RTW Investments

Tenax Therapeutics, Inc. is a specialty pharmaceutical company dedicated to the identification, development, and commercialization of products aimed at addressing cardiovascular and pulmonary diseases with significant unmet medical needs. Headquartered in Morrisville, North Carolina, Tenax focuses on levosimendan, a pharmaceutical product designed to reduce morbidity and mortality in cardiac surgery patients at risk for low cardiac output syndrome. The company is also actively conducting a Phase 2 clinical trial to explore the efficacy of levosimendan in treating pulmonary hypertension associated with heart failure with preserved ejection fraction. Additionally, Tenax is involved in developing novel formulations of imatinib mesylate, a kinase inhibitor with the potential to serve as a disease-modifying therapy for pulmonary arterial hypertension. Founded in 1967, Tenax Therapeutics underscores its commitment to advancing treatments within these critical therapeutic areas.

Windward Bio is a clinical-stage biopharmaceutical company dedicated to enhancing outcomes for patients with advanced immunological diseases, focusing on severe respiratory conditions. The company is advancing a fully human monoclonal antibody targeting thymic stromal lymphopoietin (TSLP) into phase 2 clinical trials. This antibody, administered subcutaneously, potently binds to the TSLP ligand, extending its half-life and silencing its effector functions. Windward Bio is also developing innovative bispecific programs for other immunological disorders, led by a team of experienced biopharmaceutical executives aiming to address significant unmet medical needs in this field.

Ikena Oncology, Inc. is a biotechnology company focused on discovering and developing biomarker-driven therapies for cancer treatment. It specializes in precision oncology by targeting pathways essential for cancer growth and therapeutic resistance, particularly within the Hippo and RAS signaling networks. The company's portfolio includes several preclinical and discovery-stage programs, such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-412, a kynurenine-degrading enzyme. Additionally, Ikena is developing IK-930, an oral small-molecule inhibitor of the TEAD transcription factor involved in the Hippo pathway. Ikena Oncology, originally founded as Kyn Therapeutics in 2016, rebranded in December 2019 and is headquartered in Boston, Massachusetts. The company aims to address significant medical needs in oncology through innovative drug development.

RAPT Therapeutics is a clinical-stage biopharmaceutical company specializing in immunology. It is dedicated to discovering, developing, and commercializing oral small-molecule therapies aimed at addressing significant unmet medical needs in oncology and inflammatory diseases. The company employs a proprietary discovery and development engine to create highly selective small molecules that modulate immune responses related to these conditions. RAPT has advanced two notable drug candidates: FLX475, which targets the inhibition of immunosuppressive regulatory T cells in tumors, and RPT193, which selectively inhibits type 2 T helper cells in inflamed tissues. Both candidates have progressed to clinical trials within a short timeframe since the company's inception. Additionally, RAPT is exploring several other targets in the discovery phase, including general control nonderepressible 2 and hematopoietic progenitor kinase 1.

Ottimo Pharma is a biopharmaceutical company dedicated to developing innovative cancer therapies for solid tumors. Its primary focus is Jankistomig, a bi-functional antibody that simultaneously targets two critical pathways in cancer growth: immune checkpoint inhibition and angiogenesis. By addressing these pathways concurrently, Ottimo Pharma aims to improve treatment outcomes and reduce the burden of cancer on patients and the healthcare system.

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

Aktis Oncology is a biotechnology company focused on the discovery and development of a new class of targeted radiopharmaceuticals aimed at treating various solid tumor cancers. The company has developed proprietary platforms to create tumor-targeting agents optimized for alpha radiotherapy. These agents are engineered for high tumor penetration and prolonged residence time, allowing for effective tumor elimination while reducing the side effects typically associated with cancer treatments. Additionally, Aktis Oncology's innovative approach enables clinicians to visualize and confirm target engagement before administering therapeutic radioisotopes, enhancing treatment precision and efficacy.

Jade Biosciences is a biopharmaceutical company dedicated to developing innovative therapies for unmet needs in autoimmune diseases. Its lead asset, JADE-001, targets the APRIL pathway for treating IgA nephropathy, with initial clinical trials planned. Additionally, Jade has two preclinical antibody discovery programs, JADE-002 and JADE-003. The company was founded upon assets licensed from Paragon Therapeutics.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing innovative CAR T-cell therapies to improve outcomes for cancer patients. The company aims to address the challenges faced by existing autologous CAR therapies, which are only curative for less than half of cancer patients and hindered by issues such as manufacturing limitations, supply challenges, and reimbursement barriers. Cargo Therapeutics is advancing next-generation cell therapies designed to enhance effectiveness, safety, and supply reliability. One of its key products, CRG-022, is a novel CAR T-cell candidate targeting CD22, an antigen present in a majority of B-cell malignancies, specifically designed to overcome treatment resistance. By pursuing these novel solutions, Cargo Therapeutics seeks to facilitate broader access to curative therapies and significantly improve the treatment experience for cancer patients.

Lycia Therapeutics operates as a biotechnology company focused on discovering and developing first-in-class therapeutics using its proprietary Lysosomal Targeting Chimeras (LYTACs) platform. This platform enables the degradation of extracellular and membrane-bound proteins associated with challenging-to-treat diseases like cancer and autoimmune disorders, aiming to provide effective treatments for patients.

Cullinan Therapeutics is a developer of an externally sourced cancer therapeutics intended to end a drug program quickly if the early research suggests it won't work. The company's therapeutics are developed via assets sourced internally through dry lab or externally through business, academic and pharma collaborations, enabling researchers to get a highly diversified portfolio of oncology therapeutics with a unique, cost-efficient business model.

Obsidian Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing next-generation cell and gene therapies aimed at enhancing adoptive immunotherapy for cancer patients. Its innovative approach utilizes pharmacologic operating systems that enable precise control over protein activity within cells. This technology allows for the creation of adoptive cell therapies with novel functionalities, which can be regulated by treating physicians using simple and safe orally active medications. Through these advancements, Obsidian Therapeutics seeks to improve upon existing cell therapies and expand treatment options for a broader patient demographic.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies aimed at transforming the treatment landscape for inflammatory bowel disease (IBD). By leveraging advanced antibody engineering, rational therapeutic combinations, and precision medicine strategies for patient selection, Spyre Therapeutics seeks to create next-generation products that address the chronic inflammation associated with IBD, which includes ulcerative colitis and Crohn's disease. The company aims to enhance existing treatment options, which currently include anti-inflammatory drugs, immunosuppressants, and biologics, thereby improving outcomes for patients suffering from these debilitating gastrointestinal disorders.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to treat aging and age-related diseases. The company utilizes a proprietary systems biology and artificial intelligence platform to identify significant drug targets that influence aging, leveraging extensive datasets to uncover the molecular drivers of age-related pathologies. Among its key offerings is BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor that activates HIF-1 target genes involved in various biological processes, including tissue regeneration and vascular remodeling. Another notable product is BGE-175, an orally administered inhibitor targeting the prostaglandin D2 DP1 signaling pathway, which is linked to an increased risk of mortality and infections, including those associated with immune aging. Founded in 2015 and headquartered in Richmond, California, BioAge Labs is dedicated to harnessing the biology of human aging to create innovative therapies for metabolic and age-related diseases.

Basking Biosciences is a startup focused on developing innovative therapies for ischemic stroke. The company is creating a reversible thrombolytic therapy aimed at restoring blood flow to the brain during such events, which can help prevent hemorrhage and minimize long-term damage. Their platform features a paired therapy that combines an active agent targeting a critical component of clot stability with a designed reversal agent. This reversal agent can be administered if bleeding occurs, neutralizing the active compound and restoring normal clotting ability. This dual approach enables healthcare professionals to provide effective treatment for acute ischemic stroke patients.

Dianthus Therapeutics is a clinical-stage biotechnology company focused on developing innovative treatments for severe autoimmune diseases through targeted therapies. The company's lead candidate, DNTH103, is a monoclonal antibody designed to selectively inhibit a specific component of the immune system, offering potential advantages over existing complement therapies. This treatment aims to improve safety and efficacy for patients suffering from serious autoimmune and inflammatory conditions. Dianthus is committed to advancing its pipeline of next-generation complement therapeutics, supported by a team of experienced professionals in the biotech and pharmaceutical sectors. The company envisions that DNTH103 could be administered as a self-injection for certain patients, enhancing convenience and accessibility in treatment.

Jixing Pharmaceuticals is a global biotechnology company focused on developing and commercializing innovative therapeutics for cardiovascular and ophthalmic diseases. The company’s asset pipeline includes treatments for conditions such as hypertrophic cardiomyopathy, heart failure, presbyopia, hypertension, and dry eye disease. Jixing Pharmaceuticals has in-licensed a small molecule cardiac myosin inhibitor, designed to address the specific needs of patients suffering from hypertrophic cardiomyopathy. By providing advanced treatment options, the company aims to meet the significant unmet medical needs of patients in China and beyond, particularly those facing serious, life-threatening health challenges in the fields of cardiology and ophthalmology.

Immunic Therapeutics is a clinical-stage biopharmaceutical company focused on developing selective oral immunology therapies to treat chronic inflammatory and autoimmune diseases. The company's pipeline includes three small molecule products. The lead program, IMU-838, is a selective immune modulator that blocks the enzyme DHODH, inhibiting the metabolism of activated immune cells and exhibiting antiviral effects. It is being developed for multiple sclerosis, ulcerative colitis, Crohn's disease, and primary sclerosing cholangitis. IMU-935, a selective inverse agonist of the transcription factor RORγt, targets psoriasis, castration-resistant prostate cancer, and Guillain-Barré syndrome. Additionally, IMU-856 aims to restore intestinal barrier function for diseases involving bowel barrier dysfunction.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies aimed at transforming the treatment landscape for inflammatory bowel disease (IBD). By leveraging advanced antibody engineering, rational therapeutic combinations, and precision medicine strategies for patient selection, Spyre Therapeutics seeks to create next-generation products that address the chronic inflammation associated with IBD, which includes ulcerative colitis and Crohn's disease. The company aims to enhance existing treatment options, which currently include anti-inflammatory drugs, immunosuppressants, and biologics, thereby improving outcomes for patients suffering from these debilitating gastrointestinal disorders.

Arvinas, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, that specializes in the discovery, development, and commercialization of innovative therapies aimed at degrading disease-causing proteins. The company's lead product candidates include ARV-110, a proteolysis targeting chimera (PROTAC) currently in phase I clinical trials for metastatic castration-resistant prostate cancer, and ARV-471, which targets the estrogen receptor for patients with metastatic ER-positive/HER2-negative breast cancer. In addition to these, Arvinas is developing various other PROTACs aimed at degrading clinically relevant androgen receptor mutations and treatments for neurodegenerative diseases, including tauopathies. The company has established collaborations with prominent pharmaceutical firms such as Pfizer Inc., Genentech, and Bayer AG, enhancing its research capabilities and reach within the biopharmaceutical landscape. Founded in 2015, Arvinas aims to improve patient outcomes by eliminating harmful proteins from the body through its proprietary technology.

SAB Biotherapeutics is a clinical-stage biopharmaceutical company established in 2014, headquartered in Sioux Falls, South Dakota with additional offices in Cambridge, Massachusetts. The company specializes in developing immunotherapies using its proprietary DiversitAb platform, which employs transchromosomic cattle to produce large quantities of targeted human polyclonal antibodies without relying on human plasma or serum. SAB's pipeline includes therapies for conditions such as Type 1 diabetes (SAB-142), seasonal influenza (SAB-176), and Middle East Respiratory Syndrome Coronavirus (MERS-CoV, SAB-301).

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, focused on developing adeno-associated virus (AAV)-based therapies for monogenic diseases of the central nervous system (CNS). Founded in 2019, the company aims to create curative treatments for severe and life-threatening conditions. Its pipeline includes several product candidates, 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 collaborates with The University of Texas Southwestern Medical Center to enhance its development and commercialization efforts, leveraging expertise in gene therapy to advance its mission of eradicating monogenic CNS diseases.

Abdera Therapeutics is an oncology company focused on developing targeted alpha therapies (TATs) for patients suffering from relapsed, refractory, and metastatic cancers. The company employs a precision oncology platform that utilizes purpose-built vectors to deliver high-energy radio-isotopes directly to tumors and metastatic lesions. This innovative approach to targeted radiotherapy aims to improve treatment efficacy for patients with challenging cancer diagnoses. Abdera's work in this emerging field aligns with a significant market potential, as targeted alpha therapies are expected to play a major role in the future of nuclear medicine.

Milestone Pharmaceuticals Inc. is a biopharmaceutical company focused on developing and commercializing etripamil, a novel calcium channel blocker designed for the treatment of various cardiovascular conditions. Etripamil is a rapid-onset nasal spray intended for self-administration to terminate episodes of paroxysmal supraventricular tachycardia (PSVT). The company is currently conducting Phase III clinical trials for etripamil in the United States and Canada, specifically targeting PSVT, as well as ongoing Phase II trials for patients with atrial fibrillation experiencing a rapid ventricular rate. Founded in 2003 and headquartered in Montréal, Canada, Milestone Pharmaceuticals aims to address unmet needs in the treatment of transient cardiovascular conditions such as atrial arrhythmias and angina.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing innovative CAR T-cell therapies to improve outcomes for cancer patients. The company aims to address the challenges faced by existing autologous CAR therapies, which are only curative for less than half of cancer patients and hindered by issues such as manufacturing limitations, supply challenges, and reimbursement barriers. Cargo Therapeutics is advancing next-generation cell therapies designed to enhance effectiveness, safety, and supply reliability. One of its key products, CRG-022, is a novel CAR T-cell candidate targeting CD22, an antigen present in a majority of B-cell malignancies, specifically designed to overcome treatment resistance. By pursuing these novel solutions, Cargo Therapeutics seeks to facilitate broader access to curative therapies and significantly improve the treatment experience for cancer patients.

OriCell Therapeutics is a biopharmaceutical company that specializes in the development of innovative cellular drugs aimed at addressing unmet clinical needs in tumor immunotherapy. The company focuses on creating therapeutics for a range of cancers, including liver cancer, myeloma, ovarian cancer, gastric cancer, cervical cancer, and non-small cell lung cancer. With a commitment to effective and affordable treatment options, OriCell Therapeutics aims to enhance patient care through the development of clinically valuable therapies, seeking to establish itself as a leader in the field of novel oncological treatments.

Immunic Therapeutics is a clinical-stage biopharmaceutical company focused on developing selective oral immunology therapies to treat chronic inflammatory and autoimmune diseases. The company's pipeline includes three small molecule products. The lead program, IMU-838, is a selective immune modulator that blocks the enzyme DHODH, inhibiting the metabolism of activated immune cells and exhibiting antiviral effects. It is being developed for multiple sclerosis, ulcerative colitis, Crohn's disease, and primary sclerosing cholangitis. IMU-935, a selective inverse agonist of the transcription factor RORγt, targets psoriasis, castration-resistant prostate cancer, and Guillain-Barré syndrome. Additionally, IMU-856 aims to restore intestinal barrier function for diseases involving bowel barrier dysfunction.

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.

Allakos Inc. is a clinical-stage biopharmaceutical company founded in 2012 and headquartered in Redwood City, California. The company focuses on developing antibody-based therapeutics aimed at treating diseases associated with dysregulation of the T-helper type 2 immune response, which plays a crucial role in various allergic and inflammatory conditions. Its lead product, antolimab (AK002), is being investigated for the treatment of eosinophilic gastritis, eosinophilic gastroenteritis, chronic urticaria, indolent systemic mastocytosis, and severe allergic conjunctivitis. Additionally, Allakos is developing AK006, an antibody that targets the Siglec-6 receptor on mast cells, designed to activate the receptor's inhibitory function and reduce mast cell activation. Through these innovative therapies, Allakos aims to address the underlying mechanisms of diseases characterized by excessive inflammatory responses.

Immunocore Limited is a biotechnology company based in Abingdon, United Kingdom, with an additional office in Conshohocken. Founded in 1999, the company focuses on the discovery and development of innovative T cell receptor-based therapeutics aimed at addressing significant unmet medical needs in cancer, viral diseases, and autoimmune diseases. Immunocore specializes in engineering T cell receptors (TCRs) and linking them to antibody fragments, which has led to the development of its lead product candidate, IMCgp100, currently undergoing clinical trials for metastatic melanoma. The company's proprietary TCR technology platform is validated and designed for efficient manufacturing, supporting both clinical and commercial supply. Immunocore was previously known as Avidex Limited before adopting its current name in 2008.

Mineralys Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for hypertension. The company is advancing its lead product candidate, MLS-101, a highly selective and potent aldosterone synthase inhibitor licensed from Mitsubishi Tanabe Pharma Corporation. Mineralys aims to provide a targeted treatment for patients with uncontrolled hypertension, particularly those who do not achieve adequate blood pressure control despite using multiple antihypertensive medications. By addressing the underlying factors associated with elevated aldosterone levels, Mineralys seeks to improve treatment outcomes for individuals suffering from this condition.

MoonLake Immunotherapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies utilizing Nanobody technology. The company is primarily dedicated to advancing Sonelokimab, an investigational Nanobody, aimed at treating various inflammatory conditions, particularly those affecting the skin and joints. Through its research and development efforts, MoonLake seeks to transform treatment outcomes for patients suffering from immunologic diseases.

Third Harmonic Bio is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for severe allergies and inflammation. The company is focused on advancing its lead product candidate, THB001, which is a first-in-class, highly selective oral inhibitor of the KIT receptor, a key regulator of mast cell function and survival. Mast cells play a crucial role in mediating inflammation in allergic diseases, making this targeted approach significant for treatment. Additionally, Third Harmonic Bio is developing THB335, another potent and selective oral small-molecule KIT inhibitor aimed at addressing various mast cell-driven inflammatory conditions affecting the skin, airway, and gastrointestinal tract. Through its research and development efforts, Third Harmonic Bio seeks to provide new options for patients suffering from inflammatory diseases.

Acelyrin is a late-stage clinical biopharmaceutical company dedicated to advancing new treatment options for patients. The company specializes in identifying, acquiring, and accelerating the development and commercialization of promising drug candidates. By leveraging its expertise, Acelyrin aims to rapidly bring transformative medicines to market, ultimately improving patient outcomes and quality of life.

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

Science 37, Inc. is a technology-enabled clinical trial company that specializes in developing patient-centric models for clinical research, aimed at accelerating biomedical discovery. The company utilizes its innovative platform, NORA (Network Oriented Research Assistant), a cloud-based mobile research tool that facilitates communication between researchers, patients, and mobile nurses through videos, surveys, and photographs. By providing decentralized clinical trial services, Science 37 enables access to diverse patient populations that traditional site-based research may overlook. The company has established itself as a leader in conducting virtual trials, leveraging an extensive network of telemedicine investigators and home-health nurses to enhance patient enrollment and retention. Serving a wide range of clients, including pharmaceutical sponsors, biotech firms, and academic institutions, Science 37 aims to transform clinical research through its comprehensive, fully integrated trial platform. Founded in 2014 and headquartered in Los Angeles, California, Science 37 continues to innovate in the field of clinical research.

Roivant Sciences is a biopharmaceutical company dedicated to advancing the development of innovative late-stage drug candidates and enhancing healthcare delivery. The company aims to expedite the process of bringing new medicines to patients while reducing associated costs, thereby benefiting the healthcare system. Roivant achieves this by creating specialized entities known as "Vants," which are agile biotech and healthcare technology companies that leverage unique talent sourcing and incentive alignment strategies. In addition to its focus on biopharmaceuticals, Roivant incubates discovery-stage companies and health technology startups that complement its core business. Among its commercial-stage drug candidates is VTAMA, a treatment for plaque psoriasis in adults, along with several other candidates, including Batoclimab, IMVT-1402, Brepocitinib, and Namilumab, which are at various stages of development.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing innovative therapies for autoimmune diseases and inflammatory disorders. Incorporated in 2018, Ventyx focuses on creating selective inhibitors targeting TYK2, a key mediator in various inflammatory conditions. The company's clinical pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, which aims to provide a safer alternative by avoiding the toxicities linked to broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator intended for ulcerative colitis treatment, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which plays a significant role in multiple inflammatory diseases.

Aadi Bioscience, Inc. is a clinical-stage biopharmaceutical company based in Pacific Palisades, California, focused on developing precision therapies for genetically-defined cancers. The company specializes in mTOR inhibitors, particularly for patients with mTOR pathway driver alterations, such as those involving the TSC1 or TSC2 genes. Aadi's primary aim is to create transformational therapies for patients with specific cancer types, including the ultra-rare perivascular epithelioid cell tumor (PEComa). The company addresses challenges faced by existing mTOR inhibitors, including issues related to pharmacology, drug delivery, safety, and targeting. Founded in 2011, Aadi Bioscience has positioned itself to innovate in the treatment of oncology and cardiovascular diseases.

Lycia Therapeutics operates as a biotechnology company focused on discovering and developing first-in-class therapeutics using its proprietary Lysosomal Targeting Chimeras (LYTACs) platform. This platform enables the degradation of extracellular and membrane-bound proteins associated with challenging-to-treat diseases like cancer and autoimmune disorders, aiming to provide effective treatments for patients.

Artios Pharma Limited is a biotechnology company based in Cambridge, United Kingdom, that specializes in developing innovative cancer treatments targeting DNA damage response (DDR) pathways. Founded in 2016, the company is dedicated to creating first-in-class therapies that selectively kill cancer cells by exploiting their reliance on DDR mechanisms. Artios’ product pipeline includes a range of promising candidates, such as a therapy that targets DNA polymerase theta, a protein involved in DNA repair processes, and another program focusing on a novel DDR target. The company collaborates with prominent research organizations, including Cancer Research Technology, to enhance its development efforts and leverage expertise in DNA repair. Artios is led by a skilled team with extensive experience in drug discovery, positioning it to advance its next-generation DDR programs and contribute significantly to the field of cancer treatment.

Jixing Pharmaceuticals is a global biotechnology company focused on developing and commercializing innovative therapeutics for cardiovascular and ophthalmic diseases. The company’s asset pipeline includes treatments for conditions such as hypertrophic cardiomyopathy, heart failure, presbyopia, hypertension, and dry eye disease. Jixing Pharmaceuticals has in-licensed a small molecule cardiac myosin inhibitor, designed to address the specific needs of patients suffering from hypertrophic cardiomyopathy. By providing advanced treatment options, the company aims to meet the significant unmet medical needs of patients in China and beyond, particularly those facing serious, life-threatening health challenges in the fields of cardiology and ophthalmology.

Alcyone Therapeutics is focused on advancing precision genetic therapies for central nervous system (CNS) disorders. The company develops innovative neural intervention technology aimed at addressing neurological conditions through targeted drug therapies. By integrating micro-fabrication technologies with advanced biomedical engineering, Alcyone Therapeutics seeks to enhance treatment options for patients suffering from chronic neuropathological conditions, including hydrocephalus. Their work aims to unlock new possibilities in CNS therapy development, addressing the unique challenges associated with treating these complex disorders.

GH Research PLC is a clinical-stage biopharmaceutical company committed to advancing treatments for psychiatric and neurological disorders. The company's primary focus is on developing novel and proprietary therapies using Mebufotenin 5-Methoxy-N, N-Dimethyltryptamine, commonly known as 5-MeO-DMT, specifically targeting patients with Treatment-Resistant Depression.

UroGen Pharma Ltd. is a clinical-stage biopharmaceutical company based in Princeton, New Jersey, focused on developing innovative therapies for specialty cancers and urologic diseases. The company is dedicated to transforming the standard of care in uro-oncology through its diverse pipeline of product candidates. Its lead products, including UGN-101 and UGN-102, aim to ablate tumors non-surgically and address various forms of non-muscle invasive urothelial cancer, such as low-grade upper tract urothelial carcinoma and low-grade non-muscle invasive bladder cancer. UroGen is also advancing UGN-201, an immunotherapy candidate targeting high-grade non-muscle invasive bladder cancer. Furthermore, the company's proprietary RTGel technology enhances drug delivery by enabling sustained release, improving therapeutic efficacy. UroGen has established licensing agreements with Allergan Pharmaceuticals for the development of RTGel-based products and with Agenus Inc. to commercialize treatments for urinary tract cancers. Founded in 2004, UroGen Pharma continues to innovate in the field of urology and oncology.

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing innovative therapies for autoimmune diseases and inflammatory disorders. Incorporated in 2018, Ventyx focuses on creating selective inhibitors targeting TYK2, a key mediator in various inflammatory conditions. The company's clinical pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, which aims to provide a safer alternative by avoiding the toxicities linked to broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator intended for ulcerative colitis treatment, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which plays a significant role in multiple inflammatory diseases.

Visus Therapeutics is a clinical-stage company focused on developing innovative ophthalmic therapies aimed at enhancing vision for individuals worldwide. The company is known for its presbyopia-correcting eye drops, which are designed to provide a prolonged solution to the loss of near vision associated with presbyopia. In addition to these eye drops, Visus Therapeutics is exploring various ophthalmic product candidates that address conditions such as ocular surface disease, glaucoma, and age-related macular degeneration, thereby broadening its impact on eye health and vision improvement.

Encoded Therapeutics, Inc. is a biotechnology company focused on developing precision gene therapies for severe genetic disorders. Utilizing its proprietary platform, the company identifies genomic sequences that regulate gene expression, facilitating advancements in viral gene therapy. Encoded Therapeutics has a diverse therapy pipeline targeting various genetic and acquired disorders across multiple disease pathways, including neurocircuitry, metabolic disorders, neurodegeneration, and cardiovascular diseases. Founded in 2014 and headquartered in South San Francisco, California, the company is dedicated to improving patient outcomes through innovative gene therapy solutions.

Frequency Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

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

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates aimed at targeting and eliminating disease-causing proteins for the treatment of cancer, neurodegenerative diseases, and other conditions. The company's lead product candidate, CFT7455, is an orally bioavailable degrader targeting IKZF1/3, designed to treat multiple myeloma and various lymphomas. Additionally, C4 Therapeutics is advancing CFT8634, which targets BRD9 for synovial sarcoma and SMARCB1-deleted solid tumors, as well as programs targeting BRAF V600E and RET for genetically defined resistant solid tumors. The company employs its proprietary Degronimid platform, which utilizes small molecule binders to flag harmful proteins for degradation by the cellular proteasome system, allowing for the potential treatment of previously undruggable targets. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics engages in strategic collaborations to enhance its research and development efforts.

Athira Pharma, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, focused on developing small molecules aimed at restoring neuronal health and halting neurodegeneration. The company's lead product candidate, ATH-1017, is a small molecule designed to penetrate the blood-brain barrier and acts as a hepatocyte growth factor/MET activator, currently undergoing various clinical trials for treating Alzheimer’s and Parkinson’s diseases. In addition to ATH-1017, Athira is developing several other candidates, including ATH-1019 for depression and ATH-1018 for peripheral neuropathy, which are in the preclinical stage. Founded in 2011 and initially known as M3 Biotechnology, Athira Pharma changed its name in April 2019 to reflect its mission of advancing innovative therapies to improve the lives of individuals affected by brain disorders.

iTeos Therapeutics is a clinical-stage biopharmaceutical company focused on developing immuno-oncology therapeutics aimed at improving outcomes for cancer patients. Founded in 2011 and headquartered in Gosselies, Belgium, with additional operations in Cambridge, Massachusetts, the company is advancing a pipeline of innovative drug candidates. Notable among these is EOS-850, a small molecule antagonist targeting the adenosine A2a receptor, currently undergoing an open-label Phase 1/2a clinical trial in adult patients. Another key candidate is EOS-448, an antagonist of TIGIT, which is also in Phase 1/2a trials. iTeos leverages expertise in tumor immunology to create small molecule immunomodulators designed to counteract cancer-induced immune suppression, thereby enhancing the effectiveness of existing treatments and emerging immunotherapies.

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.

Immunocore Limited is a biotechnology company based in Abingdon, United Kingdom, with an additional office in Conshohocken. Founded in 1999, the company focuses on the discovery and development of innovative T cell receptor-based therapeutics aimed at addressing significant unmet medical needs in cancer, viral diseases, and autoimmune diseases. Immunocore specializes in engineering T cell receptors (TCRs) and linking them to antibody fragments, which has led to the development of its lead product candidate, IMCgp100, currently undergoing clinical trials for metastatic melanoma. The company's proprietary TCR technology platform is validated and designed for efficient manufacturing, supporting both clinical and commercial supply. Immunocore was previously known as Avidex Limited before adopting its current name in 2008.

Avadel Pharmaceuticals plc is a specialty pharmaceutical company based in Dublin, Ireland, with operations in the United States and France. The company focuses on developing and commercializing innovative pharmaceutical products, particularly in the fields of urology, central nervous system disorders, and hospital settings. Avadel's key product, FT218, is in Phase 3 clinical trials for treating narcolepsy-related excessive daytime sleepiness and cataplexy. Additionally, Avadel markets several sterile injectable drugs for hospital use, including Akovaz for hypotension during anesthesia, Bloxiverz for reversing neuromuscular blockade effects, and Vazculep for treating hypotension. The company also offers LUMRYZ, an extended-release formulation of sodium oxybate for narcolepsy management. Avadel Pharmaceuticals was incorporated in 2015 and was previously known as Flamel Technologies SA before rebranding in January 2017.

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

Frequency Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

Encoded Therapeutics, Inc. is a biotechnology company focused on developing precision gene therapies for severe genetic disorders. Utilizing its proprietary platform, the company identifies genomic sequences that regulate gene expression, facilitating advancements in viral gene therapy. Encoded Therapeutics has a diverse therapy pipeline targeting various genetic and acquired disorders across multiple disease pathways, including neurocircuitry, metabolic disorders, neurodegeneration, and cardiovascular diseases. Founded in 2014 and headquartered in South San Francisco, California, the company is dedicated to improving patient outcomes through innovative gene therapy solutions.

Roivant Sciences is a biopharmaceutical company dedicated to advancing the development of innovative late-stage drug candidates and enhancing healthcare delivery. The company aims to expedite the process of bringing new medicines to patients while reducing associated costs, thereby benefiting the healthcare system. Roivant achieves this by creating specialized entities known as "Vants," which are agile biotech and healthcare technology companies that leverage unique talent sourcing and incentive alignment strategies. In addition to its focus on biopharmaceuticals, Roivant incubates discovery-stage companies and health technology startups that complement its core business. Among its commercial-stage drug candidates is VTAMA, a treatment for plaque psoriasis in adults, along with several other candidates, including Batoclimab, IMVT-1402, Brepocitinib, and Namilumab, which are at various stages of development.

Milestone Pharmaceuticals Inc. is a biopharmaceutical company focused on developing and commercializing etripamil, a novel calcium channel blocker designed for the treatment of various cardiovascular conditions. Etripamil is a rapid-onset nasal spray intended for self-administration to terminate episodes of paroxysmal supraventricular tachycardia (PSVT). The company is currently conducting Phase III clinical trials for etripamil in the United States and Canada, specifically targeting PSVT, as well as ongoing Phase II trials for patients with atrial fibrillation experiencing a rapid ventricular rate. Founded in 2003 and headquartered in Montréal, Canada, Milestone Pharmaceuticals aims to address unmet needs in the treatment of transient cardiovascular conditions such as atrial arrhythmias and angina.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company focused on developing novel antisense oligonucleotide medicines aimed at addressing the underlying causes of severe genetic diseases. Founded in 2014 and headquartered in Bedford, Massachusetts, the company utilizes its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to enhance gene expression through RNA splicing. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke Therapeutics has also partnered with Invitae Corporation to offer complementary genetic testing services.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

ARMO BioSciences is a biotechnology company based in Redwood City, California, focused on developing immune modulatory biologic therapeutics. The company specializes in immuno-oncology, aiming to activate the immune system of cancer patients to recognize and eradicate tumors. Its pipeline includes several product candidates: AM0010, a long-acting form of interleukin 10 (IL-10) that stimulates the immune system; AM0001, an anti-PD-1 monoclonal antibody; AM0003, an anti-LAG-3 checkpoint inhibitor; AM0015, a form of recombinant human interleukin-15 (IL-15); and AM0012, a form of recombinant human interleukin-12 (IL-12). The company was incorporated in 2010 and formerly known as Targenics.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a 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. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

AveXis, operating as Novartis Gene Therapies, Inc., is a biotechnology company focused on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. Acquired by Novartis in 2018, the company specializes in innovative treatments such as motor neuron cell-targeted gene replacement therapy aimed at conditions like spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. These therapies are designed to assist healthcare institutions in addressing diseases that cause progressive muscle weakness, paralysis, and ultimately, death. Through its advancements in gene therapy, AveXis is dedicated to improving the lives of patients and families impacted by these severe genetic disorders.

Immunocore Limited is a biotechnology company based in Abingdon, United Kingdom, with an additional office in Conshohocken. Founded in 1999, the company focuses on the discovery and development of innovative T cell receptor-based therapeutics aimed at addressing significant unmet medical needs in cancer, viral diseases, and autoimmune diseases. Immunocore specializes in engineering T cell receptors (TCRs) and linking them to antibody fragments, which has led to the development of its lead product candidate, IMCgp100, currently undergoing clinical trials for metastatic melanoma. The company's proprietary TCR technology platform is validated and designed for efficient manufacturing, supporting both clinical and commercial supply. Immunocore was previously known as Avidex Limited before adopting its current name in 2008.

Regenxbio Inc. is a clinical-stage biotechnology company focused on developing gene therapy products to address genetic disorders and enable cells to produce therapeutic proteins and antibodies. The company leverages its proprietary NAV Technology Platform, which utilizes adeno-associated virus vectors for gene delivery. Regenxbio's lead product candidate, RGX-314, is under investigation in a Phase I/IIa clinical trial for wet age-related macular degeneration. Other notable product candidates include RGX-121 and RGX-111, which are in clinical trials for mucopolysaccharidosis type II and I, respectively, as well as RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia. Additionally, Regenxbio licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and collaborates with Neurimmune AG to develop vectorized antibodies for neurodegenerative diseases. Founded in 2008 and headquartered in Rockville, Maryland, the company was previously known as ReGenX Biosciences, LLC before rebranding in 2014.

Verastem, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative treatments aimed at improving the survival and quality of life for cancer patients. Based in Needham, Massachusetts, Verastem markets COPIKTRA (duvelisib), an oral medication indicated for adult patients with relapsed or refractory chronic lymphocytic leukemia and follicular lymphoma after multiple prior therapies. In addition to this approved therapy, the company is advancing its pipeline with the focal adhesion kinase inhibitor defactinib, which is being studied in combination with immunotherapies for various cancers, including pancreatic and ovarian cancers, as well as non-small cell lung cancer. Verastem's research emphasizes inhibiting critical signaling pathways that promote cancer cell survival and tumor growth, particularly those associated with the RAS/MAPK pathway. The company has formed collaborative agreements with Yakult Honsha Co., Ltd. and CSPC Pharmaceutical Group Limited to enhance its development efforts. Founded in 2010, Verastem continues to focus on late-stage development of its anticancer agents.

CytomX Therapeutics, Inc. is an oncology-focused biopharmaceutical company based in South San Francisco, California. It specializes in developing a novel class of antibody therapeutics utilizing its Probody technology platform, which aims to enhance the targeting of tumors while minimizing drug activity in healthy tissues. The company's clinical-stage product candidates include CX-072, a therapeutic targeting programmed cell death ligand 1; CX-2009, a drug conjugate targeting CD166; BMS-986249 and BMS-986288, both targeting CTLA-4 for metastatic melanoma and solid tumors; and CX-2029, a drug conjugate for CD71 in solid tumors. CytomX collaborates strategically with several industry leaders, including AbbVie, Amgen, and Bristol-Myers Squibb, to advance its Probody therapeutics. Founded in 2008, CytomX Therapeutics is committed to providing innovative and less toxic treatment options for patients with severe illnesses such as cancer.