Perceptive Advisors

Ocuphire Pharma is a clinical-stage biopharmaceutical company based in Farmington Hills, Michigan, that specializes in developing and commercializing therapies for various ophthalmic disorders. The company’s lead product candidate is Nyxol, a once-daily preservative-free eye drop formulation designed to treat conditions such as dim light vision disturbances, pharmacologically-induced mydriasis, and presbyopia. Nyxol is currently in late-stage clinical development, supported by extensive safety and efficacy data from previous trials. Additionally, Ocuphire is advancing APX3330, an oral tablet aimed at addressing diabetic retinopathy and diabetic macular edema, which is in Phase 2 clinical development. The company is also exploring opportunities to acquire further ophthalmic assets and seeks strategic partnerships for global commercialization of its products.

Kardigan is a patient-driven heart health firm that is revolutionizing cardiovascular drug development to produce medicines that take patients from symptom management to functional cures.

RAPT Therapeutics is a clinical-stage biopharmaceutical company specializing in immunology. It focuses on discovering, developing, and commercializing oral small-molecule therapies aimed at addressing significant unmet medical needs in oncology and inflammatory diseases. RAPT's proprietary discovery engine has led to the advancement of two drug candidates: FLX475, which selectively inhibits the migration of immunosuppressive regulatory T cells into tumors, and RPT193, which targets type 2 T helper cells in inflamed tissues. Both candidates have progressed to clinical trials within a short time frame since the company's inception. Additionally, RAPT is exploring further therapeutic targets, including general control nonderepressible 2 and hematopoietic progenitor kinase 1, which are currently in the discovery phase.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative cancer therapies through the discovery of precision medicines that target biologically validated drivers of cancer. The company focuses on less toxic kinase inhibitor drugs, particularly selective inhibitors of histone deacetylases (HDACs). Its lead candidate, OKI-179, has demonstrated significant and safe tumor growth reduction in pre-clinical models for both hematological and solid tumors, either alone or in combination with other targeted therapies. Additionally, OnKure is advancing another candidate, OKI-422, into pre-clinical toxicology studies. The company's pipeline includes tumor-agnostic therapies that aim to enhance treatment efficacy and tolerability by directly targeting malignant cells and optimizing the tumor microenvironment for improved checkpoint inhibition.

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 is based in London and was founded in March 2015 to fund and develop novel, innovative specialist focused products from large pharmaceutical or biotechnology companies. Pharmaceutical companies face increasingly difficult choices in the allocation of internal resources to their drug development programs. With significant P&L constraints and rich pipelines it has become increasingly difficult for many companies to fully fund and advance development all of their drug candidates, especially beyond Phase 2. Mereo has been formed to take advantage of the global pharmaceutical industry's drive for creative ways to progress their clinical development pipelines. Mereo has acquired an initial mid-late stage portfolio of three exceptionally well characterised novel products for the treatment of diseases with considerable unmet medical need from Novartis Pharmaceuticals. Each of these programmes has a comprehensive dataset for both pre-clinical and proof-of-concept clinical studies. Additional product opportunities are under evaluation. Mereo's focus is the development of innovative medicines that have the potential to significantly transform the lives of patients suffering from rare and other specialised conditions around the world. To do this, the Company depends on the combination of its team's expertise in selecting and acquiring product opportunities, creating value in the development pipeline and structuring creative transactions. Mereo's internal expertise is complemented by a unique partnership with a leading global CRO, ICON. Mereo combines the operational discipline and efficiency of a small company with the financial resources to conduct comprehensive clinical studies. Mereo will rapidly progress each of the products through further value inflection points before partnering or divesting its products. The Company also has the option to directly commercialise products, for example in orphan disease indications.

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

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.

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.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies aimed at transforming the treatment landscape for inflammatory bowel disease (IBD). This chronic condition, which encompasses disorders such as ulcerative colitis and Crohn's disease, involves inflammation in the gastrointestinal tract. Spyre Therapeutics combines advanced antibody engineering with rational therapeutic combinations and precision medicine strategies to enhance patient selection. The company's goal is to create next-generation products that improve upon existing treatment options, which include anti-inflammatory drugs, immunosuppressants, and biologics. Through its robust pipeline, Spyre Therapeutics seeks to address the unmet needs of patients suffering from IBD.

Precision BioSciences, Inc. is a biotechnology company based in Durham, North Carolina, focused on genome editing and the development of therapeutic products. Utilizing its proprietary ARCUS genome editing platform, the company aims to create innovative solutions for human diseases and food applications. Precision BioSciences operates through two primary segments: Therapeutic and Food. The Therapeutic segment is engaged in developing allogeneic CAR T immunotherapies, with several candidates in clinical trials targeting various types of cancers, including acute lymphoblastic leukemia and multiple myeloma. Additionally, the company is involved in in vivo gene correction activities and has collaborations for developing treatments for chronic Hepatitis B. The Food segment concentrates on creating nutrition products. Founded in 2006, Precision BioSciences is dedicated to translating advanced gene editing technology into impactful products across the life sciences.

Crinetics Pharmaceuticals is a clinical-stage pharmaceutical company dedicated to the discovery and development 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 for the treatment of acromegaly and neuroendocrine tumors. In addition to paltusotine, Crinetics is advancing other candidates, including CRN01941, an oral selective nonpeptide sst2 biased agonist for neuroendocrine tumors, an oral selective somatostatin type 5 receptor agonist for congenital hyperinsulinism, and an oral adrenocorticotropic hormone antagonist targeting Cushing’s disease and congenital adrenal hyperplasia. Founded in 2008 and headquartered in San Diego, California, Crinetics Pharmaceuticals aims to leverage its internal discovery efforts and objective hormonal biomarker endpoints to effectively navigate the development process and bring meaningful treatments to market.

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.

Mainstay Medical is a global medical device company based in Dublin, Ireland, with operations in the United States and Australia. The company focuses on developing innovative therapies for individuals suffering from chronic low back pain, a condition that affects a significant portion of the population. Mainstay Medical has created a FDA-approved restorative treatment aimed at addressing mechanical chronic low back pain. This treatment is designed to help restore muscle control and enhance functionality in the lumbar spine, ultimately improving the quality of life for patients. By collaborating with scientists and clinical experts, Mainstay Medical strives to provide effective solutions for those in need of relief from debilitating pain.

Impulse Dynamics N.V. is a medical device company focused on developing electrical therapies for chronic heart failure, operating primarily in the United States and Europe. The company's key innovation is its cardiac contractility modulation (CCM) therapy, which utilizes non-excitatory electrical impulses delivered to the heart muscles. This therapy aims to enhance cardiac strength and improve blood flow, offering a minimally invasive treatment option for patients with limited alternatives. Impulse Dynamics conducts clinical trials to assess the safety and efficacy of its therapies and collaborates with universities and hospitals for research initiatives. Founded in 1996 and headquartered in Willemstad, Curacao, the company also maintains offices in New York and Hong Kong, alongside product development centers in New Jersey and Israel.

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

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

Astria Therapeutics is a biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative therapies for rare and niche allergic and immunological diseases. The company focuses on bringing life-changing treatments to patients and families affected by these conditions. Its lead program, STAR-0215, is a monoclonal antibody that inhibits plasma kallikrein and is currently in preclinical development for hereditary angioedema. Additionally, Astria is developing STAR-0310, a monoclonal antibody OX40 antagonist, which is also in preclinical stages and targets atopic dermatitis, an immune disorder characterized by compromised skin barrier function and associated itching.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies aimed at transforming the treatment landscape for inflammatory bowel disease (IBD). This chronic condition, which encompasses disorders such as ulcerative colitis and Crohn's disease, involves inflammation in the gastrointestinal tract. Spyre Therapeutics combines advanced antibody engineering with rational therapeutic combinations and precision medicine strategies to enhance patient selection. The company's goal is to create next-generation products that improve upon existing treatment options, which include anti-inflammatory drugs, immunosuppressants, and biologics. Through its robust pipeline, Spyre Therapeutics seeks to address the unmet needs of patients suffering from IBD.

Soleno Therapeutics, Inc. is a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative treatments 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). This product is currently undergoing evaluation in a Phase III clinical development program. Soleno Therapeutics was incorporated in 1999 and is based in Redwood City, California. The company was previously known as Capnia, Inc. before rebranding in May 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 is developing innovative treatments for inflammatory skin diseases, with its lead product, FB-401, being a topically applied live biotherapeutic created in partnership with the National Institutes of Health and the National Institute of Allergy and Infectious Diseases. Additionally, Forte is advancing through preclinical trials for another product candidate, FB-102, which is a proprietary therapeutic molecule that may have applications in treating various autoimmune conditions, including graft-versus-host disease, vitiligo, and alopecia areata.

Lyra Therapeutics, Inc. is a clinical-stage therapeutics company focused on the development and commercialization of novel integrated drug and delivery solutions for the localized treatment of patients with ear, nose and throat diseases. The company’s proprietary technology platform, XTreo, is designed to precisely and consistently deliver medicines directly to the affected tissue for sustained periods with a single administration. The company’s initial product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed to be administered in a brief, non-invasive, in-office procedure and intended to deliver up to six months of continuous drug therapy to the sinonasal passages for the treatment of CRS. The therapeutic embedded within LYR-210 and LYR-220 is mometasone furoate, which is the active ingredient in various FDA-approved drugs and has a well-established efficacy and safety profile.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative cancer therapies through the discovery of precision medicines that target biologically validated drivers of cancer. The company focuses on less toxic kinase inhibitor drugs, particularly selective inhibitors of histone deacetylases (HDACs). Its lead candidate, OKI-179, has demonstrated significant and safe tumor growth reduction in pre-clinical models for both hematological and solid tumors, either alone or in combination with other targeted therapies. Additionally, OnKure is advancing another candidate, OKI-422, into pre-clinical toxicology studies. The company's pipeline includes tumor-agnostic therapies that aim to enhance treatment efficacy and tolerability by directly targeting malignant cells and optimizing the tumor microenvironment for improved checkpoint inhibition.

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.

Biodesix, Inc. is a diagnostic solutions company based in Boulder, Colorado, specializing in lung cancer diagnostics and biopharmaceutical services. The company offers a range of blood-based tests designed to assist physicians in evaluating the risk of malignancy in patients with suspicious lung nodules, including the Nodify XL2 and Nodify CDT tests. Additionally, it provides GeneStrat tumor profiling and VeriStrat immune profiling tests that enable more informed treatment decisions. Biodesix utilizes a proprietary mass spectrometry-based platform for the development of multivariate protein diagnostics, with its first product, VeriStrat, aiding oncologists in choosing between specific therapies for advanced lung cancer patients. The tests are processed in a CLIA-certified laboratory, with results delivered within 72 hours. Besides its diagnostic offerings, Biodesix collaborates with biopharmaceutical companies to develop companion diagnostics, enhancing the therapeutic effectiveness of various agents. Incorporated in 2005, Biodesix is committed to improving patient outcomes through innovative diagnostic solutions.

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.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

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.

Lyra Therapeutics, Inc. is a clinical-stage therapeutics company focused on the development and commercialization of novel integrated drug and delivery solutions for the localized treatment of patients with ear, nose and throat diseases. The company’s proprietary technology platform, XTreo, is designed to precisely and consistently deliver medicines directly to the affected tissue for sustained periods with a single administration. The company’s initial product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed to be administered in a brief, non-invasive, in-office procedure and intended to deliver up to six months of continuous drug therapy to the sinonasal passages for the treatment of CRS. The therapeutic embedded within LYR-210 and LYR-220 is mometasone furoate, which is the active ingredient in various FDA-approved drugs and has a well-established efficacy and safety profile.

VivoSense, also known as Vivonoetics, is a data analysis and software company focused on providing wearable sensor physiological monitoring solutions for research and clinical trials. The company develops digital clinical measures designed to enhance patient research and care by delivering digital biomarkers derived from wearable sensor data. This platform allows researchers to collect and analyze real-world data accurately, facilitating informed decision-making and risk evaluation associated with various medical treatments in regulated clinical environments. Through its innovative approach, VivoSense aims to improve the efficiency and effectiveness of clinical research.

DNAnexus, Inc. is a provider of cloud-based genome informatics and data management tools aimed at enterprises engaged in genomic medicine. Founded in 2009 and headquartered in Mountain View, California, the company offers a platform that facilitates the analysis of raw sequencing data and integration with clinical and phenotypic data, promoting seamless collaboration among users. Key offerings include MOSAIC Microbiome, a platform for collaborative microbiome research; DNAnexus Apollo, which focuses on clinico-genomic data exploration; and DNAnexus Titan, a comprehensive data analysis solution. Additionally, DNAnexus Portals provide secure online workspaces for cross-disciplinary collaboration. The Clinico-Genomic Data Solution program addresses the need for disease-specific datasets by building networks with healthcare partners to enhance diagnosis and treatment pathways. DNAnexus serves a diverse client base, including biopharmaceutical companies, genome centers, and diagnostic test providers, and has established a strategic alliance with WuXi NextCODE Genomics. The company also maintains additional offices in San Francisco, London, and Prague.

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.

Orchestra BioMed, Inc. is a biomedical innovation company focused on developing high impact, high value, evidence-based therapeutic solutions for medical procedures that address major medical conditions. Its main focus is on developing new therapies for cardiovascular diseases which are the leading cause of death worldwide, taking the lives of 17.7 million people each year, or 31% of all global deaths according to the World Health Organization. It was founded in 2017 and is headquartered in New Hope, Pennsylvania.

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.

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.

GeneDx is dedicated to providing personalized health insights that enhance diagnosis, treatment, and drug discovery. By integrating extensive genomic expertise with advanced data interpretation capabilities, the company aims to establish precision medicine as a standard in healthcare. GeneDx specializes in exome and genome testing and interpretation, leveraging one of the largest rare disease data sets available. Its operations are divided into two segments: GeneDx, which generates the majority of revenue, and Legacy Sema4 diagnostics. Through these efforts, GeneDx is positioned to significantly impact health outcomes by using genomic and large-scale clinical information.

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. is a clinical-stage pharmaceutical company dedicated to the development and commercialization of radioligand therapies for cancer treatment. The company is focused on creating a robust platform for the clinical development of radiopharmaceuticals, utilizing advanced radioisotopes such as Actinium-225 and Lutetium-177. POINT Biopharma's product pipeline includes several candidates, such as PNT2002, PNT2004, PNT2003, and PNT2001, among others. By leveraging a combination of innovative manufacturing technology and direct-to-patient targeting, POINT Biopharma aims to transform theragnostic drug development and enhance the commercialization of radioligands in the fight against cancer.

RayzeBio, Inc. is a biotechnology company based in San Diego, California, founded in 2020. It specializes in developing tumor-targeted small molecule medicines that utilize the therapeutic potential of radioisotopes, particularly Actinium-225, an alpha-emitting radioisotope aimed at treating solid tumors. RayzeBio is focused on enhancing cancer treatment outcomes by creating a robust pipeline of radiopharmaceutical candidates targeting validated oncology drug targets. The company's portfolio includes a range of drug candidates at various stages of development, from discovery to late-stage clinical programs, addressing significant market opportunities in the field of cancer therapeutics.

Born from the founders’ recognition that their diverse but complementary skills and experiences would enable them to successfully address challenges that others had not, the company set about solving a vexing problem: how to bring true proteomics to the world in a way that accelerates therapeutic development, dramatically improves medical diagnostics, and makes personalized and predictive medicine a reality. The extraordinary team at Nautilus represents a wide spectrum of disciplines and expertise, including protein chemists, chip designers, molecular biologists, data scientists, material scientists, biophysicists, optical engineers, microfluidics engineers, bioinformaticists, software engineers, and more. Nautilus is positioned to revolutionize proteomics, transform the way drugs are developed, and significantly improve the way human health is managed.

NiKang Therapeutics is an early-stage biotech company focused on discovering and developing innovative small molecule oncology medicines to help patients with unmet medical needs. Its discovery approach is informed by target structure biology and capitalizes on structure-based drug design enabling a rapid and efficient discovery and development of proprietary drug candidates with the most desirable pharmacological features into clinical studies.

Renovia Inc. was a Boston-based healthcare company founded in 2016, focused on developing prescription digital therapeutics for pelvic floor disorders. The company aimed to enhance the lives of women suffering from conditions such as stress, mixed, and urgent urinary incontinence, including overactive bladder. Renovia's innovative device provided a clinically proven, non-surgical, drug-free treatment option, allowing for real-time visualization of pelvic movement during muscle training, thereby facilitating effective therapy. However, in late 2022, Renovia ceased operations following a strategic review by its investors, which included the consideration of a potential sale. In January 2023, most of the company's assets, including its intellectual property, were acquired by Axena Health, Inc.

Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines using its innovative virus-like particle (VLP) technology platform, which allows for the multivalent display of complex viral antigens. This technology aims to provide broad and durable protection against various infectious diseases, with an initial focus on life-threatening respiratory illnesses. Icosavax's pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and SARS-CoV-2. The company is dedicated to advancing its VLP technology to discover, develop, and ultimately commercialize effective vaccines against these infectious diseases.

Pyxis Oncology is a developer of antibody therapeutics intended to promote the body's immune response to cancer. The company has engaged in deep analyses of tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and of tumor cell signaling pathways within cold tumors, providing laboratories with development of novel antibody-based immunotherapies. It was founded in 2019 and is based in Boston, Massachusetts.

Crossover Health, Inc. provides comprehensive workplace healthcare services tailored for employers and their employees. Founded in 2010, the company is headquartered in Aliso Viejo, California, with health centers located in various regions, including Silicon Valley. Crossover Health offers a wide range of services, including primary care, urgent care, chronic disease management, preventive care, health risk assessments, and health education. Additionally, it provides ancillary services such as physical therapy, chiropractic care, acupuncture, and care coordination, along with health analytics and remote care options. The company's focus is on creating a positive employee care experience that promotes a healthy and motivated workforce while delivering financial benefits to organizations.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

OnKure Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative cancer therapies through the discovery of precision medicines that target biologically validated drivers of cancer. The company focuses on less toxic kinase inhibitor drugs, particularly selective inhibitors of histone deacetylases (HDACs). Its lead candidate, OKI-179, has demonstrated significant and safe tumor growth reduction in pre-clinical models for both hematological and solid tumors, either alone or in combination with other targeted therapies. Additionally, OnKure is advancing another candidate, OKI-422, into pre-clinical toxicology studies. The company's pipeline includes tumor-agnostic therapies that aim to enhance treatment efficacy and tolerability by directly targeting malignant cells and optimizing the tumor microenvironment for improved checkpoint inhibition.

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.

Beam Therapeutics Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing precision genetic medicines using its innovative base editing technology. Founded in 2017, the company aims to provide lifelong cures for patients with serious diseases. Its therapeutic portfolio includes treatments for sickle cell disease, beta-thalassemia, pediatric T-cell acute lymphoblastic leukemia, pediatric acute myeloid leukemia, alpha-1 antitrypsin deficiency, and glycogen storage disorder 1A, as well as therapies for ocular and central nervous system disorders. Beam's base editing technology allows for targeted modifications at a single base in the genome without creating double-stranded breaks in DNA, positioning it as a pioneer in the field of genetic medicine. The company's pipeline includes several programs, such as BEAM-101, ESCAPE, BEAM-302, BEAM-301, and BEAM-201, reflecting its commitment to advancing genetic therapies.

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.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Adagio Medical is engaged in the research and development of treatments for cardiovascular diseases. Adagio Medical is committed to providing electrophysiologists innovative cryoablation catheters that create safe, continuous, transmural lesions to treat cardiac arrhythmias anywhere in the heart, including paroxysmal and persistent atrial fibrillation, atrial flutter, and ventricular tachycardia. The company was founded in 2011 and is based in Laguna Hills, California.

Prometheus Biosciences, formerly known as Prometheus Laboratories, is a specialty pharmaceutical company headquartered in San Diego, California. The company develops and commercializes innovative pharmaceutical and diagnostic products aimed at enhancing individualized patient care, particularly in the fields of gastroenterology and autoimmune diseases. Prometheus offers a range of diagnostic tests, including assays for monitoring drug and anti-drug antibody levels, as well as tests for inflammatory bowel disease and celiac disease. Among its marketed products are Entocort EC, indicated for Crohn's disease, and Imuran, used in kidney transplantation and rheumatoid arthritis management. Additionally, the company is involved in developing new treatments, such as COLAL-PRED, for gastrointestinal disorders. Through its advanced diagnostics and therapeutic offerings, Prometheus Biosciences assists healthcare providers in optimizing treatment strategies and improving patient outcomes.

Impulse Dynamics N.V. is a medical device company focused on developing electrical therapies for chronic heart failure, operating primarily in the United States and Europe. The company's key innovation is its cardiac contractility modulation (CCM) therapy, which utilizes non-excitatory electrical impulses delivered to the heart muscles. This therapy aims to enhance cardiac strength and improve blood flow, offering a minimally invasive treatment option for patients with limited alternatives. Impulse Dynamics conducts clinical trials to assess the safety and efficacy of its therapies and collaborates with universities and hospitals for research initiatives. Founded in 1996 and headquartered in Willemstad, Curacao, the company also maintains offices in New York and Hong Kong, alongside product development centers in New Jersey and Israel.

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 developing innovative therapies for oncology, addressing unmet medical needs in the treatment of challenging cancers that have not responded to conventional therapies. Founded in 2018 and based in New York, with an additional office in San Francisco, the company has a portfolio of drug development candidates aimed at improving patient outcomes. One of its key clinical-stage candidates is NUV-868, an oral small molecule BET inhibitor that targets the BRD4 protein, which plays a crucial role in regulating tumor growth and differentiation. Additionally, Nuvation Bio is advancing its proprietary small molecule Drug-Drug Conjugate platform, further expanding its therapeutic arsenal in oncology.

Metabolon, Inc. is a health technology company specializing in metabolomics, which focuses on the analysis of metabolites to aid in disease detection and precision medicine. The company develops a range of clinical diagnostic tests, including QUANTOSE IR for early-stage insulin resistance and Meta IMD for identifying metabolites linked to inherited metabolic disorders. Metabolon also offers customizable research services and multiomics solutions, enabling academic and commercial researchers to explore various life sciences areas effectively. The company's technologies address a wide array of health conditions, including cardiovascular issues, diabetes, and cancer, and are utilized across sectors such as biotechnology, pharmaceuticals, and nutrition. Founded in 2000 and headquartered in Morrisville, North Carolina, Metabolon has a global presence, including offices in Munich, London, Madrid, and China, and maintains strategic partnerships with major firms in the pharmaceutical and consumer health industries.

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.

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.

Metabolon, Inc. is a health technology company specializing in metabolomics, which focuses on the analysis of metabolites to aid in disease detection and precision medicine. The company develops a range of clinical diagnostic tests, including QUANTOSE IR for early-stage insulin resistance and Meta IMD for identifying metabolites linked to inherited metabolic disorders. Metabolon also offers customizable research services and multiomics solutions, enabling academic and commercial researchers to explore various life sciences areas effectively. The company's technologies address a wide array of health conditions, including cardiovascular issues, diabetes, and cancer, and are utilized across sectors such as biotechnology, pharmaceuticals, and nutrition. Founded in 2000 and headquartered in Morrisville, North Carolina, Metabolon has a global presence, including offices in Munich, London, Madrid, and China, and maintains strategic partnerships with major firms in the pharmaceutical and consumer health industries.

ReViral is a developer of antiviral drugs designed to help patients and enlarge the antiviral market by developing novel first-in-class compounds. The company's antiviral drugs is a fusion inhibitor of RSV which is a highly potent, novel small molecule and is being developed to treat RSV in patients with severe disease including neonates, enabling patients to be treated who are infected with respiratory syncytial virus (RSV) by developing novel compounds.

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.

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. Established in 2018 and based in Cambridge, Massachusetts, the company is advancing a diversified pipeline that includes several clinical-stage investigational therapies targeting conditions such as schizophrenia, epilepsy, Parkinson's disease, and substance use disorder. Notable products in development include CVL-231, a positive allosteric modulator currently in Phase Ib trials for schizophrenia, and CVL-865, also a positive allosteric modulator, which is in Phase II trials for drug-resistant focal onset seizures. Additionally, Tavapadon, a selective dopamine D1/D5 partial agonist, is in a Phase III program for Parkinson's disease, while CVL-936, a selective dopamine D3-preferring antagonist, is undergoing Phase I trials for substance use disorder. The company is also engaged in preclinical research aiming to address various neuroscience indications, leveraging expertise in neurocircuitry and receptor selectivity.

Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, that aims to repair or reverse damage from various degenerative diseases by utilizing the body’s own biological mechanisms. The company employs a Progenitor Cell Activation approach, which involves the use of small molecules to activate progenitor cells and promote the regeneration of functional tissue. Its leading product candidate, FX-322, is currently undergoing phase 2a clinical trials to address the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is also exploring treatments for a range of other conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. The company has established a collaboration with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics focuses on disease modification without the complexities associated with genetic engineering.

Pear Therapeutics, Inc. is a healthcare company specializing in the discovery, development, and delivery of software-based therapeutics aimed at treating behavioral health disorders, including addiction, schizophrenia, anxiety, depression, and chronic insomnia. Founded in 2013 and headquartered in Boston, with an additional office in San Francisco, Pear Therapeutics offers a range of digital therapies designed to enhance treatment outcomes. Notable products include reSET, a tool for substance abuse treatment used alongside traditional therapy; reSET-O, which addresses opioid use disorder; and Somryst, aimed at chronic insomnia. The company also provides PearConnect, a patient services center that connects users with advocates, clinicians, and insurance providers to facilitate support and resource access throughout treatment. By integrating digital interventions with pharmaceuticals, Pear Therapeutics aims to improve patient engagement, clinical outcomes, and cost-effectiveness in healthcare.

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

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.

4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing innovative gene therapy products to address unmet medical needs. The company utilizes targeted and evolved adeno-associated virus vectors to create a diverse portfolio of product candidates focused on ophthalmology, cardiology, and pulmonology. Among its key projects are 4D-125, currently in a Phase I/II clinical trial for X-linked retinitis pigmentosa; 4D-110, in a Phase I clinical trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is advancing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. Founded in 2013, the company is dedicated to transforming the treatment landscape in these therapeutic areas.

Fulcrum Therapeutics is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing therapies for patients with rare genetic diseases. The company is dedicated to addressing unmet medical needs, particularly in the realm of genetically defined conditions. Its lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy (FSHD), a progressive muscle-wasting disorder. Additionally, Fulcrum is developing FTX-6058, aimed at increasing fetal hemoglobin levels in patients with sickle cell disease and beta-thalassemia. The company also engages in research to identify drug targets for various conditions, including Duchenne muscular dystrophy and neurological diseases. Fulcrum Therapeutics has established collaborations with Acceleron Pharma Inc. and MyoKardia, Inc. to explore new therapeutic avenues in pulmonary disease and genetic cardiomyopathies, respectively. Founded in 2015, Fulcrum aims to enhance the quality of life for patients suffering from these challenging disorders.

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.

Born from the founders’ recognition that their diverse but complementary skills and experiences would enable them to successfully address challenges that others had not, the company set about solving a vexing problem: how to bring true proteomics to the world in a way that accelerates therapeutic development, dramatically improves medical diagnostics, and makes personalized and predictive medicine a reality. The extraordinary team at Nautilus represents a wide spectrum of disciplines and expertise, including protein chemists, chip designers, molecular biologists, data scientists, material scientists, biophysicists, optical engineers, microfluidics engineers, bioinformaticists, software engineers, and more. Nautilus is positioned to revolutionize proteomics, transform the way drugs are developed, and significantly improve the way human health is managed.

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.

COMPASS Pathways plc is a mental health care company based in the United Kingdom and the United States, focusing on developing innovative therapies for treatment-resistant depression. The company is conducting Phase IIb clinical trials for its psilocybin formulation, COMP360, which aims to provide a new treatment option for patients who have not responded to conventional therapies. By researching psilocybin-assisted therapy, COMPASS Pathways seeks to assess its safety and efficacy, while combining the psychedelic compound with psychological support. The company was incorporated in 2020 and aims to address the significant unmet need for effective and accessible mental health treatments, striving to empower individuals facing mental health challenges.

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.

Lyra Therapeutics, Inc. is a clinical-stage therapeutics company focused on the development and commercialization of novel integrated drug and delivery solutions for the localized treatment of patients with ear, nose and throat diseases. The company’s proprietary technology platform, XTreo, is designed to precisely and consistently deliver medicines directly to the affected tissue for sustained periods with a single administration. The company’s initial product candidates, LYR-210 and LYR-220, are bioresorbable polymeric matrices designed to be administered in a brief, non-invasive, in-office procedure and intended to deliver up to six months of continuous drug therapy to the sinonasal passages for the treatment of CRS. The therapeutic embedded within LYR-210 and LYR-220 is mometasone furoate, which is the active ingredient in various FDA-approved drugs and has a well-established efficacy and safety profile.

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.

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 is a precision oncology medicine company focused on discovering and developing small molecule therapies that are tumor-agnostic. The company’s lead product candidate, BDTX-189, targets non-canonical and oncogenic mutations in ErbB kinases, specifically the epidermal growth factor receptor and tyrosine-protein kinase. In addition to BDTX-189, Black Diamond is developing allosteric-EGFR mutation inhibitors and other early-stage pipeline programs that address allosteric mutations in kinases associated with cancer and rare genetic diseases. The company's approach is centered on its Mutation-Allostery-Pharmacology platform, which aims to create targeted treatments for genetically defined cancers, especially for patients with limited existing treatment options. Founded in 2014 and headquartered in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its name change in January 2018.

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.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, that aims to repair or reverse damage from various degenerative diseases by utilizing the body’s own biological mechanisms. The company employs a Progenitor Cell Activation approach, which involves the use of small molecules to activate progenitor cells and promote the regeneration of functional tissue. Its leading product candidate, FX-322, is currently undergoing phase 2a clinical trials to address the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is also exploring treatments for a range of other conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. The company has established a collaboration with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics focuses on disease modification without the complexities associated with genetic engineering.

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.

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.