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 focused on developing innovative treatments for severe, rare muscle disorders with significant unmet medical need. It employs a holistic drug discovery approach targeting the muscle as an organ, utilizing its proprietary platform to identify precision medicines regulating key proteins in muscle tissue.

Ocuphire Pharma is a clinical-stage biopharmaceutical company focused on developing and commercializing therapies for various ophthalmic disorders. Its lead product candidate, Nyxol eye drops, is a once-daily preservative-free formulation of phentolamine mesylate aimed at reducing pupil size for indications such as night vision disturbances, glaucoma, pharmacologically-induced mydriasis, and presbyopia.

RAPT Therapeutics is a clinical-stage biopharmaceutical company focused on discovering, developing and commercializing orally delivered small-molecule therapies for oncology and inflammatory diseases. It uses a proprietary discovery and development engine to create highly selective compounds that modulate immune responses. The company's lead oncology candidate, FLX475, targets the C-C motif chemokine receptor 4 and is in a Phase 1/2 program as monotherapy and in combination with pembrolizumab. The lead inflammation candidate, RPT193, is designed to selectively inhibit migration of type 2 T helper cells into inflamed tissues. Additional targets such as general control nonderepressible 2 and hematopoietic progenitor kinase 1 are in discovery. Founded in 2015 and headquartered in South San Francisco, the company aims to address unmet medical needs in cancer and inflammatory diseases.

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 aimed at addressing significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company has established a robust pipeline supported by a proprietary discovery engine and a wholly-owned research and development facility. It possesses an extensive library of molecules and a team with considerable expertise in developing compounds that target nuclear receptors, transcription factors, and epigenetic modulation. Inventiva is advancing clinical candidates, including lanifibranor, which is being developed for non-alcoholic steatohepatitis (NASH), a condition currently lacking approved treatments. Additionally, the company is engaged in a variety of pre-clinical therapy programs, further strengthening its portfolio in the fields of oncology, fibrosis, and rare diseases.

OnKure Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing precision medicines that target biologically validated drivers of cancers underserved by existing therapies. The company develops epigenetic therapies, including selective histone deacetylase inhibitors, and advances candidates such as OKI-179 and OKI-422 in preclinical and clinical development. Its approach aims to directly target malignant cells, combine with other targeted therapies, and prime the tumor microenvironment to enhance immunotherapies, with a pipeline of tumor-agnostic candidates designed for optimal efficacy and tolerability.

scPharmaceuticals Inc. is a pharmaceutical company focused on developing and commercializing innovative therapeutic products, particularly 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, aimed at treating congestion in patients with decompensated heart failure. Additionally, scPharmaceuticals is advancing its product pipeline with scCeftriaxone, an antibiotic targeting infections from both gram-positive and gram-negative organisms, and a carbapenem program for addressing gram-negative infections. Founded in 2013 and headquartered in Burlington, Massachusetts, scPharmaceuticals emphasizes the importance of subcutaneous drug administration, offering a convenient two-component delivery system that allows for patient self-administration, thereby providing a safer and more comfortable alternative to traditional intravenous methods. The company collaborates with West Pharmaceutical Services to enhance its SmartDose system, furthering its mission to improve patient outcomes.

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 developing next-generation CAR T-cell therapies to overcome cancer treatment resistance and access barriers. The company focuses on addressing limitations of approved autologous CAR T therapies, including manufacturing constraints, long turnaround times, and reimbursement challenges, to broaden patient access to potentially curative treatments. Its programs aim to deliver novel solutions that improve cancer treatment outcomes and patient experience. One of its product candidates, CRG-023, is a tri-specific CAR T designed to overcome relapse by targeting multiple B-cell antigens (CD19, CD20, CD22) and addressing mechanisms such as antigen loss, insufficient co-stimulation, and T cell exhaustion.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company that develops a novel class of medicines called Bicycles, synthetic short peptides constrained to form two loops that stabilize their structure. Its lead program BT1718 is a Bicycle Toxin Conjugate for oncology, with Phase I/IIa trials in tumors expressing Membrane Type 1 matrix metalloprotease. The company is also advancing BT5528, a BTC targeting EphA2, and BT8009 in preclinical development for Nectin-4, along with preclinical CD137 and THR-149, a plasma kallikrein inhibitor for diabetic macular edema. Beyond its internal programs, Bicycle Therapeutics collaborates with Cancer Research Technology, Cancer Research UK, AstraZeneca, Sanofi, Oxurion, and the Dementia Discovery Fund, and has a discovery collaboration with Genentech for immuno-oncology targets. The company was founded in 2009 and is headquartered in Cambridge, United Kingdom.

Founded in 2022, Pheon Therapeutics specializes in developing Antibody-Drug Conjugates (ADCs) for challenging-to-treat cancers. Its primary focus is a first-in-class ADC targeting a novel marker overexpressed in various solid tumors.

CinDome Pharma specializes in developing innovative treatments for individuals affected by chronic gastrointestinal disorders, with a primary focus on chronic gastroparesis.

Endeavor BioMedicines is a clinical-stage company based in Solana Beach, California, founded in 2018. The company is dedicated to developing innovative treatments that address the underlying causes of pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). Endeavor BioMedicines is focused on creating a small-molecule inhibitor that has the potential to not only halt the progression of IPF but also to reverse its effects. By concentrating on these advancements, the company aims to enable healthcare professionals to provide more precise treatments for terminal conditions, ultimately improving patient outcomes and enhancing quality of life.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative, antibody-based therapies aimed at enhancing the lives of cancer patients. The company specializes in antibody-drug conjugates and other biologics, focusing on oncologic diseases with significant unmet medical needs. TORL BioTherapeutics employs a strategy that involves target identification and early discovery work, allowing them to selectively advance drug programs through novel licensing agreements. This approach enables their researchers to develop proprietary drugs with unique and optimized profiles, contributing to the advancement of cancer treatment options.

Zevra Therapeutics is a biopharmaceutical company focused on developing safer therapies for pain, ADHD, and other CNS diseases. It employs its proprietary Ligand Activated Therapy platform to create abuse-resistant opioid pain relievers.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing precision oncology therapeutics. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond positively to specific treatments, thereby enhancing the effectiveness of its drug development efforts. Acrivon's pipeline features its lead program, ACR-368 (prexasertib), which is currently in Phase 2 trials, alongside several preclinical programs targeting key components of DNA damage response and cell cycle regulation, including WEE1 and PKMYT1. By concentrating on complex oncology drug targets that are often not addressed by traditional therapies, Acrivon aims to expedite the advancement of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials.

Neurosterix develops innovative medicines using allosteric modulation, a method that overcomes traditional treatment limitations. Their drug discovery platform focuses on neurological disorders, aiming to improve efficacy, safety, and tolerability for patients.

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 is a clinical-stage company focused on discovering, developing, and commercializing therapeutics targeting peptide hormone receptors for treating rare endocrine diseases and related cancers. Its lead product candidate, paltusotine, is an oral selective nonpeptide somatostatin receptor type 2 biased agonist in Phase I trials for acromegaly and neuroendocrine tumors.

Applied Therapeutics is a biopharmaceutical company focused on developing transformative drugs targeting fatal and debilitating diseases with high unmet medical needs. Its pipeline includes novel product candidates such as AT-007 for rare metabolic diseases like galactosemia, AT-001 for diabetic cardiomyopathy, and AT-003 for diabetic retinopathy.

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 focused on developing therapies for rare and niche allergic and immunological diseases. It is advancing monoclonal antibody programs such as STAR-0215, a plasma kallikrein inhibitor being developed for hereditary angioedema, and STAR-0310, an OX40 antagonist for atopic dermatitis, both in preclinical development to address immune-mediated inflammatory conditions.

Avalyn Pharma is a biopharmaceutical company focused on developing therapies for severe respiratory diseases, notably idiopathic pulmonary fibrosis (IPF). Established in 2011, the company is headquartered in Seattle, Washington.

Rapport Therapeutics is a clinical-stage biotechnology company dedicated to discovering and developing precision medicines for neurological disorders. The company focuses on creating transformational small-molecule therapies aimed at patients with central nervous system conditions. Leveraging foundational research in neuronal receptor biology, Rapport Therapeutics maps and targets specific neuronal receptor complexes, which are complex assemblies of proteins that include principal receptor subunits and receptor-associated proteins. These receptor-associated proteins are essential for regulating receptor expression and functionality, allowing the company to innovate in the treatment of neurological diseases.

Forte Biosciences is a biotechnology company specializing in dermatology. Its lead product, FB-401, is a topically applied live biotherapeutic developed with the National Institutes of Health and National Institute of Allergy and Infectious Diseases for treating inflammatory skin diseases.

AsclepiX Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative treatments for retinal diseases and certain cancers. The company specializes in designing short biomimetic peptides that target angiogenesis and lymphangiogenesis, utilizing advanced bioinformatics and systems biology approaches. Its lead product candidates, including AXT107, AXT201, AXT301, and AXT501, are aimed at addressing conditions such as diabetic macular edema, age-related macular degeneration, and macular edema from retinal vein occlusion, all of which are significant causes of blindness in the western world. Additionally, AXT201, AXT301, and AXT501 are in early development stages for treating various solid tumors. AsclepiX Therapeutics employs cutting-edge biomaterials and drug delivery systems, including biodegradable nano and microparticles, to enhance the therapeutic efficacy of its peptide-based treatments. Incorporated in 2011 and headquartered in Baltimore, Maryland, the company is leveraging its platform technology to advance solutions in both ophthalmology and oncology.

A-Alpha Bio, Inc. is a biotechnology company specializing in synthetic biology and advanced quantitative measurement of protein interactions to facilitate drug development. Founded in 2017 and headquartered in Seattle, Washington, the company offers its proprietary AlphaSeq platform, which enables high-throughput mapping of protein-protein interactions. This technology allows for the simultaneous identification and quantification of interactions among thousands of protein pairs, streamlining the drug discovery process by aiding in the identification of potential drug targets and enhancing the understanding of disease mechanisms. By providing biologics and molecular glue discovery and optimization services, A-Alpha Bio supports pharmaceutical companies in optimizing binding and specificity to multiple target proteins. The team comprises synthetic biologists, structural biologists, and next-generation sequencing experts dedicated to overcoming challenges in the drug development process.

Lyra Therapeutics is a clinical-stage therapeutics company focused on the development and commercialization of localized treatments for ear, nose, and throat diseases. Its XTreo platform is designed to deliver medicines directly to affected tissue for sustained effect with a single administration. The company’s initial product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices intended for in-office administration to provide up to six months of continuous therapy to the sinonasal passages for chronic rhinosinusitis, with mometasone furoate as the active ingredient. Founded in 2005 and headquartered in Watertown, Massachusetts.

OnKure Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing precision medicines that target biologically validated drivers of cancers underserved by existing therapies. The company develops epigenetic therapies, including selective histone deacetylase inhibitors, and advances candidates such as OKI-179 and OKI-422 in preclinical and clinical development. Its approach aims to directly target malignant cells, combine with other targeted therapies, and prime the tumor microenvironment to enhance immunotherapies, with a pipeline of tumor-agnostic candidates designed for optimal efficacy and tolerability.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative, antibody-based therapies aimed at enhancing the lives of cancer patients. The company specializes in antibody-drug conjugates and other biologics, focusing on oncologic diseases with significant unmet medical needs. TORL BioTherapeutics employs a strategy that involves target identification and early discovery work, allowing them to selectively advance drug programs through novel licensing agreements. This approach enables their researchers to develop proprietary drugs with unique and optimized profiles, contributing to the advancement of cancer treatment options.

CARGO Therapeutics is a clinical-stage biotechnology company developing next-generation CAR T-cell therapies to overcome cancer treatment resistance and access barriers. The company focuses on addressing limitations of approved autologous CAR T therapies, including manufacturing constraints, long turnaround times, and reimbursement challenges, to broaden patient access to potentially curative treatments. Its programs aim to deliver novel solutions that improve cancer treatment outcomes and patient experience. One of its product candidates, CRG-023, is a tri-specific CAR T designed to overcome relapse by targeting multiple B-cell antigens (CD19, CD20, CD22) and addressing mechanisms such as antigen loss, insufficient co-stimulation, and T cell exhaustion.

Fusion Pharmaceuticals is a clinical-stage biopharmaceutical company specializing in developing radiopharmaceuticals as precision medicines. It focuses on creating targeted alpha therapeutics using its proprietary linker technology to enhance the safety and efficacy of these drugs, ultimately improving cancer patient outcomes.

Biodesix is a data-driven diagnostic solutions company focused on lung disease. It provides blood-based tests across the lung cancer continuum to aid risk assessment, diagnosis, monitoring and treatment guidance. Key tests include Nodify XL2 and Nodify CDT for reclassifying malignancy risk in suspicious lung nodules, along with VeriStrat immune profiling and GeneStrat tumor profiling to support treatment decisions. The company operates a CLIA-certified laboratory and delivers results in under 72 hours. Biodesix also develops and commercializes multivariate protein diagnostics based on its proprietary mass spectrometry platform, and partners with biopharmaceutical companies to create companion diagnostics and support diagnostic research, clinical trial testing, and development and commercialization of diagnostics. In addition to in-house testing services, it provides contract services to biopharma clients across discovery, development and testing phases. Biodesix is headquartered in Boulder, Colorado.

Zenas BioPharma is a clinical-stage biopharmaceutical company focused on developing immune-based therapies for autoimmune and immune-mediated diseases. Based in Florida, United States, the company advances biologic programs and aims to modulate B cell–driven pathology. Its lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb on B cells, thereby inhibiting harmful immune activity without depleting B 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 applying precision medicine to develop medicines for severe rare diseases and cancer. It maintains a differentiated targeted oncology portfolio with lead candidates nirogacestat, a gamma secretase inhibitor, and mirdametinib, a MEK inhibitor, both in late-stage trials; the pipeline also includes BGB-3245, an oral inhibitor of activating BRAF mutations, in Phase I, and several combination programs. The company pursues collaborations with BeiGene, GlaxoSmithKline, and Allogene and has license agreements with Pfizer for nirogacestat and mirdametinib. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks aims to advance potentially registrational studies in rare tumor types and genetically defined cancers through partnerships that expand its portfolio and patient access.

Cerebral Therapeutics, Inc. is a clinical-stage pharmaceutical company based in Aurora, Colorado, focused on developing innovative drug-device combination therapies for neurologic diseases, particularly refractory epilepsy. The company employs a unique implanted, refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose of valproic acid, an anti-epileptic drug formulated as CT-010. By utilizing reformulated off-patent drugs delivered directly to the brain's ventricles, Cerebral Therapeutics aims to bypass the blood-brain barrier, thereby enhancing treatment outcomes for patients suffering from chronic neurological conditions. Established in 2010, the company is committed to improving the quality of life for those with uncontrolled neurological disorders.

Surf Bio is a preclinical biopharmaceutical company focused on developing innovative therapeutic solutions through its next-generation surfactant technology. This novel biotechnology platform aims to enhance drug effectiveness across various therapeutic areas, including diabetes, oncology, infectious diseases, and gene therapy. By utilizing this advanced technology, Surf Bio seeks to provide healthcare organizations worldwide with access to improved drugs that can better address the needs of patients suffering from critical diseases.

Lyra Therapeutics is a clinical-stage therapeutics company focused on the development and commercialization of localized treatments for ear, nose, and throat diseases. Its XTreo platform is designed to deliver medicines directly to affected tissue for sustained effect with a single administration. The company’s initial product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices intended for in-office administration to provide up to six months of continuous therapy to the sinonasal passages for chronic rhinosinusitis, with mometasone furoate as the active ingredient. Founded in 2005 and headquartered in Watertown, Massachusetts.

Founded in 2018, Neuron23 is a biotechnology company based in South San Francisco, California, with an additional office in Munich, Germany. It specializes in developing and manufacturing medicines for genetic disorders using artificial intelligence technology and genetics.

Endeavor BioMedicines is a clinical-stage company based in Solana Beach, California, founded in 2018. The company is dedicated to developing innovative treatments that address the underlying causes of pulmonary fibrosis, particularly idiopathic pulmonary fibrosis (IPF). Endeavor BioMedicines is focused on creating a small-molecule inhibitor that has the potential to not only halt the progression of IPF but also to reverse its effects. By concentrating on these advancements, the company aims to enable healthcare professionals to provide more precise treatments for terminal conditions, ultimately improving patient outcomes and enhancing quality of life.

Ceptur Therapeutics is focused on developing innovative genetic medicines for patients with unmet medical needs. The company utilizes its proprietary U1 Adaptor technology to achieve non-genotoxic, long-lasting 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 has potential applications in cancer treatment, allowing healthcare providers to offer new therapeutic options for various cancer types. Through its advancements, Ceptur aims to improve patient outcomes by providing effective solutions for difficult-to-treat conditions.

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 specializing in innovative cancer therapeutics. It focuses on enhancing antibody-drug conjugates (ADCs) and other antibody-based therapies using advanced protein engineering techniques, aiming to boost potency without compromising safety.

Founded in 2014, Sermonix Pharmaceuticals is a biotechnology company dedicated to developing and commercializing female-specific oncology products. Its primary focus is lasofoxifene, a selective estrogen receptor modulator used for treating vulvovaginal atrophy and preventing postmenopausal osteoporosis.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on developing precision oncology therapeutics. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond positively to specific treatments, thereby enhancing the effectiveness of its drug development efforts. Acrivon's pipeline features its lead program, ACR-368 (prexasertib), which is currently in Phase 2 trials, alongside several preclinical programs targeting key components of DNA damage response and cell cycle regulation, including WEE1 and PKMYT1. By concentrating on complex oncology drug targets that are often not addressed by traditional therapies, Acrivon aims to expedite the advancement of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials.

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, founded in 2018. The company specializes in the development of innovative treatments for cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at addressing resistant hypertension and primary aldosteronism. By advancing its clinical candidates toward marketing approval, CinCor seeks to provide effective solutions for patients suffering from these conditions.

Science 37 specializes in decentralized clinical trials, offering a technology-driven approach that enables patients to participate from their homes. The company's platform connects researchers with patients via telemedicine investigators and mobile nurses, accelerating trial enrollment and retention while reaching a more diverse patient population.

A-Alpha Bio, Inc. is a biotechnology company specializing in synthetic biology and advanced quantitative measurement of protein interactions to facilitate drug development. Founded in 2017 and headquartered in Seattle, Washington, the company offers its proprietary AlphaSeq platform, which enables high-throughput mapping of protein-protein interactions. This technology allows for the simultaneous identification and quantification of interactions among thousands of protein pairs, streamlining the drug discovery process by aiding in the identification of potential drug targets and enhancing the understanding of disease mechanisms. By providing biologics and molecular glue discovery and optimization services, A-Alpha Bio supports pharmaceutical companies in optimizing binding and specificity to multiple target proteins. The team comprises synthetic biologists, structural biologists, and next-generation sequencing experts dedicated to overcoming challenges in the drug development process.

HilleVax is a clinical-stage biopharmaceutical company headquartered in Boston, Massachusetts, founded in 2021. The company specializes in the development and commercialization of innovative vaccines. Its primary focus is on HIL-214, a virus-like particle (VLP) based vaccine candidate aimed at preventing moderate-to-severe acute gastroenteritis (AGE) caused by norovirus infection. HilleVax is dedicated to addressing significant public health challenges through its vaccine initiatives.

Zentera Therapeutics is a biopharmaceutical company.

GeneDx specializes in delivering personalized insights from genomic data to improve health outcomes. It combines extensive genetic expertise with advanced data science capabilities.

Amylyx Pharmaceuticals, Inc. is a biotechnology company focused on developing innovative therapies for neurodegenerative diseases, particularly Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s disease, and Wolfram syndrome. Founded in 2013 and based in Cambridge, Massachusetts, the company is best known for its investigational therapy AMX0035, a fixed-dose co-formulation of sodium phenylbutyrate and Taurursodiol. This treatment aims to address the energy crisis within mitochondria and the accumulation of toxic, unfolded proteins in the endoplasmic reticulum, both of which contribute to the progression of neurodegenerative conditions. Through its research and development efforts, Amylyx seeks to provide effective solutions to mitigate the suffering caused by these debilitating diseases.

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's portfolio comprises several assets, including PNT2002, PNT2004, PNT2003, and PNT2001. Point Biopharma leverages its expertise in radioisotopes like Actinium-225 and Lutetium-177, along with advanced manufacturing technology and a patient-centric approach, to revolutionize theragnostic drug development and radioligand commercialization on a global scale.

Forge Biologics, Inc. is a contract development and manufacturing organization (CDMO) specializing in gene therapies and genetic medicines. Founded in 2019 and based in Columbus, Ohio, the company focuses on developing and manufacturing therapeutics, particularly for viral vector gene therapies aimed at treating genetic diseases. Notably, it offers a novel approach combining adeno-associated virus (AAV) and umbilical cord transplant to address infantile Krabbe disease, a severe neurodegenerative disorder. Additionally, Forge Biologics collaborates with scientists, physicians, biotech and pharmaceutical companies, and patient groups to facilitate the progression of gene therapy programs from preclinical development through to clinical and commercial-scale manufacturing. The company is committed to enabling access to transformative medicines for patients suffering from rare genetic diseases.

Founded in 2018, Scribe Therapeutics specializes in engineering and developing in vivo therapies based on CRISPR technology to rewrite and repair genetic disorders. Its proprietary platform, X-Editing (XE), enables greater efficacy, specificity, and deliverability compared to existing CRISPR technologies.

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 biopharmaceutical company focused on discovering and developing precision medicines that target biologically validated drivers of cancers underserved by existing therapies. The company develops epigenetic therapies, including selective histone deacetylase inhibitors, and advances candidates such as OKI-179 and OKI-422 in preclinical and clinical development. Its approach aims to directly target malignant cells, combine with other targeted therapies, and prime the tumor microenvironment to enhance immunotherapies, with a pipeline of tumor-agnostic candidates designed for optimal efficacy and tolerability.

Day One Biopharmaceuticals is a clinical-stage biotechnology company focused on developing targeted therapies for genetically defined cancers across all ages, with an initial emphasis on pediatric patients. The company aims to bring effective cancer treatments to families by advancing therapies that can benefit both children and adults, prioritizing programs that can progress to the market rapidly. Day One pursues licensing or acquisition of promising products from research institutions and industry partners to expand its pipeline. Its lead candidate, DAY101, is an oral, brain-penetrant, highly selective type II pan-RAF kinase inhibitor designed to target cancers driven by the RAF signaling pathway. By applying insights from childhood cancer biology, the company seeks to address unmet needs in oncology and broaden access to precision therapies.

Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, specializing in the development of vaccines using its innovative Multiple Antigen Presenting System (MAPS) technology. Founded in 2014, the company aims to enhance the protection against a range of infectious diseases that affect both children and adults globally. MAPS technology allows for the effective binding of protective polysaccharides and proteins within a single vaccine, inducing robust immune responses. Affinivax is advancing vaccine programs targeting significant pathogens, including Streptococcus pneumoniae, which contributes to considerable morbidity and mortality worldwide. The company leverages insights from leading experts in infectious diseases and has secured exclusive intellectual property rights related to its MAPS platform from Boston Children’s Hospital, with notable backing from the Bill & Melinda Gates Foundation.

Founded in 2018, Neuron23 is a biotechnology company based in South San Francisco, California, with an additional office in Munich, Germany. It specializes in developing and manufacturing medicines for genetic disorders using artificial intelligence technology and genetics.

Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, specializing in the development of patient-specific immunotherapies designed to combat cancer. Founded in 2016, the company focuses on personalized vaccines and T cell therapies that utilize the body's immune system to target and eliminate cancer cells. Achilles Therapeutics aims to identify and target truncal tumor neoantigens and other markers present on cancer cells, allowing for precise attacks on tumors while sparing healthy tissue. The company is currently conducting clinical trials, including the CHIRON trial for non-small-cell lung cancer and the THETIS trial for recurrent or metastatic melanoma, to evaluate the efficacy and safety of its innovative therapies.

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 founded in 2019, headquartered in Shanghai, China, with an additional office in Princeton, New Jersey. The company is dedicated to the discovery and development of therapeutic drugs aimed at treating oncology and cardiorenal diseases, as well as addressing other unmet medical needs in Greater China and major Asian markets. LianBio focuses on in-licensing assets and collaborates with leading partners to enhance its pipeline, which includes clinically validated product candidates across various therapeutic areas such as cardiovascular, oncology, ophthalmology, inflammatory diseases, and respiratory indications. The company aims to accelerate the availability of innovative medicines to improve patient outcomes through a science-driven approach.

AavantiBio, Inc. is a biopharmaceutical company focused on developing gene transfer and gene editing therapies aimed at treating rare genetic diseases. Incorporated in 2019 and based in Gainesville, Florida, the company primarily concentrates on Friedreich’s Ataxia, a rare inherited disorder that leads to significant cardiac and central nervous system dysfunction. AavantiBio is committed to transforming the lives of patients suffering from this condition by advancing innovative therapies that address unmet medical needs in the field of genetic disorders. The company’s efforts are directed toward both treatment development and research, with the goal of providing effective solutions for patients facing the challenges of rare diseases.

Nuvation Bio is a global biopharmaceutical company dedicated to developing innovative therapies for oncology, focusing on patients with difficult-to-treat cancers where conventional treatments have proven ineffective.

Escient Pharmaceuticals, Inc. is a biotechnology company based in San Diego, California, specializing in the development and manufacture of G protein-coupled receptor (GPCR)-targeted drugs aimed at treating neuro-immuno-inflammatory and autoreactive diseases. Founded in 2017, Escient focuses on harnessing the therapeutic potential of specific orphan GPCRs, particularly the Mas-Related G-Protein Receptors (Mrgprs). The company aims to provide innovative therapies for a variety of conditions, including mast cell-mediated disorders and cholestatic pruritus, thereby expanding treatment options for healthcare providers facing these complex diseases.

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 targeted therapies aimed at oncogenic drivers. The company employs a genetic selection strategy to identify patients who are most likely to benefit from its treatments, utilizing a tumor-agnostic approach that prioritizes the underlying genetics of tumors over traditional histological classifications. Its lead product candidate, milademetan, is a small molecule oral inhibitor of MDM2, which plays a role in various cancers. Additionally, Rain Oncology is advancing a preclinical program aimed at inducing synthetic lethality in cancer cells by inhibiting RAD52, further diversifying its therapeutic pipeline.

ReViral Ltd is a biotechnology company based in Stevenage, United Kingdom, specializing in the discovery and development of antiviral drugs targeting respiratory syncytial virus (RSV). Founded in 2011, ReViral is focused on creating innovative first-in-class compounds, including a highly potent fusion inhibitor designed specifically for treating severe RSV infections, particularly in vulnerable populations such as neonates. By advancing these novel antiviral treatments, ReViral aims to address significant unmet medical needs and expand the antiviral market, ultimately improving patient outcomes for those affected by RSV.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company based in San Mateo, California, focused on the discovery and development of innovative cancer therapeutics. Established in 2017, the company aims to address historically challenging cancer targets through precision medicine. Its primary product candidates include entospletinib, a selective inhibitor of spleen tyrosine kinase intended for acute myeloid leukemia patients, and KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9 for treating MYC-amplified solid tumors. Kronos Bio employs advanced technologies, such as high-throughput small-molecule microarrays and targeted protein degradation, to identify potent and selective compounds that target dysregulated transcription factors and other key drivers of oncogenic signaling.

Taysha Gene Therapies develops adeno-associated virus-based gene therapies to treat monogenic diseases of the central nervous system. Its pipeline includes treatments for GM2 gangliosidosis, CLN1 disease, Rett syndrome, SLC6A1 haploinsufficiency disorder, and Surfeit locus 1 deficiency. The company collaborates with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments.

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.

Forge Biologics, Inc. is a contract development and manufacturing organization (CDMO) specializing in gene therapies and genetic medicines. Founded in 2019 and based in Columbus, Ohio, the company focuses on developing and manufacturing therapeutics, particularly for viral vector gene therapies aimed at treating genetic diseases. Notably, it offers a novel approach combining adeno-associated virus (AAV) and umbilical cord transplant to address infantile Krabbe disease, a severe neurodegenerative disorder. Additionally, Forge Biologics collaborates with scientists, physicians, biotech and pharmaceutical companies, and patient groups to facilitate the progression of gene therapy programs from preclinical development through to clinical and commercial-scale manufacturing. The company is committed to enabling access to transformative medicines for patients suffering from rare genetic diseases.

Verona Pharma is a clinical-stage biopharmaceutical company focused on developing therapies for chronic respiratory diseases with unmet medical needs. Its lead candidate, ensifentrine, is an inhaled dual inhibitor of phosphodiesterases 3 and 4 that provides bronchodilation and anti-inflammatory effects in a single molecule. Development programs include formulations of ensifentrine for chronic obstructive pulmonary disease, with options such as nebulized administration, a dry powder inhaler, and a pressurized metered-dose inhaler. Verona Pharma is also pursuing development of ensifentrine for cystic fibrosis and asthma. The company was founded in 2005 and is headquartered in London, United Kingdom.

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 developing innovative treatments for retinal diseases and certain cancers. The company specializes in designing short biomimetic peptides that target angiogenesis and lymphangiogenesis, utilizing advanced bioinformatics and systems biology approaches. Its lead product candidates, including AXT107, AXT201, AXT301, and AXT501, are aimed at addressing conditions such as diabetic macular edema, age-related macular degeneration, and macular edema from retinal vein occlusion, all of which are significant causes of blindness in the western world. Additionally, AXT201, AXT301, and AXT501 are in early development stages for treating various solid tumors. AsclepiX Therapeutics employs cutting-edge biomaterials and drug delivery systems, including biodegradable nano and microparticles, to enhance the therapeutic efficacy of its peptide-based treatments. Incorporated in 2011 and headquartered in Baltimore, Maryland, the company is leveraging its platform technology to advance solutions in both ophthalmology and oncology.

C4 Therapeutics is a biopharmaceutical company developing targeted protein degradation therapies using its proprietary Degronimid platform. This platform enables the selective destruction of disease-causing proteins, expanding the scope of treatable conditions and potentially reducing drug resistance. The company focuses on oncology indications and has strategic collaborations with industry leaders.

C4 Therapeutics is a biopharmaceutical company developing targeted protein degradation therapies using its proprietary Degronimid platform. This platform enables the selective destruction of disease-causing proteins, expanding the scope of treatable conditions and potentially reducing drug resistance. The company focuses on oncology indications and has strategic collaborations with industry leaders.

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 is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for brain disorders. Its lead product candidate, ATH-1017, aims to treat neurodegenerative diseases like Alzheimer's and Parkinson's by restoring neuronal health.

Atea Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on discovering and developing next-generation antiviral therapeutics for severe viral diseases. 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 the SARS-CoV-2 virus. Additionally, Atea is advancing several candidates in clinical trials, including AT-787 for hepatitis C and AT-752 for dengue, both in phase 2 trials. The company aims to provide innovative treatments for infections caused by RNA and DNA viruses, improving outcomes for patients suffering from serious viral infections. Atea Pharmaceuticals was incorporated in 2012.

COMPASS Pathways is a UK-based mental health care company dedicated to accelerating patient access to innovative treatments for mental health challenges. The company focuses on developing psilocybin therapy, administered in conjunction with psychological support, for patients with treatment-resistant depression who have not responded to conventional treatments.

Dynacure is a clinical-stage drug development company dedicated to enhancing the lives of patients with rare and orphan diseases. The company focuses on creating innovative therapeutics, notably developing DYN101, an investigational antisense medicine aimed at modulating the expression of dynamin 2 for the treatment of Centronuclear Myopathies, in collaboration with Ionis Pharmaceuticals. In addition to DYN101, Dynacure is expanding its research portfolio to address other orphan disorders, including a program targeting hereditary spastic paraplegias caused by specific genetic mutations. Through its commitment to rare disease drug development, Dynacure seeks to provide effective treatment options for patients with limited therapeutic alternatives.

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, Inc. is a women's healthcare company based in Princeton, New Jersey, dedicated to the research, development, and commercialization of prescription contraceptive products. The company's lead product is Twirla, a once-weekly transdermal contraceptive patch that combines levonorgestrel and ethinyl estradiol, designed to provide a non-daily option for contraception. Agile Therapeutics is also advancing a pipeline of additional products and extensions related to Twirla, including regimens aimed at offering women extended cycles, shorter and lighter periods, and a progestin-only patch for those who cannot or prefer not to use estrogen. Utilizing proprietary Skinfusion technology, the company aims to deliver effective contraceptive solutions that address the unmet health needs of women today. Founded in 1997, Agile Therapeutics continues to focus on innovating women's healthcare options.

Lyra Therapeutics is a clinical-stage therapeutics company focused on the development and commercialization of localized treatments for ear, nose, and throat diseases. Its XTreo platform is designed to deliver medicines directly to affected tissue for sustained effect with a single administration. The company’s initial product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices intended for in-office administration to provide up to six months of continuous therapy to the sinonasal passages for chronic rhinosinusitis, with mometasone furoate as the active ingredient. Founded in 2005 and headquartered in Watertown, Massachusetts.

Cerebral Therapeutics, Inc. is a clinical-stage pharmaceutical company based in Aurora, Colorado, focused on developing innovative drug-device combination therapies for neurologic diseases, particularly refractory epilepsy. The company employs a unique implanted, refillable catheter and pump system to deliver a continuous intracerebroventricular (ICV) dose of valproic acid, an anti-epileptic drug formulated as CT-010. By utilizing reformulated off-patent drugs delivered directly to the brain's ventricles, Cerebral Therapeutics aims to bypass the blood-brain barrier, thereby enhancing treatment outcomes for patients suffering from chronic neurological conditions. Established in 2010, the company is committed to improving the quality of life for those with uncontrolled neurological disorders.

Quellis Biosciences, Inc. is a biopharmaceutical company based in Waltham, Massachusetts, established in 2017. The company is committed to discovering and developing novel monoclonal antibody therapies aimed at treating rare diseases, particularly for patients who are underserved by existing treatment options. Quellis focuses on optimizing multiple therapeutic leads to identify effective antibodies that can enhance patient outcomes, thus striving to improve the quality of life for individuals facing serious health challenges. Through its innovative approach, Quellis aims to deliver best-in-class therapies that address significant unmet medical needs in the realm of rare diseases.

Zentalis Pharmaceuticals is a clinical-stage biopharmaceutical company dedicated to developing small molecule therapeutics targeting cancer's underlying biological pathways. Its pipeline includes two lead product candidates: ZN-c5 for advanced estrogen receptor-positive breast cancer, and ZN-c3 for advanced solid tumors and platinum-resistant ovarian cancer.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

Achilles Therapeutics Ltd. is a biopharmaceutical company based in London, United Kingdom, specializing in the development of patient-specific immunotherapies designed to combat cancer. Founded in 2016, the company focuses on personalized vaccines and T cell therapies that utilize the body's immune system to target and eliminate cancer cells. Achilles Therapeutics aims to identify and target truncal tumor neoantigens and other markers present on cancer cells, allowing for precise attacks on tumors while sparing healthy tissue. The company is currently conducting clinical trials, including the CHIRON trial for non-small-cell lung cancer and the THETIS trial for recurrent or metastatic melanoma, to evaluate the efficacy and safety of its innovative therapies.

Oncorus, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative viral immunotherapies for cancer treatment. The company's lead product candidate, ONCR-177, utilizes an oncolytic herpes simplex virus type 1 platform and is currently undergoing Phase I clinical trials to assess its efficacy against various cancers. In addition to ONCR-177, Oncorus is advancing its ONCR-GBM program aimed at treating brain cancer, along with synthetic viral immunotherapies derived from Coxsackievirus A21 and Seneca Valley Virus. The company has established a clinical trial collaboration with MSD International GmbH to evaluate the safety and tolerability of ONCR-177 in combination with Merck’s KEYTRUDA immunotherapy. Founded in 2015, Oncorus is committed to improving patient outcomes and has pledged to contribute a portion of its product sales to support cancer research and care in developing regions.

Landos Biopharma, Inc. is a clinical-stage biopharmaceutical company based in Blacksburg, Virginia, focused on developing innovative oral therapeutics for autoimmune diseases, particularly inflammatory bowel disease (IBD), Crohn's disease, and ulcerative colitis. Founded in 2017, the company aims to address unmet medical needs through its unique approach to treatment, leveraging novel mechanisms of action. Its lead product candidate, NX-13, is a gut-selective NLRX1 agonist designed to modulate immune responses, and it is part of a broader pipeline that includes other internally discovered compounds targeting various pathways at the intersection of immunity and metabolism. Landos Biopharma's commitment to developing first-in-class therapeutics underscores its dedication to improving patient outcomes in the field of autoimmune disorders.

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 based in San Mateo, California, focused on the discovery and development of innovative cancer therapeutics. Established in 2017, the company aims to address historically challenging cancer targets through precision medicine. Its primary product candidates include entospletinib, a selective inhibitor of spleen tyrosine kinase intended for acute myeloid leukemia patients, and KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9 for treating MYC-amplified solid tumors. Kronos Bio employs advanced technologies, such as high-throughput small-molecule microarrays and targeted protein degradation, to identify potent and selective compounds that target dysregulated transcription factors and other key drivers of oncogenic signaling.

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, Inc. 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 specializes in genome engineering technologies and is focused on creating targeted treatments, including a pipeline of autologous and allogeneic chimeric antigen receptor T cell (CAR-T) therapies aimed at addressing hematological malignancies and solid tumors. Additionally, Poseida is advancing gene therapies for rare and life-threatening diseases, such as Ornithine transcarbamylase deficiency and methylmalonic acidemia, along with therapies for genetic liver diseases. Founded in 2014, Poseida Therapeutics is committed to improving patient outcomes through its cutting-edge research and development efforts.

Fusion Pharmaceuticals is a clinical-stage biopharmaceutical company specializing in developing radiopharmaceuticals as precision medicines. It focuses on creating targeted alpha therapeutics using its proprietary linker technology to enhance the safety and efficacy of these drugs, ultimately improving cancer patient outcomes.