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Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colorado, that specializes in developing small molecule therapies for severe, rare musculoskeletal diseases. Founded in 2017, the company employs a precision medicine approach to create innovative treatments targeting various muscle disorders, including Duchenne and Becker muscular dystrophies, as well as limb girdle muscular dystrophies. Utilizing its proprietary muscle-focused drug discovery platform, Edgewise integrates expertise in muscle biology and small molecule engineering to identify and develop therapies that address genetically defined muscle disorders. The company emphasizes a holistic drug discovery method that assesses integrated muscle function, enabling it to create orally bioavailable therapies aimed at improving outcomes for patients with significant unmet medical needs in neuromuscular and cardiac diseases.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for eye diseases using its proprietary Mucus Penetrating Particles (MPP) technology. This technology enhances drug distribution and pharmacokinetics by coating mucosal surfaces with biocompatible, drug-loaded particles, allowing for improved local drug concentrations while minimizing systemic exposure. Kala's lead product candidate, KPI-121 0.25%, has completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS has also completed two Phase III trials for managing inflammation and pain after ocular surgery. The company is advancing KPI-285, a receptor tyrosine kinase inhibitor, in preclinical studies targeting retinal diseases. Established in 2009 and headquartered in Watertown, Massachusetts, Kala Pharmaceuticals continues to explore diverse product opportunities while focusing on internal development and research collaborations to address significant clinical needs.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

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

AnaptysBio, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on developing therapeutic antibodies for inflammation and immuno-oncology. The company’s key product candidates include Etokimab, an anti-IL-33 treatment for atopic dermatitis, eosinophilic asthma, and chronic rhinosinusitis; ANB019, an anti-IL-36 receptor for generalized pustular psoriasis; and several immune cell modulating antibodies, including checkpoint agonists for autoimmune diseases. AnaptysBio's pipeline features notable immuno-oncology candidates such as TSR-042 (anti-PD-1), TSR-022 (anti-TIM-3), and TSR-075 (anti-PD-1/LAG-3 bispecific). The company employs a proprietary platform based on somatic hypermutation for antibody discovery and optimization, addressing unmet medical needs in therapeutic applications. AnaptysBio has formed partnerships with various organizations, including TESARO and Celgene, to enhance its research and development efforts. Founded in 2005, AnaptysBio has established itself in the biotechnology sector as a developer of innovative therapies targeting significant health challenges.

AltruBio is a biopharmaceutical company focused on developing targeted antibody therapeutics for cancer and immune-related inflammatory diseases. Established in Delaware with its principal office in the San Francisco Bay Area, AltruBio integrates global resources to advance its research and development initiatives. The company has its roots in Taipei, Taiwan, where it has been working since June 2000 on discovering and developing novel therapeutics to address unmet medical needs. AltruBio employs a sophisticated discovery platform to identify and validate new antibody therapeutics. Currently, the company is developing several potential therapeutic antibodies that target a range of conditions, including autoimmune diseases and various types of cancer, such as pancreatic, stomach, colorectal, ovarian, and lung cancers, at different stages of development.

BridgeBio Oncology Therapeutics is a biopharmaceutical company specializing in the development of precision oncology therapeutics. It focuses on targeting RAS-dependent cancers, leveraging a profound understanding of RAS signaling biology to create drugs that selectively inhibit RAS-driven PI3K activation and a wide spectrum of KRAS mutants. The company aims to provide patients with tailored treatments that address both active and inactive cancer-driving states.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cells for the treatment of cancer and autoimmune diseases. The company aims to enhance the efficacy of cell therapy by leveraging the inherent ability of NK cells to identify and eliminate abnormal cells without the need for genetic alteration. This approach allows for a broader application across various hematologic and solid tumor malignancies while potentially minimizing side effects associated with traditional T-cell therapies. Nkarta combines its NK expansion platform technology with proprietary cell engineering methods to produce a substantial supply of NK cells, enhancing their ability to recognize therapeutic targets and improving their persistence in the body for sustained activity. Through this innovative strategy, Nkarta seeks to provide more potent, well-tolerated, and rapidly available therapies for patients facing diverse cancer types.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing selective inhibitors of TYK2 for autoimmune diseases. Founded in 2018, the company focuses on creating innovative therapies for patients suffering from inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, aimed at treating a variety of autoimmune conditions while minimizing the toxicities associated with broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator for ulcerative colitis, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which is implicated in several inflammatory conditions. Through these efforts, Ventyx aims to provide effective treatment options for millions of patients.

BlossomHill Therapeutics, Inc. is a small molecule drug discovery and development company focused on unmet medical needs in oncology and autoimmune disorders.

Synnovation Therapeutics is a precision oncology company dedicated to developing small molecule therapies that target key mechanisms driving cancer. By leveraging expertise in medicinal chemistry, cancer biology, and patient-focused precision medicine, the company aims to create a diverse pipeline of innovative targeted therapies. Its focus on highly validated disease targets allows healthcare providers to address limitations in current treatment standards, ultimately striving to enhance patient outcomes and transform cancer care. Synnovation Therapeutics is committed to advancing these therapies efficiently into clinical trials, with the goal of improving the lives of individuals affected by cancer.

VYNE Therapeutics Inc. is a biopharmaceutical company specializing in the development and commercialization of innovative therapies for dermatological conditions. It offers AMZEEQ, a topical minocycline treatment for non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older. The company is advancing its pipeline with FMX103, currently in Phase III clinical trials for moderate-to-severe papulopustular rosacea in adults, and FCD105, a topical combination foam under investigation in Phase II trials for moderate-to-severe acne vulgaris. Additionally, VYNE is developing Serlopitant, an oral NK1 receptor antagonist aimed at addressing pruritus associated with prurigo nodularis, as well as exploring therapies for other immuno-inflammatory conditions. Formerly known as Menlo Therapeutics Inc., the company rebranded in September 2020 and is headquartered in Bridgewater, New Jersey.

AgomAb Therapeutics N.V., based in Gent, Belgium, specializes in the development of molecular therapies aimed at regenerating damaged tissues. The company focuses on creating agonistic monoclonal antibodies, known as agomAbs, which are designed to stimulate molecular and cellular repair mechanisms. These therapies have the potential to restore organ function in patients suffering from fibrotic, inflammatory, autoimmune, and degenerative diseases. AgomAb targets biologically validated pathways, including Transforming Growth Factor β and Hepatocyte Growth Factor, while employing specialized capabilities in organ-specific small molecules and high-affinity antibodies. The company boasts a diversified clinical pipeline addressing various fibrotic conditions and possesses comprehensive research and development expertise, alongside a strong track record in business development.

Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for estrogen receptor (ER) positive breast cancer. The company specializes in the discovery and commercialization of targeted treatments aimed at improving outcomes for women with cancer. Olema utilizes its extensive knowledge of endocrine-driven cancers and the molecular mechanisms of the ER to create drug candidates that are designed to outperform existing therapies. Currently, the company's product pipeline includes OP-1250 and OP-3136, both of which have completed discovery and preclinical studies. Olema's mission is to transform the standard of care for both pre- and post-menopausal women by providing more effective and convenient treatment options.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing innovative small-molecule therapies for neurological disorders. The company focuses on understanding the intricate biology of neuronal receptor complexes, which are essential for proper neuronal function and signaling. By mapping and targeting these receptor systems, Rapport Therapeutics aims to create precision medicines that can effectively address the unmet needs of patients suffering from central nervous system disorders. Through its foundational research, the company seeks to transform treatment options and improve outcomes for individuals with these challenging conditions.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

BioAtla, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-based therapeutics for cancer treatment. The company utilizes its proprietary platforms, Conditionally Active Biologics (CAB) and Comprehensive Integrated Antibody Optimization (CIAO!), to create highly selective and effective drugs. Its lead product candidate, BA3011, is a conditionally active biologic antibody-drug conjugate targeting soft tissue and bone sarcomas, non-small cell lung cancer, and other tumor types. Additionally, BioAtla is developing BA3021, aimed at non-small cell lung cancer and melanoma, as well as BA3071, which targets multiple cancers, including renal cell carcinoma and hepatocellular carcinoma. The company holds over 150 issued patents and applications, emphasizing its commitment to improving drug selectivity and safety while expanding the range of treatable cancers.

Orionis Biosciences is a biotechnology company focused on early-stage drug discovery and development, particularly targeting oncology and immunotherapies. Headquartered in Waltham, Massachusetts, with additional research facilities in Ghent, Belgium, the company utilizes a range of innovative technologies to create conditionally active drug modalities aimed at treating diseases with significant unmet medical needs. By inducing molecular proximity and cooperativity, Orionis enhances drug potency and precision, allowing for novel target access. Their approach facilitates the development of a diverse pipeline of drug candidates, including therapies that engage both adaptive and innate immune systems. The company collaborates with strategic partners, including VIB in Belgium, and is supported by a team of seasoned entrepreneurs and scientists, as well as a transatlantic network of investors.

MOMA Therapeutics is a biotechnology company based in Cambridge, Massachusetts, founded in 2019. The company focuses on discovering precision medicines by targeting molecular machines that are fundamental to human disease. MOMA Therapeutics utilizes a unique platform that leverages the common features of the ATPase target class, which includes significant conformational changes and energy-driven mechanics. By combining these insights with advancements in structural biology and small-molecule drug discovery, MOMA Therapeutics aims to develop drugs targeting previously challenging enzymes, contributing to the next generation of targeted therapies. The company was formerly known as ATPases NewCo, Inc. before rebranding in March 2020.

MoonLake Immunotherapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for immunologic diseases. The company specializes in leveraging Nanobody technology to create new treatments aimed at addressing inflammatory conditions, particularly in the skin and joints. Its lead investigational product, Sonelokimab, represents a novel approach to managing inflammation, with the potential to significantly improve patient outcomes. Through its research and development efforts, MoonLake aims to advance the understanding and treatment of various inflammatory diseases, contributing to the evolving landscape of biopharmaceuticals.

Star Therapeutics is a biotechnology company focused on developing innovative therapies for a wide range of rare diseases. The company aims to identify diseases that share common biological characteristics and create novel therapeutics that can effectively treat multiple conditions with a single treatment. This approach not only addresses the needs of patients suffering from rare diseases but also presents a unique opportunity for pharmaceutical companies to broaden their therapeutic offerings within a streamlined pipeline. By concentrating on life-changing therapies, Star Therapeutics aspires to make a significant impact on the lives of millions of patients.

Ventus Therapeutics is a biopharmaceutical company focused on discovering and developing novel small molecule medicines aimed at treating autoimmune diseases, inflammatory diseases, and cancer by targeting the innate immune system. The company employs a structural immunology platform that provides deep insights into the mechanisms and molecular structures involved in immune responses. This platform integrates proprietary protein engineering capabilities with advanced rational and structure-based drug design tools, allowing for precise targeting of the innate immune system. Ventus is actively building a pipeline of drug programs that address critical targets within this system, particularly proteins involved in inflammasome and nucleic acid-sensing signaling pathways, thereby enabling the treatment of both acute and chronic inflammatory and immune-related conditions.

Enliven Therapeutics is a clinical-stage precision oncology company based in Boulder, Colorado, focused on developing innovative small molecule therapies to enhance patient outcomes. Established in July 2019 by Sam Kintz, Joe Lyssikatos, and Anish Patel, the company employs a unique discovery process that leverages clinically validated biological targets alongside advanced chemistry. This methodology aims to overcome the limitations of current therapies by creating potentially first-in-class treatments that address critical challenges such as tolerability, combinability, resistance, and disease progression, particularly in cases involving brain metastases. Enliven's product pipeline includes ELVN-001, a selective small molecule kinase inhibitor targeting the BCR-ABL gene fusion in chronic myeloid leukemia, and ELVN-002, an irreversible HER2 inhibitor designed to penetrate the central nervous system and act against various HER2 variants.

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.

Expansion Therapeutics is a drug discovery and development company focused on creating small molecule medicines for RNA-mediated diseases. The company specializes in developing novel RNA-targeted drug candidates that have the potential to treat a wide range of severe health conditions. By leveraging its innovative technology, Expansion Therapeutics aims to provide effective treatment options for patients suffering from these challenging diseases.

BioAtla, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-based therapeutics for cancer treatment. The company utilizes its proprietary platforms, Conditionally Active Biologics (CAB) and Comprehensive Integrated Antibody Optimization (CIAO!), to create highly selective and effective drugs. Its lead product candidate, BA3011, is a conditionally active biologic antibody-drug conjugate targeting soft tissue and bone sarcomas, non-small cell lung cancer, and other tumor types. Additionally, BioAtla is developing BA3021, aimed at non-small cell lung cancer and melanoma, as well as BA3071, which targets multiple cancers, including renal cell carcinoma and hepatocellular carcinoma. The company holds over 150 issued patents and applications, emphasizing its commitment to improving drug selectivity and safety while expanding the range of treatable cancers.

858 Therapeutics is a biotechnology company dedicated to the discovery of small molecule therapeutics aimed at treating cancers and other diseases, particularly those resistant to existing therapies. The company employs innovative strategies in areas such as synthetic lethality, innate immunity, and RNA modulation to develop its therapeutic candidates. Its lead program focuses on a PARG inhibitor, which is currently in early clinical development. By leveraging its platform to investigate the role of RNA modifying proteins in disease biology, 858 Therapeutics is generating a pipeline of targeted small molecules that address critical unmet medical needs in oncology and immunology.

Umoja Biopharma specializes in innovative immunotherapy aimed at transforming cancer treatment and enhancing patient quality of life. The company focuses on reprogramming T cells within the patient's body to effectively target cancer, including solid tumors and hematological cancers that often have poor responses to conventional therapies. Its proprietary CAR T-cell gene therapy platform is designed to deliver scalable therapeutic regimens tailored to various stages of cancer, enabling medical practitioners to directly and safely attack cancer cells. By harnessing the body's immune response, Umoja Biopharma seeks to improve overall patient outcomes and provide more effective treatment options for those affected by cancer.

Elpiscience Biopharmaceuticals Co., Ltd. is a clinical-stage immunology company based in Pudong, China, with an additional office in Suzhou. The company specializes in developing next-generation immunotherapies for cancer treatment, focusing on a diverse pipeline of innovative molecules that target various areas within immuno-oncology. Founded by experienced leaders and scientists in the biopharmaceutical industry, Elpiscience aims to advance at least one new therapeutic candidate into clinical trials each year, striving to enhance treatment options for cancer patients globally.

BlissBio is a biopharmaceutical company dedicated to the development of innovative therapies for cancer treatment. The company specializes in tumor targeting and immunotherapy, focusing on the creation of antibody-drug conjugates and site-specific conjugate technologies. With a robust pipeline of products, BlissBio possesses independent intellectual property rights that support the advancement and commercialization of its anti-tumor therapies, aiming to provide effective solutions for patients battling cancer.

GH Research PLC is a clinical-stage biopharmaceutical company that aims to transform the treatment of psychiatric and neurological disorders. The company is primarily focused on developing novel and proprietary therapies based on 5-MeO-DMT, a compound known as Mebufotenin, to address the needs of patients suffering from Treatment-Resistant Depression (TRD). Through its innovative approach, GH Research seeks to provide new therapeutic options for individuals who have not responded to conventional treatments.

Ventus Therapeutics is a biopharmaceutical company focused on discovering and developing novel small molecule medicines aimed at treating autoimmune diseases, inflammatory diseases, and cancer by targeting the innate immune system. The company employs a structural immunology platform that provides deep insights into the mechanisms and molecular structures involved in immune responses. This platform integrates proprietary protein engineering capabilities with advanced rational and structure-based drug design tools, allowing for precise targeting of the innate immune system. Ventus is actively building a pipeline of drug programs that address critical targets within this system, particularly proteins involved in inflammasome and nucleic acid-sensing signaling pathways, thereby enabling the treatment of both acute and chronic inflammatory and immune-related conditions.

Tyra Biosciences is a clinical-stage biotechnology company based in Carlsbad, California, that specializes in developing small molecule therapies for cancer treatment. Founded in 2018, the company aims to address drug resistance in cancer therapies through its proprietary SNÅP platform, which facilitates rapid and precise drug design by generating iterative molecular snapshots. Tyra is particularly focused on creating selective inhibitors of the Fibroblast Growth Factor Receptor (FGFR) family, which are implicated in approximately 7% of all cancers. The company's lead product candidate, TYRA-300, specifically targets FGFR3 and is initially aimed at patients with metastatic urothelial carcinoma of the bladder and urinary tract. Through its innovative approach, Tyra Biosciences seeks to improve treatment outcomes for cancer patients by providing alternatives to existing therapies.

EpimAb Biotherapeutics is a biopharmaceutical company based in Shanghai, China, focused on the research and development of novel bispecific antibody therapeutics. Utilizing its proprietary Fabs-In-Tandem Immunoglobulin (FIT-Ig) platform, the company creates bispecific molecules that possess antibody-like properties. This innovative approach aims to address the challenges associated with developing effective bi-specific antibodies, which are engineered from two different monoclonal antibodies to combine their therapeutic features into a single entity. These bispecific antibodies hold promise for treating various life-threatening conditions, particularly in immuno-oncology, by offering new therapeutic options. Despite the potential benefits of bi-specific antibodies, many candidates face hurdles in development due to issues related to their binding properties, pharmacokinetics, and production costs. EpimAb Biotherapeutics is positioned to contribute to the growing need for advanced bi-specific therapeutics in the pharmaceutical industry.

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Neurelis is a specialty pharmaceutical company based in Encinitas, California, focused on addressing unmet medical needs in epilepsy and the broader central nervous system market. Established in 2007, the company develops and commercializes product candidates utilizing innovative technologies to improve therapeutic benefits and patient care. Neurelis leverages its expertise in neuroscience to enhance drug delivery mechanisms, enabling the effective administration of a wide range of therapeutic agents, including proteins, peptides, and both large and small molecules. Through its differentiated approach, Neurelis aims to provide solutions that significantly improve treatment outcomes for patients suffering from epilepsy and related conditions.

AgomAb Therapeutics N.V., based in Gent, Belgium, specializes in the development of molecular therapies aimed at regenerating damaged tissues. The company focuses on creating agonistic monoclonal antibodies, known as agomAbs, which are designed to stimulate molecular and cellular repair mechanisms. These therapies have the potential to restore organ function in patients suffering from fibrotic, inflammatory, autoimmune, and degenerative diseases. AgomAb targets biologically validated pathways, including Transforming Growth Factor β and Hepatocyte Growth Factor, while employing specialized capabilities in organ-specific small molecules and high-affinity antibodies. The company boasts a diversified clinical pipeline addressing various fibrotic conditions and possesses comprehensive research and development expertise, alongside a strong track record in business development.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to the discovery and development of innovative cancer therapies. The company specializes in creating less toxic kinase inhibitor drugs and epigenetic therapies, including selective inhibitors of histone deacetylases. Its lead candidate, OKI-179, has demonstrated significant efficacy in preclinical models of hematological and solid tumor cancers, effectively reducing tumor growth both alone and in combination with other targeted therapies. Additionally, OnKure is advancing its HDAC3 selective inhibitor, OKI-422, into preclinical toxicology studies. The company's drug candidates are designed to directly target malignant cells, enhance the effects of small molecule therapies, and optimize the tumor microenvironment for improved checkpoint inhibition, addressing unmet medical needs in cancer treatment.

BlossomHill Therapeutics, Inc. is a small molecule drug discovery and development company focused on unmet medical needs in oncology and autoimmune disorders.

DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics for patients with rare and chronic diseases. The company specializes in RNA-based therapeutics, utilizing a delivery technology platform that enhances the distribution of nucleic acid drugs to various tissues and organ systems beyond the liver. This innovative approach addresses limitations associated with previous-generation RNA delivery methods, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's products target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017 and based in San Diego, California, DTx Pharma aims to provide personalized treatment options across multiple therapeutic areas.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

Visen Pharmaceuticals is a biopharmaceutical company that specializes in the development and commercialization of innovative therapies for endocrine diseases. The company is dedicated to addressing significant unmet medical needs in this field, particularly within the Chinese market, and aims to provide both first-in-class and best-in-class treatment solutions for patients of all ages, including those with rare endocrine conditions. With a commitment to patient-centric care, Visen strives to improve treatment outcomes and quality of life for individuals affected by these diseases. The company is backed by a team of experienced professionals with extensive backgrounds in the pharmaceutical industry and operates offices in major Chinese cities such as Shanghai, Beijing, Hong Kong, and Taipei. To further enhance its research, development, and manufacturing capabilities, Visen has established a Greater China research and development site in Suzhou, aiming to deliver advanced treatment options to Chinese patients more effectively.

Biomea Fusion is a preclinical-stage biopharmaceutical company dedicated to advancing precision medicine for cancer treatment. The company's focus is on the discovery and development of irreversible small molecule drugs that target specific genetic alterations associated with tumor growth. Its lead product candidate, BMF-219, is designed as a potent and selective irreversible inhibitor of menin, a key transcriptional regulator involved in oncogenic signaling across various cancers. Biomea Fusion aims to expedite the development of these innovative therapies to provide highly effective treatment options for patients with genetically defined cancers.

Immuneering Corporation is a biopharmaceutical company specializing in genetic, genomic, and proteomic data analysis services for pharmaceutical companies. It focuses on generating biological insights that enhance the clinical and commercial success of medicines, particularly for patients with cancer, autoimmune diseases, and neurodegenerative conditions. Immuneering employs advanced methodologies such as microarray analysis, single-nucleotide polymorphism assessment, and next-generation sequencing to uncover difficult-to-find signals and formulate biological hypotheses. The company has over a decade of experience in translational bioinformatics, which informs its drug discovery programs and supports patient treatment response analysis. Immuneering's proprietary computational platform, known as Disease Cancelling Technology, underpins its capabilities, allowing for a comprehensive approach to drug development. Founded in 2008 and headquartered in Cambridge, Massachusetts, with an additional office in New York City, Immuneering operates as a subsidiary of Teva Pharmaceutical Industries Limited.

Pharvaris is a clinical-stage company dedicated to the discovery and development of therapies for rare diseases, particularly focusing on hereditary angioedema (HAE) and other conditions mediated by bradykinin. The company is advancing an oral bradykinin B2 receptor antagonist, with its lead candidate, PHA121, currently undergoing Phase 1 clinical trials. Pharvaris aims to provide new treatment options that offer alternatives to injected therapies for all sub-types of HAE. The company was co-founded by key individuals involved in the development of icatibant, a previously established treatment for HAE, ensuring a strong foundation in this therapeutic area.

Ambrx, Inc. is a clinical-stage biopharmaceutical company based in La Jolla, California, that specializes in the development of engineered precision biologics using an advanced genetic code technology platform. This innovative approach enables the incorporation of synthetic amino acids into proteins, leading to the creation of next-generation therapeutics, including antibody drug conjugates (ADCs), bispecific antibodies, and long-acting therapeutic peptides. Ambrx's development pipeline features several product candidates, such as ARX788, ARX517, and ARX305, which target various therapeutic areas, including oncology, metabolic, and cardiovascular diseases. The company collaborates with organizations like MabSpace Biosciences, BeiGene, and NovoCodex Biopharmaceuticals to enhance its research and development efforts. Founded in 2003, Ambrx aims to optimize the efficacy, safety, and usability of its biopharmaceutical products.

Longboard Pharmaceuticals is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative medicines for neurological diseases. The company's proprietary pipeline includes several product candidates, notably LP352, a next-generation 5-HT2C agonist intended for treating developmental and epileptic encephalopathies. Additionally, Longboard is evaluating LP143, a full agonist of the cannabinoid type 2 receptor, which targets conditions such as amyotrophic lateral sclerosis and Parkinson's disease, as well as LP659, a selective modulator for multiple central nervous system neuroinflammatory disorders. Longboard Pharmaceuticals aims to optimize pharmacology and pharmacokinetics across its drug candidates, enhancing therapeutic outcomes for patients with rare neurological conditions. The company was incorporated in 2020 and was previously known as Arena Neuroscience, Inc.

Flame Biosciences, Inc. is a clinical-stage biotechnology company based in New York, dedicated to the research, development, and commercialization of innovative therapies targeting cancer and inflammatory diseases. The company is focused on creating transformative treatments, including FL-101, which addresses lung cancers associated with inflammation, and FL-201, aimed at reducing chronic inflammation in patients. Established in 2017, Flame Biosciences seeks to improve the quality of life for individuals suffering from debilitating and life-threatening conditions through its advanced therapeutic solutions.

BioShin Limited is a clinical-stage biopharmaceutical company based in Pudong, China, focused on developing innovative medicines and therapies for neurological, neuropsychiatric diseases, and rare disorders. Established in 2018, it operates as a wholly owned subsidiary of Biohaven Pharmaceuticals. The company specializes in late-stage product candidates and utilizes clinical trial platforms to explore treatments such as calcitonin gene-related peptide (CGRP) receptor antagonism for migraine and pain, glutamate modulation for Alzheimer’s disease and anxiety disorder, and myeloperoxidase (MPO) inhibition for inflammation and neurodegeneration. BioShin aims to address unmet medical needs in the neurological field, particularly within the Asia-Pacific market.

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

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

I-Mab Biopharma (Shanghai) Co., Ltd. is a clinical-stage biopharmaceutical company based in Shanghai, China, with additional offices in Beijing and Rockville, Maryland. Founded in 2016, the company focuses on the discovery, development, and commercialization of innovative biologics aimed at addressing significant unmet medical needs, particularly in the fields of immuno-oncology and immuno-inflammation. I-Mab's diverse drug pipeline targets various therapeutic areas, including multiple myeloma, autoimmune diseases, pediatric growth hormone deficiency, and several cancer indications such as head and neck cancer. Key candidates in its portfolio include Uliledlimab, an antibody for solid tumors, along with Ragistomig and Givastomig, highlighting the company's commitment to developing transformative therapies for patients.

Rain Oncology Inc. is a late-stage precision oncology company focused on developing therapies that target oncogenic drivers. The company employs a tumor-agnostic approach, selecting patients based on the underlying genetics of their tumors rather than their histological characteristics. Its leading product candidate, milademetan, is an oral small molecule inhibitor of MDM2, which plays a role in various cancers. In addition to milademetan, Rain Oncology is advancing a preclinical program aimed at inducing synthetic lethality in cancer cells through the inhibition of RAD52. This targeted strategy aims to improve treatment outcomes for patients who are genetically predisposed to benefit from these therapies.

Tango Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative cancer therapies through the principle of synthetic lethality. Established in 2014, the company aims to discover and target specific driver genes involved in cancer progression. Its research concentrates on three main areas: counteracting the loss of tumor suppressor genes, reversing cancer cells' ability to evade the immune system, and identifying effective drug combinations that outperform single-agent therapies. Tango's pipeline includes TNG908, a selective inhibitor of PRMT5 designed for cancer cells with an MTAP deletion, alongside other discovery programs like TNG462, TNG348, and TNG260, which target various cancer types with limited treatment options.

Praxis Precision Medicines is a clinical-stage biopharmaceutical company focused on developing therapies for central nervous system disorders marked by neuronal imbalance. Established in 2015 and headquartered in Cambridge, Massachusetts, the company leverages genetic insights to create innovative treatments for neurological and psychiatric conditions. Its leading product candidates include PRAX-114, an allosteric modulator targeting GABAA receptors, currently undergoing Phase IIa trials for major depressive disorder and perimenopausal depression, and PRAX-944, a selective inhibitor of T-type calcium channels in Phase IIa trials for essential tremor. Additionally, Praxis is advancing PRAX-562, a sodium current blocker in Phase I trials for severe pediatric epilepsy and adult cephalgia, along with PRAX-222, an antisense oligonucleotide aimed at treating patients with SCN2A epilepsy, and a program focused on KCNT1-related epilepsy. The company has established collaborations and licensing agreements with various organizations to enhance its research and development efforts.

Olema Oncology is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for estrogen receptor (ER) positive breast cancer. The company specializes in the discovery and commercialization of targeted treatments aimed at improving outcomes for women with cancer. Olema utilizes its extensive knowledge of endocrine-driven cancers and the molecular mechanisms of the ER to create drug candidates that are designed to outperform existing therapies. Currently, the company's product pipeline includes OP-1250 and OP-3136, both of which have completed discovery and preclinical studies. Olema's mission is to transform the standard of care for both pre- and post-menopausal women by providing more effective and convenient treatment options.

BioAtla, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-based therapeutics for cancer treatment. The company utilizes its proprietary platforms, Conditionally Active Biologics (CAB) and Comprehensive Integrated Antibody Optimization (CIAO!), to create highly selective and effective drugs. Its lead product candidate, BA3011, is a conditionally active biologic antibody-drug conjugate targeting soft tissue and bone sarcomas, non-small cell lung cancer, and other tumor types. Additionally, BioAtla is developing BA3021, aimed at non-small cell lung cancer and melanoma, as well as BA3071, which targets multiple cancers, including renal cell carcinoma and hepatocellular carcinoma. The company holds over 150 issued patents and applications, emphasizing its commitment to improving drug selectivity and safety while expanding the range of treatable cancers.

New Horizon Health is a biotechnology company based in Hangzhou, China, with an additional office in Beijing, specializing in the development of innovative technologies for early cancer detection and screening. Founded in 2015, the company primarily focuses on gastrointestinal cancers, offering products such as the ColoClear and Pupu Tube, which utilize fecal gene analysis (FIT-DNA) technology for colorectal cancer screening. These products enable users to conduct in-home tests, allowing for the early detection of colon cancer and precancerous lesions that may not be identified through traditional blood tests. New Horizon Health aims to reduce cancer morbidity and mortality rates by promoting accessible and efficient health services, leveraging big data and artificial intelligence in its screening technologies. The company's commitment to early cancer screening reflects its mission to enhance public health by facilitating widespread access to vital diagnostic tools.

Atea Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that focuses on discovering and developing antiviral therapies for severe viral infections. The company's lead candidate, AT-527, is an antiviral agent currently undergoing phase 3 clinical trials for the treatment of COVID-19, caused by SARS-CoV-2. Additionally, Atea is developing several other product candidates, including AT-787, which is in phase 2 trials for hepatitis C, and AT-752 for dengue. Other candidates, such as AT-889 and AT-934, are also in phase 2 clinical trials targeting respiratory syncytial virus. Established in 2014, Atea Pharmaceuticals aims to improve treatment options for patients suffering from serious viral infections.

Erasca, Inc. is a clinical-stage precision oncology company based in San Diego, California, focused on discovering and developing therapies for cancers driven by the RAS/MAPK pathway. Founded in 2018, the company utilizes its artificial intelligence-driven oncology pattern recognition algorithm (OPRA) to analyze extensive datasets and uncover novel tumor biology, which aids in the identification of key oncogenes. Erasca's drug development pipeline includes Naporafenib, a pan-RAF inhibitor with potential applications in NRAS mutant melanoma and other RAS/MAPK pathway-driven tumors, as well as ERAS-007, an oral ERK1/2 inhibitor, and ERAS-601, an oral SHP2 inhibitor. The company aims to provide precision oncology options through collaborations with academic and biopharmaceutical partners, ultimately seeking to offer innovative treatment solutions for patients.

MOMA Therapeutics is a biotechnology company based in Cambridge, Massachusetts, founded in 2019. The company focuses on discovering precision medicines by targeting molecular machines that are fundamental to human disease. MOMA Therapeutics utilizes a unique platform that leverages the common features of the ATPase target class, which includes significant conformational changes and energy-driven mechanics. By combining these insights with advancements in structural biology and small-molecule drug discovery, MOMA Therapeutics aims to develop drugs targeting previously challenging enzymes, contributing to the next generation of targeted therapies. The company was formerly known as ATPases NewCo, Inc. before rebranding in March 2020.

Tango Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative cancer therapies through the principle of synthetic lethality. Established in 2014, the company aims to discover and target specific driver genes involved in cancer progression. Its research concentrates on three main areas: counteracting the loss of tumor suppressor genes, reversing cancer cells' ability to evade the immune system, and identifying effective drug combinations that outperform single-agent therapies. Tango's pipeline includes TNG908, a selective inhibitor of PRMT5 designed for cancer cells with an MTAP deletion, alongside other discovery programs like TNG462, TNG348, and TNG260, which target various cancer types with limited treatment options.

Aligos Therapeutics, Inc. is a clinical-stage biopharmaceutical company founded in 2018 and headquartered in South San Francisco, California. The company specializes in developing innovative therapeutics to address unmet medical needs in viral and liver diseases, particularly targeting chronic hepatitis B (CHB) and non-alcoholic steatohepatitis (NASH). Its lead drug candidate, ALG-010133, is a synthetic oligonucleotide currently undergoing Phase I clinical trials for CHB. Aligos is also advancing several other candidates, including ALG-000184, a capsid assembly modulator for CHB; ALG-020572, an oligonucleotide for CHB; ALG-125097, an siRNA drug candidate for CHB; and ALG-055009, a small molecule THR-ß agonist for NASH. The company's strategy leverages the extensive expertise of its team in liver disease and viral hepatitis to develop targeted antiviral therapies and expedite its drug development pipeline.

Forma Therapeutics, a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, specializes in developing and commercializing novel therapeutics for rare hematologic diseases and cancers. Its pipeline includes FT-4202 in Phase 1 trials for sickle cell disease, FT-7051 for metastatic castration-resistant prostate cancer, FT-2102 in Phase 2 trials for relapsed/refractory AML and exploratory Phase 1 trials for glioma, and selective fatty acid synthase inhibitors FT-4101 and FT-8225. Forma's approach integrates innovative drug discovery technologies and oncology expertise to target high-value, challenging drug targets validated through genomic medicine.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

Viela Bio, Inc. is a clinical-stage biotechnology company based in Gaithersburg, Maryland, focused on the research and development of innovative treatments for severe inflammation and autoimmune diseases. The company's lead product candidate, inebilizumab, is a humanized monoclonal antibody targeting CD19, designed for conditions such as neuromyelitis optica spectrum disorder, kidney transplant desensitization, myasthenia gravis, and IgG4-related diseases. In addition to inebilizumab, Viela Bio is developing VIB4920, aimed at preventing kidney transplant rejection and treating Sjögren's syndrome, and VIB7734, which targets cutaneous lupus erythematosus. The company has formed a strategic partnership with Mitsubishi Tanabe Pharma Corporation to develop and commercialize inebilizumab for autoimmune diseases across several countries in Asia. Founded in 2017, Viela Bio is committed to addressing critical pathways underlying autoimmune diseases to improve patient outcomes.

Erasca, Inc. is a clinical-stage precision oncology company based in San Diego, California, focused on discovering and developing therapies for cancers driven by the RAS/MAPK pathway. Founded in 2018, the company utilizes its artificial intelligence-driven oncology pattern recognition algorithm (OPRA) to analyze extensive datasets and uncover novel tumor biology, which aids in the identification of key oncogenes. Erasca's drug development pipeline includes Naporafenib, a pan-RAF inhibitor with potential applications in NRAS mutant melanoma and other RAS/MAPK pathway-driven tumors, as well as ERAS-007, an oral ERK1/2 inhibitor, and ERAS-601, an oral SHP2 inhibitor. The company aims to provide precision oncology options through collaborations with academic and biopharmaceutical partners, ultimately seeking to offer innovative treatment solutions for patients.

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.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company headquartered in Bedford, Massachusetts, focused on developing novel antisense oligonucleotide medicines to address the underlying causes of severe genetic diseases. The company employs its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology to enhance gene expression by targeting RNA splicing, thereby selectively restoring protein levels. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a serious and progressive genetic epilepsy. Established in 2014, Stoke Therapeutics also collaborates with Invitae Corporation to provide genetic testing services, further supporting its mission to advance gene-targeted therapies for patients with severe genetic conditions.

Turning Point Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative small molecule targeted therapies for oncology. The company designs a range of tyrosine kinase inhibitors (TKIs) aimed at addressing genetic drivers of cancer in both TKI-naïve and TKI-pretreated patients. Its lead drug candidate, repotrectinib, is currently under evaluation in the ongoing Phase I/II TRIDENT-1 trial, targeting patients with ROS1+ advanced non-small-cell lung cancer and those with ROS1+, NTRK+, or ALK+ advanced solid tumors. Additionally, Turning Point's pipeline includes other multi-targeted drug candidates, such as TPX-0046, a RET/SRC inhibitor; TPX-0022, a MET/CSF1R/SRC inhibitor; and TPX-0131, which is an ALK inhibitor in preclinical development. Founded in 2013 and headquartered in San Diego, California, the company aims to advance precision medicine in oncology by addressing unmet medical needs.

Atreca, Inc. is a biopharmaceutical company based in South San Francisco, California, focused on discovering and developing antibody-based immunotherapeutics for various solid tumor types. Its lead product candidate, ATRC-101, is a monoclonal antibody currently in clinical development, characterized by a novel mechanism of action. ATRC-101 has demonstrated reactivity in vitro with a significant majority of human cancer samples, including ovarian, non-small cell lung, colorectal, and breast cancers. Atreca employs a unique technology to identify antibodies produced during immune responses without prior knowledge of antigens. The company collaborates strategically with Merck Sharp & Dohme Corp. to pinpoint antigenic targets of selected antibodies that may have applications in oncology. Founded in 2010, Atreca is dedicated to advancing innovative treatments for cancer.

PhaseBio Pharmaceuticals, Inc., established in 2002 and based in Malvern, Pennsylvania, is a clinical-stage biopharmaceutical company dedicated to developing novel therapies for cardiopulmonary diseases. Its primary focus is on PB2452, a reversal agent for the antiplatelet drug ticagrelor, currently in Phase III trials for managing uncontrolled bleeding events or urgent surgeries. Additionally, PhaseBio is developing PB1046, a fusion protein in Phase IIb trials for treating pulmonary arterial hypertension, and PB6440, an oral agent for resistant hypertension. The company's proprietary technology, elastin-like polypeptides (ELPs), enhances the stability, bioavailability, and efficacy of proteins and peptides, aiming to improve patient outcomes and reduce side effects.

Innovent Biologics, Inc. is a prominent biopharmaceutical company based in Suzhou, China, specializing in the discovery, development, manufacturing, and commercialization of monoclonal antibodies. The company focuses on therapeutic areas such as oncology, ophthalmology, immunology, and metabolic diseases. Its flagship product, Tyvyt (sintilimab), is an anti-PD-1 monoclonal antibody approved for treating various cancers, including Hodgkin's lymphoma and esophageal carcinoma. Innovent's pipeline includes several important candidates, such as IBI-301, a rituximab biosimilar for non-Hodgkin's lymphoma, and IBI-303, an adalimumab biosimilar for autoimmune diseases. The company is also developing innovative therapies for conditions like hypercholesterolemia and various cancers. With a robust portfolio of commercialized products and ongoing strategic collaborations with major pharmaceutical companies, Innovent aims to expand its impact in the global biopharmaceutical landscape. Founded in 2011, Innovent is recognized as one of China's leading biotechnology firms.

Corvidia Therapeutics, Inc. is a biotechnology company based in Waltham, Massachusetts, specializing in the development of precision cardiovascular therapies. Founded in 2014, the company focuses on researching and commercializing innovative treatments for cardio-renal diseases. Corvidia is particularly engaged in developing therapies for chronic kidney disease associated with atherosclerotic cardiovascular disease, inflammation, and high triglyceride-induced acute pancreatitis. As of July 2020, Corvidia operates as a subsidiary of Novo Nordisk A/S, leveraging its expertise to advance its clinical-stage programs aimed at addressing specific biological pathways in patients with unique sensitivities.

Eidos Therapeutics, Inc. is a clinical stage biopharmaceutical company based in San Francisco, California, that specializes in developing treatments for diseases related to transthyretin (TTR) amyloidosis. The company is advancing AG10, an orally administered small molecule currently in phase 3 clinical trials, which aims to stabilize tetrameric TTR and interrupt the molecular processes leading to amyloidosis. Founded in 2013, Eidos Therapeutics operates as a subsidiary of BridgeBio Pharma, which focuses on creating genetically targeted therapies designed to address the underlying causes of various diseases. Through its innovative approach, Eidos Therapeutics seeks to provide potentially disease-modifying therapies to meet the significant medical needs associated with TTR-related conditions.

Kiniksa Pharmaceuticals, Ltd. is a clinical-stage biopharmaceutical company dedicated to discovering, acquiring, developing, and commercializing therapeutic medicines for patients with debilitating diseases that have significant unmet medical needs. The company has a diverse pipeline of clinical-stage product candidates, including Rilonacept, which is undergoing Phase III trials for recurrent pericarditis; Mavrilimumab, a monoclonal antibody in Phase II trials for giant cell arteritis; and KPL-716, currently in Phase 2a trials for prurigo nodularis and Phase 1a trials for atopic dermatitis. Kiniksa also has preclinical candidates, such as KPL-404, which targets the CD40/CD40L interaction in immune responses. Additionally, the company is collaborating with Kite Pharma to explore the combination of Yescarta and Mavrilimumab in patients with relapsed or refractory Large B-Cell lymphoma. Founded in 2015 and headquartered in Hamilton, Bermuda, Kiniksa Pharmaceuticals aims to address multiple autoimmune and autoinflammatory conditions through its innovative approaches.

X4 Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of innovative therapeutics for primary immune deficiencies and cancer. The company's lead drug candidate, mavorixafor, is an oral small molecule antagonist of the chemokine receptor CXCR4, currently undergoing multiple clinical trials. These include a Phase III trial for patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome, as well as Phase Ib and Phase IIa trials focused on severe congenital neutropenia and clear cell renal cell carcinoma, respectively. In addition, X4 Pharmaceuticals is advancing X4P-002, targeting glioblastoma multiforme, and X4P-003 aimed at primary immune deficiencies. The company has also established a license agreement with Abbisko Therapeutics to develop mavorixafor in combination with checkpoint inhibitors for oncology applications. Founded in 2014, X4 Pharmaceuticals was previously known as Arsanis, Inc. before rebranding in March 2019.

Tricida, Inc. is a late-stage pharmaceutical company based in South San Francisco, California, dedicated to the development and commercialization of its lead drug candidate, TRC101. This non-absorbed, orally-administered polymer is designed to treat metabolic acidosis, a condition often associated with chronic kidney disease (CKD). Metabolic acidosis can contribute to the progression of CKD and is linked to various health issues such as muscle wasting, loss of bone density, and increased mortality risk. Tricida has successfully completed a Phase 3, double-blind, placebo-controlled trial of TRC101, aiming to provide an effective treatment option for patients suffering from this condition. The company was founded in 2013 and is focused on addressing the unmet medical needs of individuals with CKD.

InflaRx is a clinical-stage biopharmaceutical company based in Jena, Germany, that focuses on developing innovative therapeutics for acute and chronic inflammation. The company leverages its proprietary anti-C5a/C5aR technology to create potent inhibitors targeting the complement activation factor C5a and its receptor C5aR, which are implicated in various autoimmune and inflammatory diseases. InflaRx's lead product candidate, vilobelimab, is a novel monoclonal antibody administered intravenously that selectively binds to free C5a. It has shown promising disease-modifying activity and tolerability across multiple clinical settings. Founded in December 2017, InflaRx aims to translate scientific discoveries into effective treatments for patients suffering from inflammatory conditions.

Autolus Therapeutics is a biopharmaceutical company based in London, established in 2014 by Tim Funnell and Martin Pule. The company focuses on developing next-generation programmed T-cell therapies aimed at treating cancer. Its clinical-stage pipeline includes several candidates, such as Obe-cel (obecabtagene autoleucel), AUTO1/22, AUTO3, AUTO5, AUTO6, AUTO7, and AUTO8.

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.

Atreca, Inc. is a biopharmaceutical company based in South San Francisco, California, focused on discovering and developing antibody-based immunotherapeutics for various solid tumor types. Its lead product candidate, ATRC-101, is a monoclonal antibody currently in clinical development, characterized by a novel mechanism of action. ATRC-101 has demonstrated reactivity in vitro with a significant majority of human cancer samples, including ovarian, non-small cell lung, colorectal, and breast cancers. Atreca employs a unique technology to identify antibodies produced during immune responses without prior knowledge of antigens. The company collaborates strategically with Merck Sharp & Dohme Corp. to pinpoint antigenic targets of selected antibodies that may have applications in oncology. Founded in 2010, Atreca is dedicated to advancing innovative treatments for cancer.

Apellis Pharmaceuticals Inc., established in 2009, is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts. The company specializes in developing therapeutic compounds that inhibit the complement system, focusing on autoimmune and inflammatory diseases with high unmet needs. Its lead product candidate, pegcetacoplan, is currently in Phase III trials for treating geographic atrophy in age-related macular degeneration and paroxysmal nocturnal hemoglobinuria, and in Phase II trials for various kidney and blood disorders. Additionally, Apellis is developing APL-9 for preventing immune system activation during adeno-associated virus administration. The company collaborates with Swedish Orphan Biovitrum AB (publ) to advance pegcetacoplan for treating multiple rare diseases.

Kezar Life Sciences, Inc. is a clinical-stage biotechnology company specializing in the discovery and development of small molecule therapeutics aimed at addressing unmet medical needs in autoimmunity and cancer. The company's lead candidate, KZR-616, is a selective immunoproteasome inhibitor currently undergoing Phase 2 clinical trials for various autoimmune conditions, including lupus nephritis and autoimmune hemolytic anemia, as well as Phase 1b/2 trials in systemic lupus erythematosus. In addition to KZR-616, the company is developing KZR-TBD, which targets both oncology and autoimmune disorders. Founded in 2015 and based in South San Francisco, California, Kezar Life Sciences leverages insights from protein homeostasis to advance its drug discovery efforts.

Zai Lab Limited is a biopharmaceutical company based in Shanghai that focuses on discovering, developing, and commercializing innovative therapeutics for oncology, autoimmune, and infectious diseases. Founded in 2013, the company has established a diverse pipeline of proprietary drug candidates, including Niraparib for various solid tumors, Ripretinib for KIT and PDGFRa-driven cancers, and Margetuximab for breast and gastric cancers. Zai Lab also develops antibiotics such as Omadacycline and Durlobactam for bacterial infections. The company seeks to address unmet medical needs in China and globally by leveraging partnerships with leading biopharmaceutical firms and utilizing its in-house manufacturing capabilities. Zai Lab aims to establish itself as a fully integrated biopharmaceutical entity, enhancing its drug development efforts through collaborations with academic institutions and expanding its commercial reach in the Chinese market.

Turning Point Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative small molecule targeted therapies for oncology. The company designs a range of tyrosine kinase inhibitors (TKIs) aimed at addressing genetic drivers of cancer in both TKI-naïve and TKI-pretreated patients. Its lead drug candidate, repotrectinib, is currently under evaluation in the ongoing Phase I/II TRIDENT-1 trial, targeting patients with ROS1+ advanced non-small-cell lung cancer and those with ROS1+, NTRK+, or ALK+ advanced solid tumors. Additionally, Turning Point's pipeline includes other multi-targeted drug candidates, such as TPX-0046, a RET/SRC inhibitor; TPX-0022, a MET/CSF1R/SRC inhibitor; and TPX-0131, which is an ALK inhibitor in preclinical development. Founded in 2013 and headquartered in San Diego, California, the company aims to advance precision medicine in oncology by addressing unmet medical needs.

Dicerna Pharmaceuticals is a biotechnology company dedicated to the discovery and development of innovative treatments for rare inherited diseases and other conditions affecting the liver. Utilizing its proprietary ribonucleic acid interference (RNAi) technology, specifically the GalXC platform, Dicerna focuses on silencing disease-causing genes to develop pharmaceuticals for various therapeutic areas, including liver diseases, cardiovascular diseases, and cancers. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and additional programs targeting undisclosed rare liver diseases. The company has established strategic collaborations with several major pharmaceutical firms to enhance its research and development efforts. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna Pharmaceuticals aims to address significant unmet medical needs through its innovative approaches.

Iconic Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutics for retinal diseases and cancer by leveraging tissue factor biology in processes such as angiogenesis and inflammation. The company's lead product, ICON-1, is a fusion protein designed to combat vision loss associated with age-related macular degeneration. Additionally, Iconic Therapeutics has developed hI-con1, a novel recombinant protein that targets tissue factor to activate the immune system, facilitating the destruction of pathological blood vessels linked to conditions like wet age-related macular degeneration and certain cancers. This approach not only promotes cell-mediated cytotoxicity but may also help reduce levels of vascular endothelial growth factor (VEGF), which plays a critical role in these diseases. Phase 1 clinical studies of hI-con1 have shown promising results without significant toxicities, indicating dose-related biological activity. Founded in 2002 and headquartered in South San Francisco, California, Iconic Therapeutics aims to transform scientific insights into effective treatments for serious health conditions.

Ardelyx, Inc. is a biopharmaceutical company specializing in the development and commercialization of innovative oral therapeutics for cardiorenal diseases. Founded in 2007 and headquartered in Fremont, California, the company focuses on addressing significant unmet medical needs through first-in-class medicines. Its lead product candidate, tenapanor, has completed Phase 3 clinical trials for treating irritable bowel syndrome with constipation and hyperphosphatemia in end-stage renal disease patients on dialysis. Additionally, Ardelyx is advancing RDX013, a small molecule potassium secretagogue targeting hyperkalemia, and has other candidates in its pipeline, including RDX002 and RDX009, which are in preclinical development for chronic kidney disease and type 2 diabetes, respectively. Ardelyx employs a proprietary drug discovery platform aimed at creating non-systemic products that target specific transporters and receptors to minimize side effects associated with systemic exposure.

Mersana Therapeutics is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of antibody-drug conjugates (ADCs) for cancer treatment. The company utilizes its proprietary biodegradable polymer platform, Dolaflexin, to create a pipeline of ADC product candidates aimed at addressing oncology indications with significant unmet needs. Its lead candidate, XMT-1536, is currently in Phase I clinical trials targeting NaPi2b for the treatment of ovarian cancer, non-small cell lung cancer, and other rare conditions. Mersana is also advancing additional candidates, including XMT-1660, targeting B7-H4, and XMT-2056, a STING agonist ADC, both designed to improve tolerability and therapeutic outcomes. The company has established strategic partnerships with Merck KGaA and Asana BioSciences to further develop its ADC candidates. Founded in 2005, Mersana Therapeutics aims to deliver innovative treatments to patients through its advanced research and development efforts.

ImmuneXcite is a biopharmaceutical company based in Lexington, Massachusetts, founded in 2007. The company specializes in developing monoclonal antibodies for cancer treatment, utilizing its proprietary mAbXcite platform technology. This innovative approach enhances the efficacy of therapeutic monoclonal antibodies by chemically linking a unique carbohydrate that activates neutrophils, thereby targeting cancer cells for destruction. The research underpinning ImmuneXcite's technology originated from the Whitehead Institute at MIT and has received support from various institutions, including the Massachusetts Technology Transfer Center and the National Cancer Institute. By leveraging both innate and adaptive immune responses, ImmuneXcite aims to create next-generation immune-activating biotherapeutics that selectively treat a wide range of tumor types, ultimately helping to limit tumor growth and metastasis.

Apellis Pharmaceuticals Inc., established in 2009, is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts. The company specializes in developing therapeutic compounds that inhibit the complement system, focusing on autoimmune and inflammatory diseases with high unmet needs. Its lead product candidate, pegcetacoplan, is currently in Phase III trials for treating geographic atrophy in age-related macular degeneration and paroxysmal nocturnal hemoglobinuria, and in Phase II trials for various kidney and blood disorders. Additionally, Apellis is developing APL-9 for preventing immune system activation during adeno-associated virus administration. The company collaborates with Swedish Orphan Biovitrum AB (publ) to advance pegcetacoplan for treating multiple rare diseases.

Iconic Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutics for retinal diseases and cancer by leveraging tissue factor biology in processes such as angiogenesis and inflammation. The company's lead product, ICON-1, is a fusion protein designed to combat vision loss associated with age-related macular degeneration. Additionally, Iconic Therapeutics has developed hI-con1, a novel recombinant protein that targets tissue factor to activate the immune system, facilitating the destruction of pathological blood vessels linked to conditions like wet age-related macular degeneration and certain cancers. This approach not only promotes cell-mediated cytotoxicity but may also help reduce levels of vascular endothelial growth factor (VEGF), which plays a critical role in these diseases. Phase 1 clinical studies of hI-con1 have shown promising results without significant toxicities, indicating dose-related biological activity. Founded in 2002 and headquartered in South San Francisco, California, Iconic Therapeutics aims to transform scientific insights into effective treatments for serious health conditions.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

X4 Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of innovative therapeutics for primary immune deficiencies and cancer. The company's lead drug candidate, mavorixafor, is an oral small molecule antagonist of the chemokine receptor CXCR4, currently undergoing multiple clinical trials. These include a Phase III trial for patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome, as well as Phase Ib and Phase IIa trials focused on severe congenital neutropenia and clear cell renal cell carcinoma, respectively. In addition, X4 Pharmaceuticals is advancing X4P-002, targeting glioblastoma multiforme, and X4P-003 aimed at primary immune deficiencies. The company has also established a license agreement with Abbisko Therapeutics to develop mavorixafor in combination with checkpoint inhibitors for oncology applications. Founded in 2014, X4 Pharmaceuticals was previously known as Arsanis, Inc. before rebranding in March 2019.

Clearside Biomedical, Inc. is a biopharmaceutical company focused on developing treatments for serious eye diseases with the aim of restoring and preserving vision. Founded in 2011 and headquartered in Alpharetta, Georgia, the company specializes in a proprietary ocular microinjection platform that allows for targeted delivery of therapeutic products to specific compartments of the eye. This innovative approach includes the use of the SCS Microinjector for administering therapies directly into the suprachoroidal space, enabling a non-surgical, repeatable procedure. Among its product pipeline, Clearside is developing XIPERE, a triamcinolone acetonide injectable suspension for macular edema, and CLS-AX, an axitinib for suprachoroidal injection. The company is dedicated to advancing pharmacological therapies for sight-threatening conditions by ensuring precise and effective treatment delivery.

Aclaris Therapeutics is a clinical-stage biopharmaceutical company based in the United States, specializing in the development of innovative therapies for dermatological and immuno-inflammatory conditions. Founded by the creators of Vicept Therapeutics, the company is dedicated to addressing significant unmet needs in these fields, focusing on areas where no FDA-approved treatments exist or where treatment options are limited. Aclaris operates primarily in two segments: therapeutics and contract research. The therapeutics segment is engaged in the identification and development of novel drug candidates, while the contract research segment generates revenue through laboratory services. Among its developmental candidates is Zunsemetinib, an oral MK2 inhibitor aimed at treating immuno-inflammatory diseases.

Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.