Perceptive Advisors

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.

Beta Bionics is a biotechnology company focused on developing an integrated bionic pancreas system known as the iLet. This innovative device aims to alleviate the challenges and costs associated with diabetes care by providing a dual-chamber infusion pump that delivers insulin and glucagon with precision. The iLet autonomously calculates and administers the necessary doses, enabling patients to effectively manage their blood sugar levels. The system has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal options, and is compatible with Zealand Pharma’s dasiglucagon, a glucagon analogue known for its stability in a ready-to-use aqueous solution. Through this technology, Beta Bionics seeks to improve the quality of life for individuals living with diabetes.

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.

Orpyx Medical Technologies Inc. specializes in developing mobility sensor devices aimed at diabetic patients. The company's flagship product, the Orpyx SI sensory insoles, employs advanced sensor technology to monitor pressure, temperature, and movement, thereby helping to manage and prevent diabetic foot ulcers and limb loss. Additionally, Orpyx offers Kinetyx, a mobile in-shoe pressure management system that captures clinical plantar pressure data for sports medicine and footwear design, along with SurroGait Rx, which aids multiple sclerosis patients in enhancing mobility and balance. Founded in 2010 and headquartered in Calgary, Canada, Orpyx Medical Technologies is committed to empowering individuals with diabetes to maintain their mobility while promoting a prevention-focused healthcare model.

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.

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

SOPHiA GENETICS is a healthcare technology company focused on advancing data-driven medicine and enhancing life sciences research. The company has developed the SOPHiA DDM™ Platform, a cloud-based software-as-a-service solution that analyzes complex multimodal data sets and various diagnostic modalities. This platform is utilized by over 780 hospitals, laboratories, and biopharma institutions worldwide. Headquartered in Switzerland, SOPHiA GENETICS also maintains a presence in France, the United States, and Brazil, underscoring its commitment to improving healthcare outcomes through innovative technology.

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

Zevra Therapeutics is a development-stage biopharmaceutical company dedicated to the discovery and development of innovative therapies for pain, ADHD, and other central nervous system diseases. The company is particularly focused on creating safer, abuse-resistant opioid pain relievers. Utilizing its proprietary Ligand Activated Therapy (LAT) platform, Zevra aims to enhance existing drugs and streamline the development process while safeguarding its intellectual property. Additionally, Zevra addresses the needs of patients with rare diseases by employing unique, data-driven strategies to overcome challenges in drug development. The company's mission centers on delivering transformative therapies to individuals facing limited or no treatment options, ultimately striving to improve the quality of life for those affected by these conditions.

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

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

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

Capstan Therapeutics is a biotechnology company focused on enhancing the therapeutic potential of in vivo RNA-based therapies. It develops proprietary targeted delivery systems, including targeted lipid nanoparticles, to improve the efficacy and control of dosage in engineered cell therapies. This innovative platform aims to address a variety of diseases, particularly in the fields of oncology, fibrosis, and inflammation, where there are significant unmet clinical needs. By advancing precision in vivo cell engineering, Capstan Therapeutics seeks to create transformative therapeutics that can offer new solutions for patients facing these challenging conditions.

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.

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.

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.

Freenome, Inc. is a biotechnology company based in South San Francisco, California, founded in 2014. It specializes in developing artificial intelligence-driven genomic solutions for the early detection of cancer. The company focuses on creating simple, non-invasive blood tests that can identify early-stage cancer and assist in treatment selection, thus enabling proactive disease management. Freenome’s multi-omics platform employs proprietary algorithms to enhance disease screening processes, aiming to facilitate timely interventions and improve treatment effectiveness. In addition to its diagnostic products, Freenome also offers clinical research services to support its mission of transforming cancer management through systematic early detection.

enGene is a developer of a mucosal immunotherapy platform designed to treat inflammatory bowel disease and diabetes. The company's mucosal immunotherapy platform has developed a flexible nucleotide delivery technology targeting mucosal tissues to treat numerous prevalent, chronic diseases via the induction or suppression of protein expression levels, enabling physicians and doctors to regenerate physiologic, meal-regulated insulin secretion from the gut of subjects with diabetes.

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.

enVVeno Medical Corp is a medical device company dedicated to advancing the treatment of venous disease through the development of bioprosthetic devices. The company's flagship product, the VenoValve, represents a pioneering surgical implant designed to address severe deep venous Chronic Venous Insufficiency (CVI). By creating innovative tissue-based solutions, enVVeno Medical aims to enhance the standard of care for patients suffering from debilitating venous conditions. The company is committed to improving patient outcomes while streamlining surgical procedures for healthcare providers. Based in Irvine, California, enVVeno Medical continues to focus on developing technologies that promise to transform the management of venous diseases.

Beta Bionics is a biotechnology company focused on developing an integrated bionic pancreas system known as the iLet. This innovative device aims to alleviate the challenges and costs associated with diabetes care by providing a dual-chamber infusion pump that delivers insulin and glucagon with precision. The iLet autonomously calculates and administers the necessary doses, enabling patients to effectively manage their blood sugar levels. The system has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal options, and is compatible with Zealand Pharma’s dasiglucagon, a glucagon analogue known for its stability in a ready-to-use aqueous solution. Through this technology, Beta Bionics seeks to improve the quality of life for individuals living with diabetes.

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.

AsclepiX Therapeutics represents the next generation of biologics and drug development technology. They use bioinformatics and systems biology methods to design classes of short biomimetic anti-angiogenic (anti-neovascular), and anti-lymphangiogenic peptides. They also use the latest developments in biomaterials and drug delivery to design long-lasting biodegradable nano and microparticles to deliver these biomimetic therapeutic peptides throughout the body. AsclepiX Therapeutics utilizes its platform technology for indications in ophthalmology, oncology, and for other angiogenesis and lymphangiogenesis-dependent diseases.

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

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.

Satellos Bioscience is a private regenerative medicine company dedicated to developing innovative therapeutics aimed at stimulating or restoring muscle regeneration, particularly in severe disorders. The company's primary focus is on creating an oral therapeutic drug for Duchenne muscular dystrophy, targeting the dysregulation that is recognized as a fundamental cause of the disease's progressive characteristics. Through its research and development efforts, Satellos aims to address significant unmet medical needs within the field of muscle-related conditions.

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.

Anteris Technologies Ltd is a structural heart company based in Toowong, Australia, that focuses on the development and commercialization of innovative medical technologies and devices. The company is known for its DurAVR transcatheter heart valve, designed for younger patients requiring a durable heart valve that mimics the natural blood flow of a healthy aortic valve. Additionally, Anteris is engaged in the research and development of the ComASUR delivery system, which enhances the precision of valve placement. The company also manufactures and distributes proprietary ADAPT regenerative tissue products and is involved in regenerative medicine and immunotherapies. Anteris Technologies aims to provide clinically superior solutions that enable healthcare professionals to achieve life-changing outcomes for patients.

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.

Zenas BioPharma is a biopharmaceutical company that focuses on developing immune-based therapies for patients. The company is headquartered in Florida, USA.

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.

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

Surf Bio is a preclinical biopharmaceutical company leveraging a breakthrough next-generation surfactant to develop enhanced therapeutic solutions for diabetes, oncology, infectious diseases, gene therapy, and other therapeutic areas.

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.

Beta Bionics is a biotechnology company focused on developing an integrated bionic pancreas system known as the iLet. This innovative device aims to alleviate the challenges and costs associated with diabetes care by providing a dual-chamber infusion pump that delivers insulin and glucagon with precision. The iLet autonomously calculates and administers the necessary doses, enabling patients to effectively manage their blood sugar levels. The system has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal options, and is compatible with Zealand Pharma’s dasiglucagon, a glucagon analogue known for its stability in a ready-to-use aqueous solution. Through this technology, Beta Bionics seeks to improve the quality of life for individuals living with diabetes.

Synthego Corporation is a genome engineering company focused on advancing life science research and therapeutic development to improve human health. Founded in 2012 and headquartered in Redwood City, California, Synthego offers a comprehensive platform that integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology. The company's product portfolio includes engineered cells, CRISPR kits, and bioinformatics tools designed for various applications such as pathway analysis, target validation, disease modeling, and diagnostics. By leveraging machine learning and automation, Synthego enables researchers in biotechnology and academia to conduct efficient and precise gene editing, facilitating rapid advancements in drug discovery and development. The company serves a global customer base, distributing its products through various channels, including online sales and partnerships in multiple countries.

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.

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

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

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

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

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

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

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

HilleVax is a biopharmaceutical company that focuses on the development and commercialization of novel vaccines. HilleVax was founded in 2021 and was headquartered in Boston, Massachusetts.

enVVeno Medical Corp is a medical device company dedicated to advancing the treatment of venous disease through the development of bioprosthetic devices. The company's flagship product, the VenoValve, represents a pioneering surgical implant designed to address severe deep venous Chronic Venous Insufficiency (CVI). By creating innovative tissue-based solutions, enVVeno Medical aims to enhance the standard of care for patients suffering from debilitating venous conditions. The company is committed to improving patient outcomes while streamlining surgical procedures for healthcare providers. Based in Irvine, California, enVVeno Medical continues to focus on developing technologies that promise to transform the management of venous diseases.

Emulate, Inc. specializes in organ-on-chip technology that simulates human biology to enhance the understanding of how diseases, medications, chemicals, and food impact human health. The company develops organ-chips for various organs, including the lung, intestine, liver, and skin, which replicate normal functions and disease conditions. Emulate's innovative technology offers researchers a more accurate alternative to traditional experimental methods, such as cell cultures and animal testing, by providing detailed insights into human biological responses. Additionally, the company supplies drug development and instrumentation software to facilitate research and development workflows for its clients. Emulate serves a diverse range of sectors, including biotechnology, pharmaceuticals, consumer health, cosmetics, chemicals, food, agrochemicals, government agencies, and academic institutions. Founded in 2013 and headquartered in Boston, Massachusetts, Emulate was previously known as Emulate Living MicroDevices, Inc. until its name change in 2014.

SomaLogic, Inc. is a commercial-stage company based in Boulder, Colorado, specializing in protein biomarker discovery and clinical diagnostics. The company utilizes its proprietary technology platform, SomaScan, which allows for the measurement of proteins with a precision comparable to DNA measurement. This technology enhances the understanding of the molecular bases of health and disease, surpassing traditional genomic methods. SomaLogic develops and offers SOMAmers, modified nucleic acid-based protein-binding reagents, and provides diagnostic tests and protein measurement solutions for various diseases, including those in oncology, neurology, and cardiovascular health. Their products serve multiple applications, including life sciences research, therapeutic drug development, and clinical diagnostics, making significant contributions to the fields of biological and medical sciences. Founded in 1999, SomaLogic continues to advance the capabilities of proteomics in both research and clinical settings.

Bridge to Life is a U.S.-based company headquartered in Northbrook, Illinois, dedicated to enhancing organ transplantation through innovative preservation solutions and technologies. The company specializes in the development and licensing of preservation products aimed at improving the quality of service provided to organ procurement organizations and transplant centers. By focusing on advancements in organ preservation and machine perfusion, Bridge to Life collaborates with surgeons and transplant professionals to explore new scientific developments in the field. As a leading supplier of preservation solutions worldwide, the company is committed to facilitating better storage and transportation of intra-abdominal organs to optimize transplant outcomes.

Zentera Therapeutics is a biopharmaceutical company.

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.

Cue helps users track their health at the molecular level, revealing the interplay of activity, food, and sleep. Consumers can track five key health and lifestyle indicators: inflammation, vitamin D, fertility, influenza, and testosterone in just minutes, at home. The Cue sends the data via Bluetooth 4.0 to the user's smartphone, where the free Cue app reveals the interplay of activity, food, and sleep shaping the user at a molecular level.

Forge Biologics is a gene therapy development engine, focused on enabling access to life-changing gene therapies and helping bring them from an idea into reality. The company partners with innovators in the gene therapy community: scientists, physicians, biotech/pharma companies, and patient groups. It was founded in 2020 and headquartered in Grove City, Ohio.

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.

Absci is a drug and target discovery company that utilizes deep learning AI and synthetic biology to enhance the therapeutic potential of proteins. By employing its Integrated Drug Creation Platform, Absci identifies novel drug targets and discovers optimal biotherapeutic candidates while simultaneously generating the necessary cell lines for manufacturing. This innovative approach allows biotech and pharmaceutical partners to develop next-generation protein-based drugs, including Bionic Proteins that incorporate nonstandard amino acids and other unique drug designs that may be unattainable through traditional methods. Absci's mission focuses on advancing the development of improved medicines by effectively translating innovative ideas into viable drugs.

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.

Pipeline Therapeutics is a developer of regenerative therapies designed to promote functional recovery of neurological diseases. The company's therapies leverage the natural repair process in several different nervous system cell types to target diseases such as multiple sclerosis, providing patients with a treatment to repair damage to the nervous system.

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.

Earli is a company focused on developing innovative cancer treatment devices that utilize synthetic biomarkers for early cancer detection. The company's approach involves a novel gene therapy technique known as Synthetic Biopsy, which prompts cancer cells to produce synthetic, non-human biomarkers. These biomarkers can be detected through various means, including blood, urine, or breath, allowing for timely tracking and treatment of cancer. By facilitating the early identification of cancer when it is still homogeneous, Earli aims to significantly enhance survival rates. Their technology not only aids in detection but is also applicable for therapeutic interventions, thereby personalizing treatment and improving patient outcomes.

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

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

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.

Achilles Therapeutics is a developer of immunotherapies intended to offer next-generation, patient-specific therapies to treat cancer. The company's immunotherapies harness the immune system to destroy cancer cells that target truncal tumor neo-antigens and flags to the immune system present on the surface of every cancer cell, enabling scientists to target and destroy tumors without harming healthy tissues.

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.

LianBio’s mission is to catalyze the development and accelerate availability of paradigm-shifting medicines to patients in China and major Asian markets through partnerships that provide access to the best science-driven therapeutic discoveries. LianBio collaborates with world-class partners across a diverse array of therapeutic and geographic areas to build out a pipeline based on disease relevance and the ability to impact patients with transformative mechanisms and precision-based therapeutics.

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.

Freenome, Inc. is a biotechnology company based in South San Francisco, California, founded in 2014. It specializes in developing artificial intelligence-driven genomic solutions for the early detection of cancer. The company focuses on creating simple, non-invasive blood tests that can identify early-stage cancer and assist in treatment selection, thus enabling proactive disease management. Freenome’s multi-omics platform employs proprietary algorithms to enhance disease screening processes, aiming to facilitate timely interventions and improve treatment effectiveness. In addition to its diagnostic products, Freenome also offers clinical research services to support its mission of transforming cancer management through systematic early detection.

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.