing severe genetic diseases. The company specializes in creating programmable delivery systems that enhance in vivo gene editing and facilitate the delivery of therapeutic cargo. By integrating epigenetic editing technologies with innovative delivery methods, Nvelop Therapeutics targets unmet medical needs, particularly in therapeutically relevant cells and tissues. Their focus on novel gene editing delivery technologies enables medical professionals to effectively deliver a range of therapeutic payloads, thereby improving treatment options for patients suffering from these challenging conditions.

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.

Pheon Therapeutics is an Antibody-Drug Conjugate (ADC) specialist that creates ADCs for a variety of difficult-to-treat cancers. Its lead program is a first-in-class ADC against a novel target that is highly expressed in solid tumors across a broad range of hard-to-treat cancer types. The company was founded in 2022 and is based in London, England.

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, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic 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.

Matchpoint Therapeutics is a biotechnology company focused on developing precision covalent medicines aimed at transforming the treatment of immune diseases and other serious conditions. Utilizing its proprietary Advanced Covalent Exploration (ACE) platform, the company combines advanced chemoproteomics, machine learning, and the evolution of covalent chemistry libraries to enhance drug discovery. This innovative approach allows for a deeper exploration of the proteome, enabling the identification and targeting of new binding sites on disease-causing proteins. Matchpoint is building an emerging pipeline of novel covalent medicines, with an initial emphasis on immunology.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic 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.

Day One Biopharmaceuticals is a clinical-stage biotechnology company that specializes in developing targeted cancer therapies for patients of all ages, with a particular focus on pediatric patients. Recognizing the advancements in cancer research, the company aims to create treatments that can benefit both children and adults diagnosed with genetically defined cancers. Day One Biopharmaceuticals prioritizes the rapid development of therapies to meet the urgent needs of families facing cancer diagnoses. Their lead product candidate, DAY101, is an oral, highly-selective pan-RAF kinase inhibitor designed to penetrate the brain, addressing a significant treatment gap in pediatric oncology. By licensing and acquiring promising products from research institutions and other companies, Day One Biopharmaceuticals strives to bring effective medicines to market for those impacted by cancer.

Ikena Oncology is a biotechnology company based in Boston, Massachusetts, focused on developing innovative therapies for cancer treatment. The company specializes in biomarker-driven and precision therapies that target specific mechanisms involved in cancer growth, spread, and resistance. Its product pipeline includes several candidates such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-930, an oral small-molecule inhibitor of the TEAD transcription factor in the Hippo signaling pathway. Additionally, Ikena is exploring a kynurenine-degrading enzyme with IK-412 and other discovery-stage programs targeting various oncogenic pathways. Established in 2016, Ikena Oncology aims to address unmet medical needs through a combination of chemistry, translational science, and patient-centered drug development.

Vigil Neuroscience Inc. is a therapeutics company focused on microglia, the brain's immune sentinel cells, to address both rare and common neurodegenerative diseases. The company aims to restore the vigilance of microglia to improve patient outcomes. Vigil is advancing its lead candidate, a monoclonal antibody TREM2 agonist, through Phase 1 studies and is also developing an orally available small molecule TREM2 agonist, VG-3927, which is positioned to enter Investigational New Drug (IND) application. Additionally, Vigil is conducting the IGNITE trial, a Phase 2 proof-of-concept study that represents the first interventional trial for patients with adult-onset leukodystrophy. By leveraging modern neuroscience and various therapeutic modalities, Vigil seeks to deliver precision therapies that enhance the lives of patients and their families while expanding its pipeline and supporting the understanding of microglia biology as a critical therapeutic target.

F-Star Therapeutics is a clinical-stage biopharmaceutical company focused on creating innovative bispecific antibody therapies aimed at improving cancer treatment. Utilizing its proprietary Modular Antibody Technology, F-Star develops bispecific antibodies that enhance the capabilities of traditional monoclonal antibodies while minimizing production challenges. The company's unique approach allows for the rapid discovery and development of these therapies, which seek to block tumor immune evasion and provide patients with more effective and durable treatment options compared to existing immuno-oncology therapies. Founded in 2006, F-Star has established a solid patent position and has attracted significant investment from leading venture capital firms and strategic corporate partners. The company has also formed alliances with major pharmaceutical companies, allowing it to expand its research and development efforts. With a dedicated team based in Cambridge, UK, F-Star is advancing a pipeline of product candidates that leverage its innovative technology.

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. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing nucleic acid-based therapeutics aimed at editing RNA to treat rare and prevalent diseases. Founded in 2018, the company has created a proprietary platform that allows for efficient and selective RNA editing, leveraging natural processes found in multicellular organisms. This innovative approach enables precise modifications to messenger RNA, allowing for targeted changes in protein structure and function across various tissues. By focusing on RNA instead of DNA, Korro Bio aims to enhance the precision and adaptability of genetic medicines, expanding their potential applications in treating a range of medical conditions.

Dyne Therapeutics is a biotechnology company based in Waltham, Massachusetts, dedicated to developing targeted therapies for genetically driven muscle diseases. Founded in 2017, the company focuses on advancing treatments for conditions such as myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral dystrophy, among others. Utilizing its innovative FORCE platform, Dyne Therapeutics aims to deliver disease-modifying therapies with enhanced precision to skeletal, cardiac, and smooth muscle tissues. The company's lead product candidates, DYNE-101 and DYNE-251, are currently undergoing phase 1/2 clinical trials, reflecting its commitment to addressing the unmet medical needs of patients with serious muscle disorders.

Aerovate Therapeutics is a clinical-stage biotechnology company based in Boston, Massachusetts, dedicated to developing innovative treatments for patients with rare cardiopulmonary diseases. The company's primary focus is on AV-101, a proprietary inhaled dry powder formulation of the drug imatinib, designed specifically for the treatment of pulmonary arterial hypertension (PAH). This formulation enables targeted delivery of the medication directly to affected lung tissues, thereby minimizing systemic side effects commonly associated with traditional treatments. Established in 2018, Aerovate aims to provide meaningful improvements in the lives of those suffering from these serious conditions.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

Day One Biopharmaceuticals is a clinical-stage biotechnology company that specializes in developing targeted cancer therapies for patients of all ages, with a particular focus on pediatric patients. Recognizing the advancements in cancer research, the company aims to create treatments that can benefit both children and adults diagnosed with genetically defined cancers. Day One Biopharmaceuticals prioritizes the rapid development of therapies to meet the urgent needs of families facing cancer diagnoses. Their lead product candidate, DAY101, is an oral, highly-selective pan-RAF kinase inhibitor designed to penetrate the brain, addressing a significant treatment gap in pediatric oncology. By licensing and acquiring promising products from research institutions and other companies, Day One Biopharmaceuticals strives to bring effective medicines to market for those impacted by cancer.

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.

Third Harmonic Bio is a clinical-stage company developing a first-in-class, highly selective, oral KIT inhibitor for treatment of severe allergy and inflammation. KIT is a cell surface receptor that acts as the master survival and functional regulator of mast cells, which are immune cells concentrated at the body’s interfaces with the external environment and that act as the key mediator of the inflammation associated with allergic diseases.

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 company developing step-change cell and gene therapies targeting cancer and rare disease. The catalyst for creating Avrobio is to accelerate scientific breakthroughs related to the convergence of cell and gene therapies. The patient’s own cells can be effectively modified to deliver novel genes to cure serious debilitating disease. Avrobio was founded by industry leaders with unmatched scientific, business, and manufacturing expertise in the field of cell and gene therapy.

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. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing nucleic acid-based therapeutics aimed at editing RNA to treat rare and prevalent diseases. Founded in 2018, the company has created a proprietary platform that allows for efficient and selective RNA editing, leveraging natural processes found in multicellular organisms. This innovative approach enables precise modifications to messenger RNA, allowing for targeted changes in protein structure and function across various tissues. By focusing on RNA instead of DNA, Korro Bio aims to enhance the precision and adaptability of genetic medicines, expanding their potential applications in treating a range of medical conditions.

Obsidian Therapeutics is a developer of a next-generation cell and gene therapies created to extend adoptive immunotherapy to every patient with cancer.The company's next-generation cell and gene therapies with pharmacologic operating systems provide exquisite control of protein activity in cells and create adoptive cell therapies with new functions that are under control of the treating physician using simple, safe, orally-active, marketed drugs, enabling patients to improve on the current generation of cell therapies.

Kymera Therapeutics is a biopharmaceutical company based in Watertown, Massachusetts, that specializes in the discovery and development of novel small molecule therapeutics aimed at selectively degrading disease-causing proteins. The company utilizes a proprietary platform for targeted protein degradation, leveraging the body’s natural protein recycling system. Kymera is developing several programs, including the IRAK4 program for immunology-inflammation diseases such as hidradenitis suppurativa and rheumatoid arthritis, the IRAKIMiD program to address MYD88-mutated diffuse large B cell lymphoma, and the STAT3 program targeting hematologic malignancies, solid tumors, and autoimmune diseases. Founded in 2015, Kymera Therapeutics is pioneering new approaches to treat diseases that have previously been considered untreatable, focusing on previously inaccessible therapeutic targets.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, established in 2015. The company is dedicated to developing innovative therapies for patients with dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, ensuring that their therapeutic candidates are tailored to the specific molecular abnormalities found in patients with significant clinical needs. Their extensive pipeline includes a range of modalities such as monoclonal antibodies, recombinant proteins, and gene therapies. Supported by prominent life science investors and global academic collaborations, Gemini Therapeutics aims to create potentially first-in-class therapeutics to address unmet medical needs in the field of ophthalmology.

Replimune Group is a biotechnology company focused on developing oncolytic immune-gene therapies for cancer treatment. Utilizing its proprietary Immulytic platform, Replimune aims to create product candidates that activate the immune system against tumors. The company's lead candidate, RP1, is a modified herpes simplex virus currently undergoing Phase I/II clinical trials for various solid tumors and Phase II trials specifically for cutaneous squamous cell carcinoma. Additionally, Replimune is developing RP2, which targets CTLA-4 to enhance immune response, and RP3, designed to express immune-activating proteins that stimulate T cells. Founded in 2015 and based in Woburn, Massachusetts, Replimune seeks to advance its therapies through clinical trials and explore combinations with other immuno-oncology treatments to enhance efficacy.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

Magenta Therapeutics is a clinical-stage biotechnology company focused on developing innovative therapies to enhance stem cell transplantation for patients with blood cancers, genetic diseases, and autoimmune disorders. The company aims to improve the safety and efficacy of stem cell transplants, which are currently high-risk procedures often considered only as a last resort. Its pipeline includes several key product candidates, such as targeted antibody-drug conjugates for transplant conditioning, a novel stem cell mobilization agent, and allogeneic stem cell therapies. Magenta is also exploring collaborations to assess the effectiveness of its therapies in combination with existing treatments, such as gene therapies for sickle cell disease. Founded in 2015 and headquartered in Cambridge, Massachusetts, Magenta Therapeutics seeks to expand the curative potential of stem cell therapies to a broader patient population by addressing the toxicity and long-term side effects associated with current transplant methods.

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. The company’s leading product candidate, tebipenem pivoxil hydrobromide, is an oral antibiotic designed to treat MDR Gram-negative infections in adults. Additionally, Spero is advancing SPR206, an intravenous agent targeting MDR Gram-negative infections, and SPR720, an oral antibiotic aimed at treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics collaborates with various organizations, including Meiji Seika Pharma for tebipenem HBr, Everest Medicines for SPR206 in Asia, and the Bill & Melinda Gates Medical Research Institute for SPR720. Founded in 2013, Spero leverages a strong team in chemistry and microbiology to address significant unmet medical needs in infectious disease treatment.

Magenta Therapeutics is a clinical-stage biotechnology company focused on developing innovative therapies to enhance stem cell transplantation for patients with blood cancers, genetic diseases, and autoimmune disorders. The company aims to improve the safety and efficacy of stem cell transplants, which are currently high-risk procedures often considered only as a last resort. Its pipeline includes several key product candidates, such as targeted antibody-drug conjugates for transplant conditioning, a novel stem cell mobilization agent, and allogeneic stem cell therapies. Magenta is also exploring collaborations to assess the effectiveness of its therapies in combination with existing treatments, such as gene therapies for sickle cell disease. Founded in 2015 and headquartered in Cambridge, Massachusetts, Magenta Therapeutics seeks to expand the curative potential of stem cell therapies to a broader patient population by addressing the toxicity and long-term side effects associated with current transplant methods.

Avrobio is a clinical stage company developing step-change cell and gene therapies targeting cancer and rare disease. The catalyst for creating Avrobio is to accelerate scientific breakthroughs related to the convergence of cell and gene therapies. The patient’s own cells can be effectively modified to deliver novel genes to cure serious debilitating disease. Avrobio was founded by industry leaders with unmatched scientific, business, and manufacturing expertise in the field of cell and gene therapy.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, established in 2015. The company is dedicated to developing innovative therapies for patients with dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, ensuring that their therapeutic candidates are tailored to the specific molecular abnormalities found in patients with significant clinical needs. Their extensive pipeline includes a range of modalities such as monoclonal antibodies, recombinant proteins, and gene therapies. Supported by prominent life science investors and global academic collaborations, Gemini Therapeutics aims to create potentially first-in-class therapeutics to address unmet medical needs in the field of ophthalmology.

Avrobio is a clinical stage company developing step-change cell and gene therapies targeting cancer and rare disease. The catalyst for creating Avrobio is to accelerate scientific breakthroughs related to the convergence of cell and gene therapies. The patient’s own cells can be effectively modified to deliver novel genes to cure serious debilitating disease. Avrobio was founded by industry leaders with unmatched scientific, business, and manufacturing expertise in the field of cell and gene therapy.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

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. The company’s leading product candidate, tebipenem pivoxil hydrobromide, is an oral antibiotic designed to treat MDR Gram-negative infections in adults. Additionally, Spero is advancing SPR206, an intravenous agent targeting MDR Gram-negative infections, and SPR720, an oral antibiotic aimed at treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics collaborates with various organizations, including Meiji Seika Pharma for tebipenem HBr, Everest Medicines for SPR206 in Asia, and the Bill & Melinda Gates Medical Research Institute for SPR720. Founded in 2013, Spero leverages a strong team in chemistry and microbiology to address significant unmet medical needs in infectious disease treatment.

Viridian Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative treatments for patients with diseases that lack effective therapies. The company focuses on addressing conditions such as thyroid eye disease, a debilitating orphan disease characterized by inflammation and swelling of the eye muscles. One of its key product candidates, VRDN-001, is an insulin-like growth factor-1 receptor monoclonal antibody designed to potentially treat this condition. Viridian Therapeutics aims to provide new options for patients suffering from these underserved medical needs through its advanced research and development initiatives.

Replimune Group is a biotechnology company focused on developing oncolytic immune-gene therapies for cancer treatment. Utilizing its proprietary Immulytic platform, Replimune aims to create product candidates that activate the immune system against tumors. The company's lead candidate, RP1, is a modified herpes simplex virus currently undergoing Phase I/II clinical trials for various solid tumors and Phase II trials specifically for cutaneous squamous cell carcinoma. Additionally, Replimune is developing RP2, which targets CTLA-4 to enhance immune response, and RP3, designed to express immune-activating proteins that stimulate T cells. Founded in 2015 and based in Woburn, Massachusetts, Replimune seeks to advance its therapies through clinical trials and explore combinations with other immuno-oncology treatments to enhance efficacy.

Intellia Therapeutics is a genome editing company headquartered in Cambridge, Massachusetts, that specializes in developing innovative therapeutics using the CRISPR/Cas9 technology. The company focuses on in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Additionally, Intellia's ex vivo pipeline includes engineered cell therapies aimed at treating various oncological and autoimmune diseases. The company has entered into collaborations with notable organizations, including Novartis and Regeneron Pharmaceuticals, to advance its research and development efforts. Founded in 2014, Intellia Therapeutics is committed to addressing severe and life-threatening diseases through its comprehensive intellectual property platform and a broad range of clinical development programs.

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. The company’s leading product candidate, tebipenem pivoxil hydrobromide, is an oral antibiotic designed to treat MDR Gram-negative infections in adults. Additionally, Spero is advancing SPR206, an intravenous agent targeting MDR Gram-negative infections, and SPR720, an oral antibiotic aimed at treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics collaborates with various organizations, including Meiji Seika Pharma for tebipenem HBr, Everest Medicines for SPR206 in Asia, and the Bill & Melinda Gates Medical Research Institute for SPR720. Founded in 2013, Spero leverages a strong team in chemistry and microbiology to address significant unmet medical needs in infectious disease treatment.

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.

Viridian Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative treatments for patients with diseases that lack effective therapies. The company focuses on addressing conditions such as thyroid eye disease, a debilitating orphan disease characterized by inflammation and swelling of the eye muscles. One of its key product candidates, VRDN-001, is an insulin-like growth factor-1 receptor monoclonal antibody designed to potentially treat this condition. Viridian Therapeutics aims to provide new options for patients suffering from these underserved medical needs through its advanced research and development initiatives.

Lysosomal Therapeutics is dedicated to innovative small-molecule research and development in the field of neurodegeneration, yielding new treatment options for patients with severe neurological diseases. Their strategy leverages the clinically-validated link between lysosome-based genetic disorders and neurodegenerative diseases to establish a unique and effective molecular platform for novel drug discovery. LTI’s lead program targets Gaucher-related neurodegeneration, Parkinson’s disease, and other synucleinopathies.

Surface Oncology, Inc. is a clinical-stage immuno-oncology company focused on developing innovative cancer therapies. The company is advancing a pipeline of monoclonal antibodies, including SRF231, which inhibits CD47; NZV930, targeting CD73; SRF617, aimed at CD39; SRF388, which targets interleukin 27; and SRF813, focused on CD112R. Additionally, Surface is exploring earlier-stage programs that address critical elements of the tumor microenvironment, such as regulatory T cells and natural killer cells. The company has established collaborations with notable partners, including Novartis Institutes for Biomedical Research for cancer therapy development and Merck Sharp & Dohme Corp. to assess the safety and efficacy of combining SRF617 with KEYTRUDA, an anti-PD-1 therapy. Founded in 2014 and headquartered in Cambridge, Massachusetts, Surface Oncology aims to create conditions that enhance anti-tumor immune responses through its targeted approaches in cancer immunotherapy.

Intellia Therapeutics is a genome editing company headquartered in Cambridge, Massachusetts, that specializes in developing innovative therapeutics using the CRISPR/Cas9 technology. The company focuses on in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Additionally, Intellia's ex vivo pipeline includes engineered cell therapies aimed at treating various oncological and autoimmune diseases. The company has entered into collaborations with notable organizations, including Novartis and Regeneron Pharmaceuticals, to advance its research and development efforts. Founded in 2014, Intellia Therapeutics is committed to addressing severe and life-threatening diseases through its comprehensive intellectual property platform and a broad range of clinical development programs.

Quartet Medicine, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative treatments for chronic pain and inflammation. Founded in 2013 by scientists from Boston Children’s Hospital and École Polytechnique Fédérale de Lausanne, Quartet aims to restore tetrahydrobiopterin (BH4) homeostasis in neuronal and inflammatory cells. Research indicates that increased levels of BH4 are essential for regulating peripheral nerve function and immune responses. By leveraging these insights, Quartet is working on novel non-opioid medications that target this biochemical pathway, providing an alternative approach to managing pain and inflammation. The company collaborates with various research partners in Europe and Asia to advance its therapeutic offerings.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

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.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

Surface Oncology, Inc. is a clinical-stage immuno-oncology company focused on developing innovative cancer therapies. The company is advancing a pipeline of monoclonal antibodies, including SRF231, which inhibits CD47; NZV930, targeting CD73; SRF617, aimed at CD39; SRF388, which targets interleukin 27; and SRF813, focused on CD112R. Additionally, Surface is exploring earlier-stage programs that address critical elements of the tumor microenvironment, such as regulatory T cells and natural killer cells. The company has established collaborations with notable partners, including Novartis Institutes for Biomedical Research for cancer therapy development and Merck Sharp & Dohme Corp. to assess the safety and efficacy of combining SRF617 with KEYTRUDA, an anti-PD-1 therapy. Founded in 2014 and headquartered in Cambridge, Massachusetts, Surface Oncology aims to create conditions that enhance anti-tumor immune responses through its targeted approaches in cancer immunotherapy.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

Viridian Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative treatments for patients with diseases that lack effective therapies. The company focuses on addressing conditions such as thyroid eye disease, a debilitating orphan disease characterized by inflammation and swelling of the eye muscles. One of its key product candidates, VRDN-001, is an insulin-like growth factor-1 receptor monoclonal antibody designed to potentially treat this condition. Viridian Therapeutics aims to provide new options for patients suffering from these underserved medical needs through its advanced research and development initiatives.

Lysosomal Therapeutics is dedicated to innovative small-molecule research and development in the field of neurodegeneration, yielding new treatment options for patients with severe neurological diseases. Their strategy leverages the clinically-validated link between lysosome-based genetic disorders and neurodegenerative diseases to establish a unique and effective molecular platform for novel drug discovery. LTI’s lead program targets Gaucher-related neurodegeneration, Parkinson’s disease, and other synucleinopathies.

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. The company’s leading product candidate, tebipenem pivoxil hydrobromide, is an oral antibiotic designed to treat MDR Gram-negative infections in adults. Additionally, Spero is advancing SPR206, an intravenous agent targeting MDR Gram-negative infections, and SPR720, an oral antibiotic aimed at treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics collaborates with various organizations, including Meiji Seika Pharma for tebipenem HBr, Everest Medicines for SPR206 in Asia, and the Bill & Melinda Gates Medical Research Institute for SPR720. Founded in 2013, Spero leverages a strong team in chemistry and microbiology to address significant unmet medical needs in infectious disease treatment.

Egalet is a specialty pharmaceutical company that develops pain management products. The company is developing a pipeline of abuse-resistant opioids that are nearing pivotal testing. Egalet has pioneered one of the world’s first erosion-based delivery technologies to enable the controlled release of drugs through gradual erosion of a tablet and has optimized the abuse deterrent properties that can be applied to a broad range of opioids and non-opioids.

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. The company’s leading product candidate, tebipenem pivoxil hydrobromide, is an oral antibiotic designed to treat MDR Gram-negative infections in adults. Additionally, Spero is advancing SPR206, an intravenous agent targeting MDR Gram-negative infections, and SPR720, an oral antibiotic aimed at treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics collaborates with various organizations, including Meiji Seika Pharma for tebipenem HBr, Everest Medicines for SPR206 in Asia, and the Bill & Melinda Gates Medical Research Institute for SPR720. Founded in 2013, Spero leverages a strong team in chemistry and microbiology to address significant unmet medical needs in infectious disease treatment.

F-Star Therapeutics is a clinical-stage biopharmaceutical company focused on creating innovative bispecific antibody therapies aimed at improving cancer treatment. Utilizing its proprietary Modular Antibody Technology, F-Star develops bispecific antibodies that enhance the capabilities of traditional monoclonal antibodies while minimizing production challenges. The company's unique approach allows for the rapid discovery and development of these therapies, which seek to block tumor immune evasion and provide patients with more effective and durable treatment options compared to existing immuno-oncology therapies. Founded in 2006, F-Star has established a solid patent position and has attracted significant investment from leading venture capital firms and strategic corporate partners. The company has also formed alliances with major pharmaceutical companies, allowing it to expand its research and development efforts. With a dedicated team based in Cambridge, UK, F-Star is advancing a pipeline of product candidates that leverage its innovative technology.

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.

Egalet is a specialty pharmaceutical company that develops pain management products. The company is developing a pipeline of abuse-resistant opioids that are nearing pivotal testing. Egalet has pioneered one of the world’s first erosion-based delivery technologies to enable the controlled release of drugs through gradual erosion of a tablet and has optimized the abuse deterrent properties that can be applied to a broad range of opioids and non-opioids.

Viridian Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative treatments for patients with diseases that lack effective therapies. The company focuses on addressing conditions such as thyroid eye disease, a debilitating orphan disease characterized by inflammation and swelling of the eye muscles. One of its key product candidates, VRDN-001, is an insulin-like growth factor-1 receptor monoclonal antibody designed to potentially treat this condition. Viridian Therapeutics aims to provide new options for patients suffering from these underserved medical needs through its advanced research and development initiatives.

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.

F-Star Therapeutics is a clinical-stage biopharmaceutical company focused on creating innovative bispecific antibody therapies aimed at improving cancer treatment. Utilizing its proprietary Modular Antibody Technology, F-Star develops bispecific antibodies that enhance the capabilities of traditional monoclonal antibodies while minimizing production challenges. The company's unique approach allows for the rapid discovery and development of these therapies, which seek to block tumor immune evasion and provide patients with more effective and durable treatment options compared to existing immuno-oncology therapies. Founded in 2006, F-Star has established a solid patent position and has attracted significant investment from leading venture capital firms and strategic corporate partners. The company has also formed alliances with major pharmaceutical companies, allowing it to expand its research and development efforts. With a dedicated team based in Cambridge, UK, F-Star is advancing a pipeline of product candidates that leverage its innovative technology.

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.

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.

Molecular Biometrics, Inc., is applying novel metabolomic technologies to develop accurate, non-invasive methodologies for evaluating normal biologic function in health and disease, and for drug discovery and development. The company's proprietary technology is being applied in reproductive health, neurodegenerative disease (Alzheimer's disease and Parkinson's disease), maternal fetal medicine, pulmonary metabolism and edema, and lactate metabolism.

F-Star Therapeutics is a clinical-stage biopharmaceutical company focused on creating innovative bispecific antibody therapies aimed at improving cancer treatment. Utilizing its proprietary Modular Antibody Technology, F-Star develops bispecific antibodies that enhance the capabilities of traditional monoclonal antibodies while minimizing production challenges. The company's unique approach allows for the rapid discovery and development of these therapies, which seek to block tumor immune evasion and provide patients with more effective and durable treatment options compared to existing immuno-oncology therapies. Founded in 2006, F-Star has established a solid patent position and has attracted significant investment from leading venture capital firms and strategic corporate partners. The company has also formed alliances with major pharmaceutical companies, allowing it to expand its research and development efforts. With a dedicated team based in Cambridge, UK, F-Star is advancing a pipeline of product candidates that leverage its innovative technology.

On-Q-ity is an innovative diagnostic company that is focused on providing physicians with the tools they need to make the best decision when treating individual cancer patients. There platform will enable better decision-making across multiple cancers: improving the quality of life for patients and ultimately increasing survival. Products currently in development are based on two core technologies: 1) DNA repair biomarkers that predict treatment response and 2) Microfluidic chip technology that enables capture, enumeration and characterization of circulating tumor cells (CTCs) in the bloodstream.

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.

Viridian Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative treatments for patients with diseases that lack effective therapies. The company focuses on addressing conditions such as thyroid eye disease, a debilitating orphan disease characterized by inflammation and swelling of the eye muscles. One of its key product candidates, VRDN-001, is an insulin-like growth factor-1 receptor monoclonal antibody designed to potentially treat this condition. Viridian Therapeutics aims to provide new options for patients suffering from these underserved medical needs through its advanced research and development initiatives.

InfaCare Pharmaceutical specializes in developing pharmaceuticals tailored for neonatal and pediatric patients, focusing on addressing unmet medical needs in these vulnerable populations. Their portfolio includes treatments for autoimmune and rare diseases across several specialty areas, such as neurology, rheumatology, nephrology, pulmonology, and immunotherapy. A notable product is a heme oxygenase inhibitor designed for the treatment of neonatal hyperbilirubinemia, which can be administered as a single intramuscular injection to both term and pre-term newborns. This innovative treatment helps to break down heme and inhibit bilirubin production, thereby reducing the necessity for phototherapy and exchange transfusions in affected infants. Through its targeted therapies, InfaCare aims to improve health outcomes for children facing critical health challenges.

Viridian Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative treatments for patients with diseases that lack effective therapies. The company focuses on addressing conditions such as thyroid eye disease, a debilitating orphan disease characterized by inflammation and swelling of the eye muscles. One of its key product candidates, VRDN-001, is an insulin-like growth factor-1 receptor monoclonal antibody designed to potentially treat this condition. Viridian Therapeutics aims to provide new options for patients suffering from these underserved medical needs through its advanced research and development initiatives.

Stromedix is a biopharmaceutical company dedicated to developing therapies for organ failure due to fibrosis. Initially, it will concentrate on chronic indications, utilizing its expertise to remodel tissues affected by this condition. In the future, Stromedix plans to expand its therapeutic pipeline to address related areas, including acute organ failure and cancer. Through its innovative approach, the company aims to provide effective treatments that improve patient outcomes in these challenging medical contexts.

NovaMed Pharmaceuticals (诺凡麦医药) is a Chinese group based on virtual integration. It is focused on complete business functions such as sales and marketing, register, circulation, manufacture, and R&D. NovaMed Pharmaceuticals was founded by senior management personnel in transnational pharmaceutical companies. The founders are professionals with broad social networks and domestic market knowledge.

Vitae Pharmaceuticals is a clinical-stage biotechnology company based in Fort Washington, Pennsylvania, dedicated to discovering and developing novel small molecule drugs for diseases with significant unmet medical needs. Its pipeline includes VTP-43742, currently in Phase I clinical trials for autoimmune disorders such as psoriasis and multiple sclerosis; VTP-38543, in Phase IIa for atopic dermatitis; and BI 1147560, aimed at treating and preventing Alzheimer's disease. Additionally, the company is developing VTP-34072, which is in Phase II trials for type 2 diabetes, and VTP-38443 for acute coronary syndrome. Vitae Pharmaceuticals employs a structure-based drug-design platform, Contour, to create best-in-class compounds targeting large markets that include chronic kidney disease and atherosclerosis. Established in 2001, the company operates as a subsidiary of Allergan plc.

F-Star Therapeutics is a clinical-stage biopharmaceutical company focused on creating innovative bispecific antibody therapies aimed at improving cancer treatment. Utilizing its proprietary Modular Antibody Technology, F-Star develops bispecific antibodies that enhance the capabilities of traditional monoclonal antibodies while minimizing production challenges. The company's unique approach allows for the rapid discovery and development of these therapies, which seek to block tumor immune evasion and provide patients with more effective and durable treatment options compared to existing immuno-oncology therapies. Founded in 2006, F-Star has established a solid patent position and has attracted significant investment from leading venture capital firms and strategic corporate partners. The company has also formed alliances with major pharmaceutical companies, allowing it to expand its research and development efforts. With a dedicated team based in Cambridge, UK, F-Star is advancing a pipeline of product candidates that leverage its innovative technology.

Prestwick Pharmaceuticals develops and markets therapeutic pharmaceutical drugs for chronic central nervous systems diseases. The company offers products for restless legs syndrome, schizophrenia, autism, Alzheimer’s, and sleep apnea ailments. Prestwick Pharmaceuticals was founded in 2002 and is based in Washington, District of Columbia.

Adnexus, a Bristol-Myers Squibb R&D Company, is leading the discovery and development of Adnectinsâ„¢, a novel, proprietary class of targeted biologics. Adnectins are proteins designed to either block or stimulate therapeutic targets of interest to fight diseases across a broad range of therapeutic areas. Their proprietary technology for engineering these versatile proteins creates an opportunity to rapidly generate highly differentiated medicines. Adnexus, a Bristol-Myers Squibb R&D Company, is a leader in the discovery and development of Adnectins, a proprietary type of targeted biologic. Adnectins are proteins designed to either block or stimulate therapeutic targets of interest to help fight diseases across a broad range of therapeutic areas. Our proprietary technology for engineering these proteins creates an opportunity to rapidly generate compounds for study as potential treatment options for patients. Targeted biologics are one of the most medically important and fastest growing areas in the biopharmaceutical industry. These drugs are specifically designed to attack targets of disease. Despite the success of the first generation targeted therapeutic products, such as antibodies, additional innovations are sought by the medical community to help meet unmet needs for improved safety, and efficacy. We believe the study of Adnectins can help to address these demands. Adnectins are derived from human fibronectin, an abundant extracellular protein that binds naturally to a number of different proteins. We create Adnectins using our proprietary protein engineering system called PROfusion™. Our scientists can rapidly create PROfusion libraries of more than 10 trillion different Adnectins. We then screen these libraries to identify Adnectins with the desired drug properties against therapeutic targets of interest. PROfusion enables discovery productivity by minimizing the timeframe for identifying high-quality candidates. In October 2007, Adnexus was acquired by Bristol-Myers Squibb Company. This acquisition has further accelerated our growth as an industry leader in the discovery and early development of proprietary targeted biologics. Together with Bristol-Myers Squibb, we are developing multiple Adnectin programs for use in the study of potential treatments in a broad range of disease types, including oncology, immunology, and cardiovascular disease.

NovaMed Pharmaceuticals (诺凡麦医药) is a Chinese group based on virtual integration. It is focused on complete business functions such as sales and marketing, register, circulation, manufacture, and R&D. NovaMed Pharmaceuticals was founded by senior management personnel in transnational pharmaceutical companies. The founders are professionals with broad social networks and domestic market knowledge.

Egalet is a specialty pharmaceutical company that develops pain management products. The company is developing a pipeline of abuse-resistant opioids that are nearing pivotal testing. Egalet has pioneered one of the world’s first erosion-based delivery technologies to enable the controlled release of drugs through gradual erosion of a tablet and has optimized the abuse deterrent properties that can be applied to a broad range of opioids and non-opioids.

Nitec is a specialty pharmaceutical company focused on the commercialization and development of medicines to treat chronic inflammation and pain-related diseases. It seeks to address areas of high unmet medical need within these markets by developing proprietary products based on well-characterized pharmaceuticals and disease targets. The Company is a spin-out of Merck KGaA and is headquartered in the Basel region of Switzerland. Since its formation in 2004, Nitec has developed and commercialized its lead product, Lodotra™. Nitecâ€:tm:s most advanced product candidate is Lodotra™, a circadian cytokine modulator, which successfully completed a European phase 3 trial in rheumatoid arthritis (RA) in 2006. In January 2009, Lodotra™ was recommended for European regulatory approval for the treatment of rheumatoid arthritis (“RA“), particularly the associated and debilitating morning stiffness, following the successful completion of a phase III trial (CAPRA-1) in 2006. Germany was the Reference Member State and Lodotra™ is now also expected approvable by the regulatory agencies of 14 other countries under the Decentralized Procedure. In March Nitec announced a European partnership for Lodotraâ€:tm:s commercialization with MundiPharma. Merck KGaA, which holds the distribution rights to Lodotra™ in Germany and Austria, have recently launched the product in Germany. Nitec is conducting a second pivotal phase 3 trial in preparation for the filing of an NDA with the FDA seeking US approval of Lodotra™ for the treatment of the signs and symptoms of RA including morning stiffness. Lodotra™ is also being developed for the treatment of severe asthma and polymyalgia rheumatica (PMR). Nitecâ€:tm:s second product candidate is TruNoc™ (tarenflurbil), which is being developed as a treatment of neuropathic pain and other chronic inflammation and pain-related diseases. Nitec is conducting pharmacokinetic studies in preparation for phase 2 trials in neuropathic pain.

Stromedix is a biopharmaceutical company dedicated to developing therapies for organ failure due to fibrosis. Initially, it will concentrate on chronic indications, utilizing its expertise to remodel tissues affected by this condition. In the future, Stromedix plans to expand its therapeutic pipeline to address related areas, including acute organ failure and cancer. Through its innovative approach, the company aims to provide effective treatments that improve patient outcomes in these challenging medical contexts.

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.

F-Star Therapeutics is a clinical-stage biopharmaceutical company focused on creating innovative bispecific antibody therapies aimed at improving cancer treatment. Utilizing its proprietary Modular Antibody Technology, F-Star develops bispecific antibodies that enhance the capabilities of traditional monoclonal antibodies while minimizing production challenges. The company's unique approach allows for the rapid discovery and development of these therapies, which seek to block tumor immune evasion and provide patients with more effective and durable treatment options compared to existing immuno-oncology therapies. Founded in 2006, F-Star has established a solid patent position and has attracted significant investment from leading venture capital firms and strategic corporate partners. The company has also formed alliances with major pharmaceutical companies, allowing it to expand its research and development efforts. With a dedicated team based in Cambridge, UK, F-Star is advancing a pipeline of product candidates that leverage its innovative technology.

Prestwick Pharmaceuticals develops and markets therapeutic pharmaceutical drugs for chronic central nervous systems diseases. The company offers products for restless legs syndrome, schizophrenia, autism, Alzheimer’s, and sleep apnea ailments. Prestwick Pharmaceuticals was founded in 2002 and is based in Washington, District of Columbia.

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, is leading the discovery and development of Adnectinsâ„¢, a novel, proprietary class of targeted biologics. Adnectins are proteins designed to either block or stimulate therapeutic targets of interest to fight diseases across a broad range of therapeutic areas. Their proprietary technology for engineering these versatile proteins creates an opportunity to rapidly generate highly differentiated medicines. Adnexus, a Bristol-Myers Squibb R&D Company, is a leader in the discovery and development of Adnectins, a proprietary type of targeted biologic. Adnectins are proteins designed to either block or stimulate therapeutic targets of interest to help fight diseases across a broad range of therapeutic areas. Our proprietary technology for engineering these proteins creates an opportunity to rapidly generate compounds for study as potential treatment options for patients. Targeted biologics are one of the most medically important and fastest growing areas in the biopharmaceutical industry. These drugs are specifically designed to attack targets of disease. Despite the success of the first generation targeted therapeutic products, such as antibodies, additional innovations are sought by the medical community to help meet unmet needs for improved safety, and efficacy. We believe the study of Adnectins can help to address these demands. Adnectins are derived from human fibronectin, an abundant extracellular protein that binds naturally to a number of different proteins. We create Adnectins using our proprietary protein engineering system called PROfusion™. Our scientists can rapidly create PROfusion libraries of more than 10 trillion different Adnectins. We then screen these libraries to identify Adnectins with the desired drug properties against therapeutic targets of interest. PROfusion enables discovery productivity by minimizing the timeframe for identifying high-quality candidates. In October 2007, Adnexus was acquired by Bristol-Myers Squibb Company. This acquisition has further accelerated our growth as an industry leader in the discovery and early development of proprietary targeted biologics. Together with Bristol-Myers Squibb, we are developing multiple Adnectin programs for use in the study of potential treatments in a broad range of disease types, including oncology, immunology, and cardiovascular disease.

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.

Nitec is a specialty pharmaceutical company focused on the commercialization and development of medicines to treat chronic inflammation and pain-related diseases. It seeks to address areas of high unmet medical need within these markets by developing proprietary products based on well-characterized pharmaceuticals and disease targets. The Company is a spin-out of Merck KGaA and is headquartered in the Basel region of Switzerland. Since its formation in 2004, Nitec has developed and commercialized its lead product, Lodotra™. Nitecâ€:tm:s most advanced product candidate is Lodotra™, a circadian cytokine modulator, which successfully completed a European phase 3 trial in rheumatoid arthritis (RA) in 2006. In January 2009, Lodotra™ was recommended for European regulatory approval for the treatment of rheumatoid arthritis (“RA“), particularly the associated and debilitating morning stiffness, following the successful completion of a phase III trial (CAPRA-1) in 2006. Germany was the Reference Member State and Lodotra™ is now also expected approvable by the regulatory agencies of 14 other countries under the Decentralized Procedure. In March Nitec announced a European partnership for Lodotraâ€:tm:s commercialization with MundiPharma. Merck KGaA, which holds the distribution rights to Lodotra™ in Germany and Austria, have recently launched the product in Germany. Nitec is conducting a second pivotal phase 3 trial in preparation for the filing of an NDA with the FDA seeking US approval of Lodotra™ for the treatment of the signs and symptoms of RA including morning stiffness. Lodotra™ is also being developed for the treatment of severe asthma and polymyalgia rheumatica (PMR). Nitecâ€:tm:s second product candidate is TruNoc™ (tarenflurbil), which is being developed as a treatment of neuropathic pain and other chronic inflammation and pain-related diseases. Nitec is conducting pharmacokinetic studies in preparation for phase 2 trials in neuropathic pain.