Perceptive Advisors

Merida Biosciences is a biotechnology company specializing in the development of precision therapies for autoimmune diseases. It employs a protein engineering platform to create novel Fc therapeutics, which selectively target and neutralize specific antibodies, aiming to treat the root cause of antibody-driven diseases with high precision and durability.

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.

Ocuphire Pharma, Inc. is a clinical-stage biopharmaceutical company based in Farmington Hills, Michigan, dedicated to developing and commercializing therapies for various ophthalmic disorders. The company's primary focus is on innovative treatment options for front and back of the eye conditions, with its lead product candidate, Nyxol eye drops, currently in Phase 3 clinical development. Nyxol is a once-daily, preservative-free formulation aimed at addressing issues such as dim light or night vision disturbances, pharmacologically-induced mydriasis, and presbyopia. Additionally, Ocuphire is advancing another candidate, APX3330, a twice-daily oral tablet, which is entering Phase 2 clinical trials to target diabetic retinopathy and diabetic macular edema by inhibiting angiogenesis and inflammation. The company also seeks to explore opportunities for acquiring additional ophthalmic assets and forming strategic partnerships for global commercialization.

Kardigan is a pioneering heart health company dedicated to transforming cardiovascular drug development. It focuses on identifying and matching critical disease drivers with responsive patients from clinical trials, aiming to deliver curative medicines that go beyond symptom management, ultimately providing functional cures for cardiovascular diseases.

RAPT Therapeutics, Inc., established in 2015 and headquartered in South San Francisco, California, is a clinical-stage biopharmaceutical company specializing in immunology-based therapies. The company focuses on developing oral small molecule treatments for patients with unmet needs in oncology and inflammatory diseases. Its lead drug candidates are FLX475, an oral C-C motif chemokine receptor 4 antagonist in Phase 2 clinical trials for various cancer types, and RPT193, which selectively inhibits type 2 T helper cell migration into inflamed tissues. Additionally, RAPT is exploring other targets like general control nonderepressible 2 and hematopoietic progenitor kinase 1 in the discovery phase.

Crescent Biopharma is a biotechnology firm focused on developing novel precision-engineered medicines targeting verified biology to improve care for patients with solid tumors.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies for patients with significant unmet medical needs, particularly in fibrosis, lysosomal storage disorders, and oncology. The company specializes in drugs that target nuclear receptors, transcription factors, and epigenetic modulation. Its primary product candidate, lanifibranor, is being developed for the treatment of non-alcoholic steatohepatitis (NASH), a condition currently lacking approved therapies. Inventiva has established a robust pipeline supported by a proprietary discovery engine, a dedicated research and development facility, and a skilled team with extensive experience in drug development. In addition to advancing lanifibranor, the company is also working on several earlier-stage programs aimed at addressing various medical conditions. All operations are conducted in France.

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.

scPharmaceuticals Inc. is a pharmaceutical company focused on developing and commercializing innovative therapeutic options for patients with serious medical conditions. The company’s lead product candidate, Furoscix, features a proprietary buffered formulation of furosemide delivered via the SmartDose drug delivery system, targeting congestion in patients with decompensated heart failure. In addition to Furoscix, scPharmaceuticals is advancing its product pipeline with scCeftriaxone, an antibiotic for infections from gram-positive and gram-negative organisms, and its scCarbapenem program, aimed at treating infections caused by gram-negative organisms. Founded in 2013 and headquartered in Burlington, Massachusetts, scPharmaceuticals aims to provide a convenient, safe, and effective alternative to intravenous drug administration through subcutaneous delivery, addressing the associated costs and risks. The company collaborates with West Pharmaceutical Services, Inc. to enhance the SmartDose system for Furoscix.

MeiraGTx Holdings is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company focuses on addressing serious medical conditions, primarily targeting disorders related to the eye, salivary gland, and central nervous system. Its research and development efforts leverage a portfolio approach, which includes licensing, acquiring, and developing advanced technologies to support a diverse range of product candidates. Key pipeline products include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, with plans to expand its focus to develop additional gene therapy treatments for various serious diseases in the future.

Mereo Biopharma, established in London in 2015, specializes in acquiring and advancing late-stage drug candidates from larger pharmaceutical companies. These candidates, often overlooked due to resource constraints, target rare and specialized diseases with significant unmet medical needs. Mereo's strategy involves rapidly progressing these assets through clinical development, either by partnering or divesting them, or directly commercializing them, particularly in orphan disease indications. The company combines operational efficiency with financial resources, leveraging a unique partnership with a leading global CRO, ICON, to conduct comprehensive clinical studies. Mereo's focus is on transforming the lives of patients worldwide by developing innovative medicines that can significantly improve their quality of life.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing next-generation CAR T-cell therapies aimed at overcoming cancer treatment resistance and improving patient access to potentially curative options. The company aims to enhance treatment outcomes by addressing the limitations of existing therapies, which currently provide curative benefits to less than half of cancer patients. Its lead program, CRG-022, is a novel CAR T-cell product candidate that targets CD22, a tumor antigen prevalent in many B-cell malignancies. By tackling issues such as limited durability, safety concerns, and supply chain obstacles, Cargo Therapeutics is committed to advancing innovative solutions that make effective cancer treatments more accessible to patients.

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.

Pheon Therapeutics is a London-based company established in 2022, specializing in the development of Antibody-Drug Conjugates (ADCs) aimed at addressing a variety of difficult-to-treat cancers. The company focuses on creating next-generation ADCs, with its lead program targeting a novel antigen that is significantly expressed in solid tumors across multiple hard-to-treat cancer types. By enhancing the therapeutic options available to healthcare providers, Pheon Therapeutics aims to provide effective treatment alternatives for patients whose cancers do not respond to existing therapies.

CinDome Pharma is a Cincinnati-based pharmaceutical company focused on developing treatments for gastrointestinal diseases, particularly chronic gastroparesis, which affects millions of individuals. The company is engaged in the creation of innovative drug therapies, including a novel chemical entity known as CIN-102, which utilizes deuteration to enhance safety, effectiveness, and tolerability. Through its commitment to addressing the challenges posed by chronic gastroparesis, CinDome Pharma aims to improve the quality of life for patients suffering from this condition.

Endeavor BioMedicines is a clinical-stage company based in Solana Beach, California, that focuses on developing innovative treatments for pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). Founded in 2018, the company is working on a small-molecule inhibitor designed to target the underlying causes of this progressive lung disease. The treatment aims to not only halt the progression of IPF but also has the potential to reverse its effects, offering healthcare professionals a new avenue for precision treatments in managing terminal conditions. Endeavor BioMedicines strives to improve patient outcomes and enhance quality of life for those affected by this challenging illness.

Zevra Therapeutics is a development-stage biopharmaceutical company focused on creating new therapies for pain, ADHD, and other central nervous system (CNS) diseases, particularly in the realm of rare diseases. The company is dedicated to developing safer, abuse-resistant opioid pain relievers and employs a proprietary Ligand Activated Therapy (LAT) approach to drug discovery and development. This innovative platform allows Zevra to enhance existing medications and streamline the development process while preserving strong intellectual property rights. By integrating scientific research with patient needs and data-driven strategies, Zevra Therapeutics aims to address the challenges of drug development, delivering transformational therapies for conditions with limited or no treatment options.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative antibody-based medicines aimed at improving outcomes for cancer patients. The company specializes in creating antibody-drug conjugates (ADCs) and monoclonal antibodies (mAbs) to address oncologic diseases that exhibit significant unmet medical needs. TORL BioTherapeutics employs a strategic approach that includes target identification and early discovery work, allowing them to license promising drug programs and develop proprietary therapies with optimized characteristics. Through this pipeline, TORL BioTherapeutics is positioned to advance novel treatments that could significantly enhance patient care in oncology.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

Neurosterix is a biopharmaceutical company focused on developing innovative medicines for neurological disorders through a unique approach known as allosteric modulation. This method aims to address the limitations of traditional treatments, enhancing efficacy, safety, and tolerability for conditions such as schizophrenia and other neuropsychiatric disorders. By leveraging a specialized drug discovery and development platform, Neurosterix aspires to provide improved therapeutic options for underserved patient populations, ultimately aiming to enhance the quality of life for individuals affected by these challenging conditions.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

Crinetics Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company headquartered in San Diego, California, dedicated to the discovery, development, and commercialization of innovative therapeutics for rare endocrine diseases and endocrine-related tumors. The company’s lead product candidate, paltusotine, is an oral selective nonpeptide somatostatin receptor type 2 biased agonist currently undergoing Phase I clinical trials aimed at treating acromegaly and neuroendocrine tumors. In addition to paltusotine, Crinetics is developing several other therapeutics, including CRN01941, an oral selective nonpeptide sst2 biased agonist for neuroendocrine tumors, and an oral somatostatin type 5 receptor agonist for congenital hyperinsulinism. The company is also working on an oral adrenocorticotrophic hormone antagonist for conditions such as Cushing’s syndrome and congenital adrenal hyperplasia. Founded in 2008, Crinetics Pharmaceuticals is focused on leveraging its internal discovery efforts to create effective treatments while utilizing hormonal biomarker endpoints to enhance the efficiency and cost-effectiveness of its clinical studies.

Applied Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New York, focused on developing innovative therapies for serious medical conditions with significant unmet needs. The company's lead product candidate, AT-007, is in phase I/II clinical trials for the treatment of galactosemia, while AT-001 is undergoing phase II trials for diabetic cardiomyopathy and phase I trials for diabetic peripheral neuropathy. Additionally, AT-003 is in phase I trials for diabetic retinopathy. The company also has preclinical candidates, such as AT-104, aimed at treating orphan hematological oncology conditions. Founded in 2016, Applied Therapeutics leverages advancements in technology and regulatory processes to create drugs targeting well-established molecular mechanisms.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Astria Therapeutics is a biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative therapies for patients affected by rare allergic and immunological diseases. The company focuses on creating life-changing treatments, with its lead program, STAR-0215, being a monoclonal antibody designed to inhibit plasma kallikrein, currently in preclinical development for hereditary angioedema. Additionally, Astria is advancing STAR-0310, another monoclonal antibody, which targets the OX40 receptor and is also in preclinical development for atopic dermatitis, a condition characterized by impaired skin barrier function and significant itching. Through its targeted approach, Astria Therapeutics aims to address unmet medical needs in the field of immunology.

Soleno Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to the development and commercialization of innovative therapeutics for rare diseases. The company's primary focus is on its lead candidate, Diazoxide Choline Controlled Release (DCCR), a once-daily oral tablet designed for the treatment of Prader-Willi Syndrome (PWS). DCCR contains diazoxide choline, a choline salt of diazoxide, which is a benzothiadiazine. Currently, the product is being evaluated in a Phase III clinical development program. Established in 1999 and headquartered in Redwood City, California, Soleno Therapeutics was formerly known as Capnia, Inc. before rebranding in May 2017.

Avalyn Pharma, Inc. is a biopharmaceutical company focused on developing therapies for idiopathic pulmonary fibrosis (IPF) and other severe respiratory diseases. The company is advancing inhaled formulations of pirfenidone, specifically AP01, which aims to enhance drug delivery to the lungs while potentially reducing side effects associated with oral treatments. This innovative approach seeks to improve the care and outcomes for patients suffering from these conditions. Established in 2011, Avalyn Pharma operates from its headquarters in Seattle, Washington, and maintains an additional office in San Diego, California. The company was previously known as Genoa Pharmaceuticals, Inc. before rebranding in July 2017.

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.

Forte Biosciences is a biotechnology company specializing in dermatology and biopharmaceuticals. The company's lead product, FB-401, is a topically applied live biotherapeutic designed to treat inflammatory skin diseases, developed in collaboration with the National Institutes of Health and the National Institute of Allergy and Infectious Diseases. Additionally, Forte Biosciences is advancing another product candidate, FB-102, which is a proprietary therapeutic molecule aimed at addressing a range of autoimmune conditions, including graft-versus-host disease, vitiligo, and alopecia areata. The company is currently in the preclinical trial stage for FB-102, reflecting its commitment to developing innovative treatments for dermatological and autoimmune disorders.

AsclepiX Therapeutics, Inc. is a biopharmaceutical company focused on the design and development of innovative treatments for diseases related to abnormal blood vessel growth, specifically through the use of short biomimetic anti-angiogenic and anti-lymphangiogenic peptides. The company's lead candidates, including AXT107, AXT201, AXT301, and AXT501, target conditions such as diabetic macular edema, wet age-related macular degeneration, and macular edema following retinal vein occlusion, which are among the leading causes of blindness. Additionally, AXT201, AXT301, and AXT501 are being explored for their potential in treating various solid tumors. AsclepiX leverages advanced computational biology, bioinformatics, and the latest developments in biomaterials to enhance drug delivery systems, aiming to create long-lasting biodegradable nanoparticles that can effectively deliver its therapeutic peptides. Incorporated in 2011 and based in Baltimore, Maryland, AsclepiX Therapeutics is committed to addressing unmet medical needs in ophthalmology, oncology, and other diseases driven by angiogenesis and lymphangiogenesis.

A-Alpha Bio, Inc. is a biotechnology company based in Seattle, Washington, established in 2017. The company specializes in synthetic biology, offering a platform known as AlphaSeq, which assists pharmaceutical companies in the development of drugs that interact with multiple targets. A-Alpha Bio provides cell-based tools for high-throughput and quantitative measurements of protein interactions, facilitating target discovery, library screening, and preclinical drug characterization. Its proprietary technology enables the mapping of protein-protein interactions at a large scale, allowing for the identification and quantification of interactions among thousands of protein pairs simultaneously. By delivering detailed maps of these interactions, A-Alpha Bio helps researchers identify potential drug targets and understand disease mechanisms, thereby streamlining the therapeutic discovery process and improving the efficiency of drug development. The company comprises a team of experts in synthetic biology, structural biology, and next-generation sequencing, focused on overcoming challenges in the drug development pipeline.

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.

Satellos Bioscience is a private regenerative medicine company dedicated to developing innovative therapeutics aimed at stimulating or restoring muscle regeneration. The company is particularly focused on addressing severe disorders, with a key program centered on creating an oral therapeutic drug for Duchenne muscular dystrophy. This drug is designed to correct the dysregulation identified by Satellos as a fundamental cause of the disease's progressive nature. Through its research and development efforts, Satellos aims to provide effective treatments that enhance muscle regeneration and improve the quality of life for individuals affected by this debilitating condition.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative antibody-based medicines aimed at improving outcomes for cancer patients. The company specializes in creating antibody-drug conjugates (ADCs) and monoclonal antibodies (mAbs) to address oncologic diseases that exhibit significant unmet medical needs. TORL BioTherapeutics employs a strategic approach that includes target identification and early discovery work, allowing them to license promising drug programs and develop proprietary therapies with optimized characteristics. Through this pipeline, TORL BioTherapeutics is positioned to advance novel treatments that could significantly enhance patient care in oncology.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing next-generation CAR T-cell therapies aimed at overcoming cancer treatment resistance and improving patient access to potentially curative options. The company aims to enhance treatment outcomes by addressing the limitations of existing therapies, which currently provide curative benefits to less than half of cancer patients. Its lead program, CRG-022, is a novel CAR T-cell product candidate that targets CD22, a tumor antigen prevalent in many B-cell malignancies. By tackling issues such as limited durability, safety concerns, and supply chain obstacles, Cargo Therapeutics is committed to advancing innovative solutions that make effective cancer treatments more accessible to patients.

Fusion Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company headquartered in Hamilton, Canada, specializing in the development of radiopharmaceuticals as precision medicines for challenging cancers. The company employs its proprietary Targeted Alpha Therapies platform and Fast-Clear linker technology to connect alpha particle-emitting isotopes to antibodies, enabling the selective delivery of therapeutic agents to tumors. Fusion's lead product candidate, FPI-1434, is currently undergoing Phase 1 clinical trials as a monotherapy for solid tumors expressing insulin-like growth factor 1 receptor. Additionally, the company is exploring the use of FPI-1434 in combination with approved checkpoint inhibitors and DNA damage response inhibitors in preclinical studies to enhance anti-tumor activity. Fusion is also advancing another product candidate, FPI-1966, into clinical development for head and neck and bladder cancers that express fibroblast growth factor receptor. Founded in 2014 as a spinout of the Centre for Probe Development and Commercialization, Fusion aims to transform how cancers are treated through innovative radiochemistry techniques.

Apogee Therapeutics is a biotechnology company based in San Francisco, California, founded in 2022. The company focuses on developing differentiated biologics for the treatment of immunological and inflammatory disorders, specifically targeting atopic dermatitis and chronic obstructive pulmonary disease. Apogee Therapeutics aims to address high unmet medical needs by advancing novel therapies that utilize advanced antibody engineering to enhance therapeutic properties such as half-life. The company's key programs, APG777 and APG808, are designed to leverage established mechanisms of action to improve treatment outcomes for patients suffering from these conditions.

Zenas BioPharma is a clinical-stage biopharmaceutical company based in Florida, dedicated to developing immune-based therapies for patients. The company's lead product candidate, obexelimab, is a bifunctional monoclonal antibody that targets both CD19 and FcγRIIb, which are found on B cells. This innovative approach aims to inhibit the activity of B cells involved in various autoimmune diseases while preserving their overall population, thus offering a potential therapeutic advantage over traditional treatments that deplete these cells.

immatics is a biopharmaceutical company focused on the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors. The company's customized Adoptive Cell Therapies (ACTengine) and antibody-like TCR Bispecifics are the two main product classes in the pipeline (TCER). With a focus on particularly difficult-to-treat solid malignancies, each therapy modality has unique characteristics to create the desired therapeutic impact for patients at various disease stages and with various types of tumors. immatics biotechnologies was established in 2000 by Hans Rammensee, Harpreet Singh, and Steffen Walter in Tübingen, Baden-Wurttemberg.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing medicines for underserved patient populations affected by rare diseases and cancer. The company employs a precision medicine approach and has a diverse portfolio of small molecule targeted oncology products. Its lead candidate, nirogacestat, an oral gamma secretase inhibitor, is currently in Phase III clinical trials for desmoid tumors. Additionally, SpringWorks is advancing several other product candidates, including mirdametinib, an oral MEK inhibitor in Phase IIb trials for neurofibromatosis type 1-associated plexiform neurofibromas, and various combinations involving nirogacestat for multiple myeloma. The company has established collaborations with organizations such as BeiGene, GlaxoSmithKline, and Allogene, aimed at enhancing the development of its therapies. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks is committed to unlocking the potential of its pipeline to provide innovative solutions for patients suffering from cancer and rare diseases.

MeiraGTx Holdings is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company focuses on addressing serious medical conditions, primarily targeting disorders related to the eye, salivary gland, and central nervous system. Its research and development efforts leverage a portfolio approach, which includes licensing, acquiring, and developing advanced technologies to support a diverse range of product candidates. Key pipeline products include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, with plans to expand its focus to develop additional gene therapy treatments for various serious diseases in the future.

Cerebral Therapeutics, Inc. is a clinical-stage pharmaceutical company based in Aurora, Colorado, focused on developing implanted drug-device combination therapies for neurologic diseases, particularly refractory epilepsy. The company utilizes a refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose of its formulation of the anti-epileptic drug valproic acid, known as CT-010. This innovative approach aims to bypass the blood-brain barrier, enhancing treatment outcomes for patients with chronic neurological conditions. Founded in 2010, Cerebral Therapeutics is dedicated to improving the lives of individuals affected by uncontrolled neurological diseases.

Surf Bio is a preclinical biopharmaceutical company focused on developing innovative therapeutic solutions using a novel biotechnology platform. This platform centers around a next-generation surfactant designed to enhance drug effectiveness for a variety of medical conditions, including diabetes, oncology, infectious diseases, and gene therapy. By improving therapeutic applications, Surf Bio aims to provide healthcare organizations worldwide with access to new and more effective treatments for patients facing critical health issues.

Neuron23, Inc. is a biotechnology company focused on developing therapies for genetic disorders, particularly neurological diseases such as Parkinson's and Alzheimer's. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company utilizes advanced artificial intelligence and genetic insights to enhance drug discovery. Neuron23's innovative platform integrates the latest advancements in human genetics with AI-driven methodologies to create targeted treatments for patients facing debilitating health challenges. By focusing on genetically defined conditions, Neuron23 aims to improve therapeutic options and outcomes for individuals affected by these disorders.

Endeavor BioMedicines is a clinical-stage company based in Solana Beach, California, that focuses on developing innovative treatments for pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). Founded in 2018, the company is working on a small-molecule inhibitor designed to target the underlying causes of this progressive lung disease. The treatment aims to not only halt the progression of IPF but also has the potential to reverse its effects, offering healthcare professionals a new avenue for precision treatments in managing terminal conditions. Endeavor BioMedicines strives to improve patient outcomes and enhance quality of life for those affected by this challenging illness.

Ceptur Therapeutics is focused on developing innovative genetic medicines aimed at addressing diseases that currently lack effective treatments. The company utilizes its proprietary U1 Adaptor technology, which enables long-lasting and non-genotoxic silencing of disease-causing genes, particularly in challenging tissues beyond the liver and central nervous system. This technology addresses the limitations of existing gene silencing methods and holds promise for cancer treatment, allowing healthcare providers to offer new therapeutic options for patients with various types of cancers.

AvengeBio is a biotechnology company based in Cambridge, Massachusetts, focused on developing cell-generated immunotherapies aimed at eradicating solid tumors. Founded in 2019, the company is advancing a drug delivery platform designed to enable the precision delivery of cytokines and other potent immunomodulatory molecules. This innovative approach allows for controlled release over time, fostering robust local and systemic anti-tumor immune responses while minimizing the toxicities typically associated with conventional immunotherapies. Through its research and development efforts, AvengeBio seeks to address the challenges posed by intractable solid tumors and improve treatment outcomes for cancer patients.

Mythic Therapeutics is a biotechnology company dedicated to advancing cancer treatment through innovative antibody-drug conjugates (ADCs). The company specializes in a protein engineering approach that enhances the potency of ADCs while maintaining safety, allowing for more effective targeted therapies across various tumor types. By improving the uptake of these therapeutics in cancerous cells and minimizing payload release in healthy cells, Mythic Therapeutics aims to increase the efficacy of treatments without causing significant side effects or toxicity. This technology positions the company to make a meaningful impact in cancer care, enabling healthcare practitioners to better utilize ADCs for a wide array of patients.

Sermonix Pharmaceuticals is a biotechnology company based in Columbus, Ohio, founded in 2014. The company specializes in the development and commercialization of female-specific oncology products, focusing on late-stage women’s health treatments. Its primary product, lasofoxifene, is a selective estrogen receptor modulator designed for the treatment of vulvovaginal atrophy and the treatment and prevention of post-menopausal osteoporosis. Additionally, Sermonix is involved in developing pharmaceutical drugs aimed at treating ESR1-mutated metastatic breast and gynecological cancers, thereby addressing critical needs in women's health and oncology.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, established in 2018. The company is dedicated to developing clinical candidates targeting cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at treating resistant hypertension and primary aldosteronism. CinCor Pharma's mission is to advance its promising clinical candidates toward marketing approval, emphasizing the development of innovative therapies for patients suffering from cardio-renal conditions.

HilleVax is a clinical-stage biopharmaceutical company established in 2021 and based in Boston, Massachusetts. The company specializes in the development and commercialization of innovative vaccines. Its primary focus is on HIL-214, a vaccine candidate that utilizes virus-like particle (VLP) technology aimed at preventing moderate-to-severe acute gastroenteritis caused by norovirus infection.

A-Alpha Bio, Inc. is a biotechnology company based in Seattle, Washington, established in 2017. The company specializes in synthetic biology, offering a platform known as AlphaSeq, which assists pharmaceutical companies in the development of drugs that interact with multiple targets. A-Alpha Bio provides cell-based tools for high-throughput and quantitative measurements of protein interactions, facilitating target discovery, library screening, and preclinical drug characterization. Its proprietary technology enables the mapping of protein-protein interactions at a large scale, allowing for the identification and quantification of interactions among thousands of protein pairs simultaneously. By delivering detailed maps of these interactions, A-Alpha Bio helps researchers identify potential drug targets and understand disease mechanisms, thereby streamlining the therapeutic discovery process and improving the efficiency of drug development. The company comprises a team of experts in synthetic biology, structural biology, and next-generation sequencing, focused on overcoming challenges in the drug development pipeline.

Zentera Therapeutics is a biopharmaceutical company.

Amylyx Pharmaceuticals develops innovative therapies for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative diseases. The company's primary product, AMX0035, is an investigational therapy that combines two active compounds, sodium phenylbutyrate and Taurursodiol. This fixed-dose formulation aims to address mitochondrial energy crises and the accumulation of toxic proteins in the endoplasmic reticulum, which contribute to neurological decline. In addition to ALS, Amylyx's research pipeline includes potential treatments for Wolfram syndrome and Alzheimer's disease. Founded in 2013 and based in Cambridge, Massachusetts, Amylyx Pharmaceuticals is dedicated to advancing solutions for serious brain disorders.

Point Biopharma Global Inc., established in 2019 and headquartered in Toronto, Canada, specializes in the development and commercialization of radioligand therapies for cancer treatment. The company leverages a portfolio of advanced radiopharmaceutical assets, expertise in radioisotopes like Actinium-225 and Lutetium-177, and innovative manufacturing technology. Point Biopharma Global's pipeline includes several promising candidates, such as PNT2002, PNT2004, PNT2003, and PNT2001, aiming to revolutionize theragnostic drug development and radioligand commercialization.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics based on CRISPR technology to address genetic disorders. Founded in 2017 and headquartered in Berkeley, California, the company utilizes its proprietary X-Editing (XE) platform, which features highly engineered CRISPR enzymes designed to improve the efficacy, specificity, and deliverability of genome editing. Scribe Therapeutics aims to overcome existing limitations in genetic medicine by creating custom-engineered enzymes and advanced delivery systems. Their approach seeks to establish CRISPR-based therapies as a new standard in clinical care, potentially transforming the treatment landscape for various conditions, including immuno-oncology, degenerative disorders, and enzyme replacement therapy. By prioritizing therapeutic precision and allele-specific targeting, Scribe Therapeutics is committed to expanding access to groundbreaking treatments that can significantly impact patient lives.

Absci is a drug and target discovery company that leverages deep learning AI and synthetic biology to enhance the therapeutic potential of proteins. Its Integrated Drug Creation Platform streamlines the process of identifying novel drug targets, discovering optimal biotherapeutic candidates, and generating the necessary cell lines for manufacturing. By combining biologic drug discovery and cell line development into a single, efficient workflow, Absci collaborates with biotech and pharmaceutical innovators to develop next-generation protein-based drugs. These innovations include Bionic Proteins, which feature nonstandard amino acids, and other unique drug designs that may be unattainable through conventional methods. Absci's mission is to facilitate the development of improved medicines by effectively translating innovative ideas into viable drugs.

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.

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.

Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, focused on developing innovative vaccines using its proprietary Multiple Antigen Presenting System (MAPS) technology. This approach allows for the combination of protective polysaccharides and proteins into a single vaccine, thereby enhancing immune responses against a variety of infectious diseases affecting both children and adults. Affinivax targets several significant pathogens, including Streptococcus pneumoniae, bacterial nosocomial infections, Salmonella typhi, and Staphylococcus aureus. The company aims to create vaccines that not only provide broader disease coverage compared to existing options but also address infectious diseases lacking effective immunization strategies. Founded in 2014, Affinivax benefits from the expertise of leading professionals in infectious diseases and vaccines, and it has received backing from the Bill & Melinda Gates Foundation, along with an exclusive license from Boston Children’s Hospital for its MAPS technology.

Neuron23, Inc. is a biotechnology company focused on developing therapies for genetic disorders, particularly neurological diseases such as Parkinson's and Alzheimer's. Founded in 2018 and headquartered in South San Francisco, California, with an additional office in Munich, Germany, the company utilizes advanced artificial intelligence and genetic insights to enhance drug discovery. Neuron23's innovative platform integrates the latest advancements in human genetics with AI-driven methodologies to create targeted treatments for patients facing debilitating health challenges. By focusing on genetically defined conditions, Neuron23 aims to improve therapeutic options and outcomes for individuals affected by these disorders.

Prometheus Biosciences, established in 1995 and headquartered in San Diego, California, specializes in developing and commercializing innovative pharmaceutical and diagnostic products. The company's portfolio includes tests that enable physicians to provide personalized patient care, such as monitoring drug and anti-drug antibody levels, diagnosing and prognosticating inflammatory bowel disease (IBD), and detecting celiac disease and lactose intolerance. Prometheus also offers therapeutic products like Entocort EC for treating Crohn's disease and has agreements to develop and market other drugs. The company's commitment to innovation positions it as a leader in precision healthcare, particularly in gastroenterology.

LianBio is a biopharmaceutical company based in Shanghai, China, with an additional office in Princeton, New Jersey. Founded in 2019, the company focuses on the discovery and development of therapeutic drugs aimed at treating oncology and cardiorenal diseases, as well as other medical conditions. LianBio's mission is to enhance access to innovative medicines by partnering with leading organizations in the field to leverage advanced scientific discoveries. The company is dedicated to addressing unmet medical needs in Greater China and other Asian markets by in-licensing assets and building a diverse pipeline of clinically validated product candidates. Its portfolio aims to establish new standards of care across various therapeutic areas, including cardiovascular, oncology, ophthalmology, inflammatory diseases, and respiratory conditions.

AavantiBio, Inc. is a biopharmaceutical company based in Gainesville, Florida, focused on developing gene transfer and gene editing therapies for rare genetic diseases. Established in 2019, the company primarily targets Friedreich’s Ataxia, a severe inherited condition that leads to significant cardiac and central nervous system dysfunction. In addition to addressing this specific disorder, AavantiBio is advancing a pipeline of innovative gene therapies aimed at treating various autoimmune diseases and other conditions with unmet medical needs. Through its commitment to research and development, the company seeks to improve the lives of patients affected by these debilitating genetic disorders.

Nuvation Bio Inc. is a biopharmaceutical company dedicated to addressing unmet needs in oncology through the development of novel therapies for challenging cancers. Founded in 2018 and headquartered in New York, with an additional office in San Francisco, Nuvation Bio focuses on treating patients whose conditions have not responded to conventional treatments. The company's leading clinical-stage candidate, NUV-868, is a BD2-selective oral small molecule BET inhibitor that targets the BRD4 protein, which plays a crucial role in regulating tumor growth and differentiation. In addition to NUV-868, Nuvation Bio is advancing a proprietary small molecule Drug-Drug Conjugate platform aimed at enhancing therapeutic efficacy. The company’s comprehensive portfolio includes multiple drug development candidates designed to meet the specific needs of cancer patients.

Escient Pharmaceuticals is a biotechnology company based in San Diego, California, that develops and manufactures drugs targeting G protein-coupled receptors (GPCRs) for the treatment of neuro-immuno-inflammatory and autoreactive diseases. Founded in 2017, the company specializes in harnessing the therapeutic potential of specific orphan GPCRs, particularly the Mas-Related G-Protein Receptors (Mrgprs), to address various neurosensory-inflammatory disorders. Escient's innovative approach aims to provide first-in-class therapies for conditions associated with mast cell-mediated disorders and cholestatic pruritus, thereby expanding treatment options for healthcare providers and patients facing these challenging diseases.

Zymergen, Inc. is a biotechnology company that focuses on researching, developing, and manufacturing microbes for various industries, including agriculture, chemicals, materials, pharmaceuticals, electronics, and personal care. Founded in 2013 and headquartered in Emeryville, California, Zymergen employs a platform that integrates automation, machine learning, and genomics to enhance the efficiency of microbial strain optimization and production processes. This technology enables the company to improve existing manufacturing strains and facilitates the development of new products by engineering novel molecules from microbes. With additional offices in Boise, Idaho; Medford, Massachusetts; Seattle, Washington; and Tokyo, Japan, Zymergen aims to partner with nature to create innovative materials and products that deliver significant value across multiple sectors.

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.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

ReViral Ltd is a biotechnology company based in Stevenage, United Kingdom, that specializes in the discovery and development of antiviral drugs targeting the respiratory syncytial virus (RSV). Founded in 2011, ReViral focuses on creating novel first-in-class compounds, including a highly potent fusion inhibitor designed to treat severe RSV infections, particularly in vulnerable populations such as neonates. The company's innovative approach aims to expand the antiviral market by providing effective treatment options for patients affected by RSV, a virus known for causing significant respiratory illness. Through its research and development efforts, ReViral seeks to address unmet medical needs in the realm of viral infections.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Cerevel Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neuroscience diseases. Founded in 2018 and headquartered in Cambridge, Massachusetts, the company is advancing a diverse pipeline that includes five clinical-stage investigational therapies and several preclinical compounds targeting conditions such as schizophrenia, epilepsy, Parkinson's disease, and substance use disorder. Key products under development include CVL-231, a positive allosteric modulator for schizophrenia; CVL-865, which is being tested for drug-resistant focal onset epilepsy and acute anxiety; and Tavapadon, a selective dopamine D1/D5 partial agonist for Parkinson's disease. Cerevel Therapeutics employs a unique approach that leverages expertise in neurocircuitry and receptor selectivity to address unmet medical needs in the field of neuroscience.

Verona Pharma is a clinical-stage biopharmaceutical company based in London, established in 2005. The company specializes in developing and commercializing therapies for respiratory diseases that have significant unmet medical needs, including chronic obstructive pulmonary disease (COPD), cystic fibrosis, and asthma. Its lead product candidate, ensifentrine, is an inhaled dual inhibitor of the enzymes phosphodiesterase 3 and 4, functioning as both a bronchodilator and an anti-inflammatory agent. Ensifentrine is being developed in various formulations, including nebulized, dry powder inhaler, and pressurized metered-dose inhaler, with the nebulized form currently in Phase 2b clinical development for the maintenance treatment of COPD. Verona Pharma's commitment is to enhance the health and quality of life for individuals suffering from chronic respiratory conditions through innovative therapeutic solutions.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

AsclepiX Therapeutics, Inc. is a biopharmaceutical company focused on the design and development of innovative treatments for diseases related to abnormal blood vessel growth, specifically through the use of short biomimetic anti-angiogenic and anti-lymphangiogenic peptides. The company's lead candidates, including AXT107, AXT201, AXT301, and AXT501, target conditions such as diabetic macular edema, wet age-related macular degeneration, and macular edema following retinal vein occlusion, which are among the leading causes of blindness. Additionally, AXT201, AXT301, and AXT501 are being explored for their potential in treating various solid tumors. AsclepiX leverages advanced computational biology, bioinformatics, and the latest developments in biomaterials to enhance drug delivery systems, aiming to create long-lasting biodegradable nanoparticles that can effectively deliver its therapeutic peptides. Incorporated in 2011 and based in Baltimore, Maryland, AsclepiX Therapeutics is committed to addressing unmet medical needs in ophthalmology, oncology, and other diseases driven by angiogenesis and lymphangiogenesis.

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.

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.

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing novel therapies for patients with genetically defined diseases that have significant unmet medical needs. The company's lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy, a rare condition characterized by progressive muscle wasting and disability. Another key candidate, FTX-6058, aims to upregulate fetal hemoglobin to treat sickle cell disease and beta-thalassemia. Fulcrum is also actively researching drug targets for other genetic disorders, including Duchenne muscular dystrophy and Friedreich ataxia, as well as neurological and pulmonary diseases. The company has established collaborations with Acceleron Pharma to explore biological targets in pulmonary disease and with MyoKardia to develop therapies for genetic cardiomyopathies. Founded in 2015, Fulcrum Therapeutics continues to focus on improving patient outcomes in rare disease contexts.

Athira Pharma, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, focused on developing small molecules aimed at restoring neuronal health and addressing neurodegenerative diseases. The company's lead candidate, ATH-1017, is a small hepatocyte growth factor/MET activator designed to penetrate the blood-brain barrier and is currently undergoing various clinical trials for the treatment of Alzheimer's and Parkinson's diseases. Additionally, Athira is advancing other candidates, including ATH-1019, which is in preclinical development for depression, and ATH-1018, aimed at treating peripheral neuropathy. Founded in 2011 and originally named M3 Biotechnology, Athira Pharma changed its name in April 2019 to reflect its commitment to innovative therapies that positively impact individuals affected by brain disorders.

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.

Dynacure is a clinical-stage drug development company dedicated to enhancing the lives of patients with rare and orphan diseases. The company is advancing its lead product, DYN101, an investigational antisense medicine aimed at modulating the expression of dynamin 2 to treat Centronuclear Myopathies, in collaboration with Ionis Pharmaceuticals. In addition to DYN101, Dynacure is developing a complementary research portfolio that targets various other orphan disorders, including a program focused on hereditary spastic paraplegias linked to specific genetic mutations. Through its innovative approach and expertise in rare disease drug development, Dynacure seeks to address significant unmet medical needs in this underserved patient population.

immatics is a biopharmaceutical company focused on the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors. The company's customized Adoptive Cell Therapies (ACTengine) and antibody-like TCR Bispecifics are the two main product classes in the pipeline (TCER). With a focus on particularly difficult-to-treat solid malignancies, each therapy modality has unique characteristics to create the desired therapeutic impact for patients at various disease stages and with various types of tumors. immatics biotechnologies was established in 2000 by Hans Rammensee, Harpreet Singh, and Steffen Walter in Tübingen, Baden-Wurttemberg.

Agile Therapeutics is a women's healthcare company dedicated to addressing the unmet health needs of women through the development and commercialization of prescription contraceptive products. The company's lead product, Twirla, is a once-weekly transdermal patch that delivers a combination of levonorgestrel and ethinylestradiol, providing a non-daily contraceptive option. In addition to Twirla, Agile Therapeutics is advancing a pipeline of related products, including various regimens designed to offer customizable menstrual cycle management options, such as AG200-ER for extended cycles and AG200-SP for lighter periods. The firm's proprietary Skinfusion technology facilitates effective drug delivery through the skin. Headquartered in Princeton, New Jersey, Agile Therapeutics aims to enhance women's reproductive health by providing innovative contraceptive solutions.

Cerebral Therapeutics, Inc. is a clinical-stage pharmaceutical company based in Aurora, Colorado, focused on developing implanted drug-device combination therapies for neurologic diseases, particularly refractory epilepsy. The company utilizes a refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose of its formulation of the anti-epileptic drug valproic acid, known as CT-010. This innovative approach aims to bypass the blood-brain barrier, enhancing treatment outcomes for patients with chronic neurological conditions. Founded in 2010, Cerebral Therapeutics is dedicated to improving the lives of individuals affected by uncontrolled neurological diseases.

Quellis Biosciences, Inc. is a biopharmaceutical company headquartered in Waltham, Massachusetts, established in 2017. The company is dedicated to developing new monoclonal antibody therapies aimed at treating rare diseases, particularly for patients who lack effective treatment options. By focusing on next-generation antibody therapies, Quellis seeks to enhance patient outcomes and deliver innovative solutions that address unmet medical needs. Through its research and development efforts, the company aims to optimize therapeutic leads and identify promising antibodies, ultimately improving the quality of life for individuals affected by serious rare conditions.

Zentalis Pharmaceuticals is a clinical-stage biopharmaceutical company based in New York, dedicated to developing small molecule therapeutics aimed at treating various cancers. Founded in 2014, the company focuses on discovering and advancing drug candidates that target critical biological pathways in cancer. Its lead product candidate, ZN-c5, is an oral selective estrogen receptor degrader currently undergoing Phase I/II clinical trials for advanced or metastatic breast cancer. In addition, Zentalis is developing ZN-c3, an inhibitor of the WEE1 protein tyrosine kinase, for advanced solid tumors; ZN-d5, a selective B-cell lymphoma 2 inhibitor for hematological malignancies; and ZN-e4, an irreversible inhibitor of mutant epidermal growth factor receptor for advanced non-small cell lung cancer, all of which are also in Phase I/II clinical trials. The company utilizes an integrated discovery engine to identify new chemical entities with the potential for differentiated therapeutic profiles.

Black Diamond Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, specializes in precision oncology medicine. The company discovers and develops small molecule therapies that are tumor-agnostic, meaning they target specific genetic mutations across various cancer types rather than being limited to a single cancer indication. Its lead product candidate, BDTX-189, inhibits non-canonical and oncogenic mutations of ErbB kinases epidermal growth factor receptor (EGFR). The company's Mutation-Allostery-Pharmacology platform enables it to target undrugged mutations in patients with genetically defined cancers, offering new treatment options where few exist. Black Diamond Therapeutics is also advancing early-stage pipeline programs targeting allosteric mutations in other cancer-related kinases.

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.

Landos Biopharma, Inc. is a clinical-stage biopharmaceutical company based in Blacksburg, Virginia, specializing in the development of oral therapeutics for autoimmune diseases. Founded in 2017, the company focuses on innovative treatments for conditions such as inflammatory bowel disease (IBD), Crohn's disease, and ulcerative colitis. Its lead product candidate, NX-13, is a novel, gut-selective agonist targeting the NLRX1 pathway. The company also has a diverse pipeline that includes other internally discovered compounds aimed at novel immunometabolic pathways. By developing first-in-class therapeutics, Landos Biopharma seeks to provide effective treatment options for patients suffering from autoimmune conditions.

Foamix Pharmaceuticals is a specialty pharmaceutical company based in Bridgewater, New Jersey, that focuses on the development and commercialization of proprietary topical drugs for dermatological conditions, as well as for ophthalmologic and gynecologic applications. Founded in 2014, the company utilizes its Molecule Stabilizing Technology platform to create innovative foam-based formulations. Its lead product, a first-in-class Topical Minocycline Foam, targets significant markets in dermatology and ophthalmology, having shown high efficacy and favorable safety in clinical trials for conditions such as acne and impetigo. Foamix's pipeline includes multiple drug candidates aimed at treating various skin conditions, including FMX101, FMX102, FMX103, and FDX104. The company is also engaged in development and licensing agreements with other pharmaceutical firms, enhancing its reach in the healthcare market.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

Neptune Wellness Solutions Inc. is a health and wellness company based in Laval, Canada, that operates in the consumer-packaged goods sector. The company develops a diverse portfolio of lifestyle brands and offers turnkey product development and supply chain solutions across various health and wellness verticals, including legal cannabis, hemp, and nutraceuticals. Neptune specializes in extraction and purification services from cannabis and hemp biomass, as well as raw material sourcing, formulation, and quality control for omega-3 and hemp-derived ingredients. Its product offerings encompass various delivery forms, such as soft gels, capsules, tinctures, and edibles. Additionally, Neptune has established a collaboration with International Flavors & Fragrances Inc. to co-develop hemp-derived CBD products for the retail and health markets. The company is committed to sustainability and innovation, aiming to provide high-quality, affordable products that meet the growing consumer demand for natural and plant-based solutions. Neptune's products are available in over 27,000 retail locations, reflecting its extensive reach in the marketplace.

Athenex, Inc. is a biopharmaceutical company focused on the discovery, development, and commercialization of innovative therapies for cancer and related conditions. Founded in 2003 and headquartered in Buffalo, New York, Athenex operates across North America and Asia, with a clinical pipeline that includes several promising product candidates. The company’s Orascovery platform features oral formulations of well-known chemotherapeutic agents such as Paclitaxel, Irinotecan, Docetaxel, Topotecan, and Eribulin, which are in various stages of clinical trials targeting multiple cancer types. Additionally, Athenex is developing Src Kinase inhibitors for skin cancers and psoriasis, as well as advanced immunotherapies and arginine deprivation therapies. Through its commitment to innovative research and collaborations, Athenex aims to become a leader in delivering effective cancer treatments and improving patient health outcomes globally.

Poseida Therapeutics is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapeutics for patients with significant unmet medical needs. The company utilizes non-viral gene engineering technologies to create a pipeline of targeted therapies, including both autologous and allogeneic chimeric antigen receptor T cell (CAR-T) product candidates aimed at treating hematological malignancies and solid tumors. Additionally, Poseida is advancing gene therapies for rare and life-threatening conditions, such as Ornithine transcarbamylase deficiency, methylmalonic acidemia, and various genetic liver diseases. Founded in 2014, Poseida is committed to addressing critical health challenges through its focused research and development efforts.

Fusion Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company headquartered in Hamilton, Canada, specializing in the development of radiopharmaceuticals as precision medicines for challenging cancers. The company employs its proprietary Targeted Alpha Therapies platform and Fast-Clear linker technology to connect alpha particle-emitting isotopes to antibodies, enabling the selective delivery of therapeutic agents to tumors. Fusion's lead product candidate, FPI-1434, is currently undergoing Phase 1 clinical trials as a monotherapy for solid tumors expressing insulin-like growth factor 1 receptor. Additionally, the company is exploring the use of FPI-1434 in combination with approved checkpoint inhibitors and DNA damage response inhibitors in preclinical studies to enhance anti-tumor activity. Fusion is also advancing another product candidate, FPI-1966, into clinical development for head and neck and bladder cancers that express fibroblast growth factor receptor. Founded in 2014 as a spinout of the Centre for Probe Development and Commercialization, Fusion aims to transform how cancers are treated through innovative radiochemistry techniques.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company focused on developing and commercializing medicines for underserved patient populations affected by rare diseases and cancer. The company employs a precision medicine approach and has a diverse portfolio of small molecule targeted oncology products. Its lead candidate, nirogacestat, an oral gamma secretase inhibitor, is currently in Phase III clinical trials for desmoid tumors. Additionally, SpringWorks is advancing several other product candidates, including mirdametinib, an oral MEK inhibitor in Phase IIb trials for neurofibromatosis type 1-associated plexiform neurofibromas, and various combinations involving nirogacestat for multiple myeloma. The company has established collaborations with organizations such as BeiGene, GlaxoSmithKline, and Allogene, aimed at enhancing the development of its therapies. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks is committed to unlocking the potential of its pipeline to provide innovative solutions for patients suffering from cancer and rare diseases.

ADMA Biologics, Inc. is a biopharmaceutical company focused on developing, manufacturing, and marketing specialty plasma-derived biologics aimed at treating immune deficiencies and infectious diseases in the United States. The company offers a range of products, including BIVIGAM and ASCENIV, both intravenous immune globulin therapies for primary humoral immunodeficiency, as well as Nabi-HB, which is used for acute exposure to Hepatitis B. ADMA is also involved in the development of additional plasma-derived therapeutics, particularly targeting infections like S. pneumonia. To support its operations, the company runs FDA-licensed source plasma collection facilities known as ADMA BioCenters, which contribute to the supply of plasma necessary for its product manufacturing. ADMA Biologics distributes its products through independent distributors, sales agents, specialty pharmacies, and other healthcare providers. Founded in 2004 and based in Ramsey, New Jersey, ADMA is dedicated to serving niche patient populations, particularly those with compromised immune systems.

Black Diamond Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, specializes in precision oncology medicine. The company discovers and develops small molecule therapies that are tumor-agnostic, meaning they target specific genetic mutations across various cancer types rather than being limited to a single cancer indication. Its lead product candidate, BDTX-189, inhibits non-canonical and oncogenic mutations of ErbB kinases epidermal growth factor receptor (EGFR). The company's Mutation-Allostery-Pharmacology platform enables it to target undrugged mutations in patients with genetically defined cancers, offering new treatment options where few exist. Black Diamond Therapeutics is also advancing early-stage pipeline programs targeting allosteric mutations in other cancer-related kinases.