RTW Investments

Umoja Biopharma specializes in innovative immunotherapy aimed at transforming cancer treatment and enhancing patient quality of life. The company focuses on reprogramming T cells within the patient's body to effectively target cancer, including solid tumors and hematological cancers that often have poor responses to conventional therapies. Its proprietary CAR T-cell gene therapy platform is designed to deliver scalable therapeutic regimens tailored to various stages of cancer, enabling medical practitioners to directly and safely attack cancer cells. By harnessing the body's immune response, Umoja Biopharma seeks to improve overall patient outcomes and provide more effective treatment options for those affected by cancer.

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, aiming to address significant unmet medical needs in these areas. Led by an experienced team of biopharmaceutical executives, the company is committed to accelerating drug development to improve the lives of patients.

Numab is a biotechnology company focused on developing antibody-based therapies for severe diseases like chronic inflammation and cancer. Its proprietary platform streamlines the discovery process by reducing randomness, predictably generating ready-to-develop multispecific biotherapeutics tailored to engage multiple targets simultaneously. This enables healthcare industries to access Numab's platform for innovative treatments in these areas.

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.

Ottimo Pharma specializes in developing innovative cancer therapies for solid tumors. Its primary focus is Jankistomig, a bi-functional antibody that targets both immune checkpoint inhibition and angiogenesis, two key pathways involved in cancer growth. By simultaneously addressing these pathways, Ottimo Pharma's approach offers healthcare professionals a dual-pronged strategy to potentially enhance 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.

Evommune, Inc. is a biotechnology company based in Los Altos, California, that focuses on developing and manufacturing tissue-based medicines aimed at treating inflammatory diseases. Founded in 2020, the company employs a proprietary tissue-based platform to accelerate the discovery of novel therapies, with a particular emphasis on immunology and dermatology. Evommune's approach seeks not only to alleviate the symptoms of inflammatory disorders but also to halt disease progression, thereby improving the quality of life for patients. Its pipeline includes initiatives targeting prevalent inflammatory conditions, with a commitment to advancing patient care by creating best-in-class treatment options. Through innovative strategies, Evommune aims to drive meaningful advancements in the field of inflammatory disease management.

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.

Santa Ana Bio is a biotechnology company focused on developing precision therapies for autoimmune and inflammatory diseases. By utilizing advanced technologies such as deep cell profiling, single-cell genomics, and multi-omics platforms, the company designs targeted biologics that aim to provide effective and personalized treatment options. Through its innovative approach, Santa Ana Bio seeks to enhance patient outcomes and improve quality of life for those affected by autoimmune conditions.

LENZ Therapeutics is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative ophthalmic therapies. It focuses on addressing vision-related issues, particularly presbyopia, which affects the near vision of individuals, especially those over the age of 45. The company's primary product is an aceclidine-based eye drop designed to restore near vision loss associated with this condition. Through its advancements in ophthalmic pharmaceuticals, LENZ Therapeutics aims to improve the quality of life for those impacted by vision challenges.

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.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to address aging and age-related diseases. The company utilizes a systems biology and artificial intelligence platform to identify key drug targets that influence aging and analyze extensive datasets to uncover the molecular drivers of age-related conditions. BioAge Labs is advancing several drug candidates, including BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor designed to activate genes involved in critical biological processes such as tissue regeneration and vascular remodeling. Additionally, BGE-175, an orally administered inhibitor of the prostaglandin D2 DP1 signaling pathway, aims to mitigate risks associated with immune aging and respiratory infections. Founded in 2015 and based in Richmond, California, BioAge Labs is committed to discovering and developing innovative treatments that harness the biology of human aging.

Basking Biosciences is a startup focused on developing a reversible thrombolytic therapy aimed at restoring blood flow to the brain during ischemic strokes, thereby preventing hemorrhage and minimizing long-term damage. The company’s innovative approach involves a paired therapy that combines an active agent targeting a crucial component of clot stability with a designed reversal agent. This reversal agent can be administered if bleeding occurs, effectively neutralizing the active compound and restoring the blood's normal clotting ability. By enabling healthcare professionals to treat acute ischemic stroke effectively, Basking Biosciences aims to improve patient outcomes in critical medical situations.

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.

Establishment Labs, founded in 2004, is a medical technology company based in Alajuela, Costa Rica. It specializes in designing, manufacturing, and marketing silicone-filled breast and body shaping implants, primarily under the Motiva Implants brand. The company offers a wide range of breast implants with various surface, shape, size, base, and projection choices. Additionally, Establishment Labs provides Q Inside Safety Technology, a radio frequency identification transponder for implant data access, and Divina 3D imaging technology for surgical planning. Its products are sold through exclusive distributors and direct sales to plastic surgeons, hospitals, and clinics in over 80 countries worldwide. The company's manufacturing facilities adhere to strict FDA and ISO standards.

Jixing Pharmaceuticals is a global biotechnology company focused on developing and commercializing innovative therapeutics for cardiovascular and ophthalmic diseases. The company has a diverse pipeline that includes treatments for conditions such as hypertrophic cardiomyopathy, heart failure, presbyopia, hypertension, and dry eye disease. A key aspect of their development efforts includes a small molecule cardiac myosin inhibitor, in-licensed from Cytokinetics, specifically designed to treat hypertrophic cardiomyopathy. Jixing Pharmaceuticals aims to provide effective treatments for patients in China, addressing significant unmet medical needs in the realms of cardiovascular and ophthalmic health.

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). 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.

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

Kyverna Therapeutics, Inc. is a cell therapy company focused on developing innovative treatments and potential cures for serious autoimmune diseases. Founded in 2018 and based in Berkeley, California, Kyverna employs advanced T cell engineering and synthetic biology technologies to target and eliminate autoreactive immune cells, which are responsible for the inflammation associated with these diseases. By addressing the root causes of autoimmune conditions, Kyverna aims to provide more effective and lasting solutions for patients suffering from these challenging health issues.

Milestone Pharmaceuticals Inc. is a biopharmaceutical company based in Montréal, Canada, focused on developing and commercializing etripamil for various cardiovascular conditions. Etripamil is a novel short-acting calcium channel blocker designed to be administered as a rapid-onset nasal spray, specifically targeting the termination of episodes of paroxysmal supraventricular tachycardia (PSVT). The company is currently conducting Phase III clinical trials for etripamil in the treatment of PSVT and is also exploring its potential applications for atrial fibrillation, angina, and other related cardiovascular disorders. Founded in 2003, Milestone Pharmaceuticals aims to address unmet medical needs in cardiovascular disease management through innovative therapeutic solutions.

OriCell Therapeutics is a biopharmaceutical company dedicated to developing innovative, effective, and affordable tumor immunotherapy treatments. It focuses on unmet clinical needs, with a pipeline targeting various cancers including liver, myeloma, ovarian, gastric, cervical, and non-small cell lung cancers.

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

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.

Immunocore Limited is a biotechnology company based in Abingdon, United Kingdom, with a focus on discovering and developing novel therapeutics for cancer, viral diseases, and autoimmune conditions. The company specializes in T cell receptor (TCR) technology, which allows it to create innovative bispecific immunotherapies. Its lead product candidate, IMCgp100, is currently undergoing clinical trials for the treatment of metastatic melanoma. Immunocore's proprietary platform enables efficient manufacturing of TCR-based drugs, addressing significant unmet medical needs. Founded in 1999 and previously known as Avidex Limited, Immunocore has expanded its operations with an additional office in Conshohocken.

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 for immunologic diseases. The company specializes in leveraging Nanobody technology to create new treatments aimed at addressing inflammatory conditions, particularly in the skin and joints. Its lead investigational product, Sonelokimab, represents a novel approach to managing inflammation, with the potential to significantly improve patient outcomes. Through its research and development efforts, MoonLake aims to advance the understanding and treatment of various inflammatory diseases, contributing to the evolving landscape of biopharmaceuticals.

22seventy bio is an immuno-oncology cell therapy company that discovers and develops cancer therapies. The company combines a deep knowledge of cancer cell biology and genetics with an understanding of the immune response to cancer to advance its pipeline. It was established in Cambridge, Massachusetts in 2021.

Magnolia Medical Technologies, Inc. is a medical device company based in Seattle, Washington, focused on enhancing the accuracy of diagnostic blood culture tests. Established in 2011, the company has developed SteriPath, a vacuum-assisted blood collection system designed to prevent contamination of blood samples, which can lead to misdiagnosis of sepsis. This innovative system operates by actively diverting and sequestering contaminated blood, allowing for the collection of uncontaminated samples through a separate flow path. This closed vein-to-bottle collection method aims to improve diagnostic specimen quality, thereby increasing diagnostic accuracy and improving patient outcomes. Magnolia Medical Technologies holds a robust intellectual property portfolio, including over 70 issued patents and more than 50 pending applications, reflecting its commitment to advancing medical technology and addressing challenges such as antibiotic resistance. The company's products are distributed throughout the United States.

Orchestra BioMed is a biomedical innovation company based in New Hope, Pennsylvania, founded in 2017. The company specializes in developing high-impact therapeutic solutions for major medical conditions, with a primary focus on cardiovascular diseases, which are responsible for approximately 31% of global deaths annually. Orchestra BioMed's business model involves strategic partnerships with medical device companies to accelerate the commercialization of its products. Its key product candidates include BackBeat Cardiac Neuromodulation Therapy for hypertension and Virtue Sirolimus AngioInfusionBalloon for atherosclerotic artery disease.

Kyverna Therapeutics, Inc. is a cell therapy company focused on developing innovative treatments and potential cures for serious autoimmune diseases. Founded in 2018 and based in Berkeley, California, Kyverna employs advanced T cell engineering and synthetic biology technologies to target and eliminate autoreactive immune cells, which are responsible for the inflammation associated with these diseases. By addressing the root causes of autoimmune conditions, Kyverna aims to provide more effective and lasting solutions for patients suffering from these challenging health issues.

Third Harmonic Bio is a clinical-stage biopharmaceutical company dedicated to developing innovative treatments for severe allergies and inflammation. The company is focused on creating first-in-class oral small-molecule inhibitors targeting the KIT receptor, which plays a crucial role in the survival and function of mast cells—key immune cells involved in allergic responses. Its lead product candidate, THB001, is designed to provide selective inhibition of KIT, while another candidate, THB335, aims to address multiple mast cell-driven inflammatory diseases affecting the skin, respiratory system, and gastrointestinal tract. Through its research and development efforts, Third Harmonic Bio seeks to advance the management of inflammatory diseases, enhancing patient outcomes and quality of life.

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, 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.

Renovacor, Inc. is a preclinical-stage biopharmaceutical company based in Philadelphia, Pennsylvania, dedicated to developing gene therapies for cardiovascular diseases. Founded in 2013, the company focuses on innovative treatments for dilated cardiomyopathy (DCM), particularly cases caused by mutations in the BAG3 gene. This condition affects over 3 million individuals in the United States, with around 35,000 patients estimated to have BAG3 mutations, classifying it as an orphan disease. Patients with BAG3-associated DCM, often younger and experiencing faster disease progression, currently have limited treatment options, primarily consisting of standard heart failure care. Renovacor's lead program involves a recombinant adeno-associated virus (AAV)-based gene therapy aimed at replacing the defective BAG3 gene, which holds the potential to prevent disease progression and improve outcomes for this vulnerable patient population.

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 2015, the company is dedicated to creating first-in-class therapies aimed at selectively killing cancer cells. Artios's product pipeline features several promising programs, including a focus on DNA polymerase theta (Polθ), which plays a crucial role in DNA repair processes. Additionally, the company is advancing an in-licensed program that targets a newly identified protein linked to DDR and is developing a novel treatment aimed at DNA nucleases involved in these pathways. Artios collaborates with Cancer Research Technology and prominent DNA repair researchers globally to enhance its research and development efforts.

Jixing Pharmaceuticals is a global biotechnology company focused on developing and commercializing innovative therapeutics for cardiovascular and ophthalmic diseases. The company has a diverse pipeline that includes treatments for conditions such as hypertrophic cardiomyopathy, heart failure, presbyopia, hypertension, and dry eye disease. A key aspect of their development efforts includes a small molecule cardiac myosin inhibitor, in-licensed from Cytokinetics, specifically designed to treat hypertrophic cardiomyopathy. Jixing Pharmaceuticals aims to provide effective treatments for patients in China, addressing significant unmet medical needs in the realms of cardiovascular and ophthalmic health.

Umoja Biopharma specializes in innovative immunotherapy aimed at transforming cancer treatment and enhancing patient quality of life. The company focuses on reprogramming T cells within the patient's body to effectively target cancer, including solid tumors and hematological cancers that often have poor responses to conventional therapies. Its proprietary CAR T-cell gene therapy platform is designed to deliver scalable therapeutic regimens tailored to various stages of cancer, enabling medical practitioners to directly and safely attack cancer cells. By harnessing the body's immune response, Umoja Biopharma seeks to improve overall patient outcomes and provide more effective treatment options for those affected by cancer.

Alcyone Therapeutics is focused on developing advanced neural intervention technologies aimed at treating neurological conditions. The company utilizes an innovative combination of micro-fabrication techniques and biomedical engineering to create precision genetic therapies for central nervous system (CNS) disorders. Its core product offerings target drug therapies and address conditions such as hydrocephalus, ultimately aiming to enhance treatment options for patients suffering from chronic neuropathological issues. By pioneering next-generation therapies, Alcyone Therapeutics seeks to overcome existing challenges in CNS therapy development and unlock new possibilities for effective treatment.

NiKang Therapeutics, Inc. is a biotechnology company established in 2017 and located in Wilmington, Delaware. The company is dedicated to discovering and developing innovative small molecule oncology medicines aimed at addressing unmet medical needs in cancer treatment. By employing a discovery approach that leverages target structure biology and structure-based drug design, NiKang Therapeutics aims to facilitate the rapid and efficient development of proprietary drug candidates with optimal pharmacological properties. The company seeks to enhance the treatment options available to healthcare providers, ultimately improving patient outcomes in the fight against cancer.

Numab is a biotechnology company focused on developing antibody-based therapies for severe diseases like chronic inflammation and cancer. Its proprietary platform streamlines the discovery process by reducing randomness, predictably generating ready-to-develop multispecific biotherapeutics tailored to engage multiple targets simultaneously. This enables healthcare industries to access Numab's platform for innovative treatments in these areas.

NeoGenomics Laboratories operates a network of cancer-focused testing facilities in the United States, Switzerland, and Singapore. The company is divided into two main segments: Clinical Services and Pharma Services. It provides a range of genetic and molecular testing services, including cytogenetics, fluorescence in-situ hybridization, flow cytometry, immunohistochemistry, and molecular testing, aimed at helping clinicians and researchers understand cancer at a genetic level. Additionally, NeoGenomics offers morphologic analysis for diagnosis and supports pharmaceutical companies in their oncology programs through testing services that facilitate drug development and clinical trials. The company's collaboration with Inivata Limited for the commercialization of the InVisionFirst-Lung liquid biopsy test exemplifies its commitment to advancing cancer diagnostics. Founded in 2001 and headquartered in Fort Myers, Florida, NeoGenomics serves hospitals, pathologists, oncologists, academic centers, and other clinical laboratories, enhancing cancer care through innovative testing solutions.

GH Research PLC is a clinical-stage biopharmaceutical company that aims to transform the treatment of psychiatric and neurological disorders. The company is primarily focused on developing novel and proprietary therapies based on 5-MeO-DMT, a compound known as Mebufotenin, to address the needs of patients suffering from Treatment-Resistant Depression (TRD). Through its innovative approach, GH Research seeks to provide new therapeutic options for individuals who have not responded to conventional treatments.

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.

Visus Therapeutics is a clinical-stage company focused on developing innovative ophthalmic therapies aimed at enhancing vision for individuals globally. The company is known for its presbyopia-correcting eye drops, which are designed to provide temporary relief from the loss of near vision associated with presbyopia. Additionally, Visus Therapeutics is engaged in the development of various ophthalmic product candidates targeting conditions such as ocular surface disease, glaucoma, and age-related macular degeneration. Through its research and product offerings, the company seeks to address significant unmet needs in vision care.

Tenaya Therapeutics is a biotechnology company based in South San Francisco, California, focused on developing innovative therapies for heart disease, particularly heart failure. Established in 2016, the company employs three main platforms to address the underlying causes of heart disease: a cellular regeneration platform that reprograms cardiac fibroblasts into cardiomyocytes using proprietary transcription factors, a gene therapy platform for targeted delivery of therapeutic payloads, and a precision medicine approach tailored to individual patients. Through these platforms, Tenaya Therapeutics aims to enable the regeneration of heart tissue and tackle various forms of cardiomyopathies, leveraging advanced research in cardiac development and regeneration.

Artiva Biotherapeutics is a biotech company focused on developing and manufacturing cellular immunotherapies for cancer patients. Founded in 2019 and based in San Diego, California, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating various hematologic cancers and solid tumors. The company's product pipeline targets specific antigens, including CD20 and CD19 for B-cell lymphomas, as well as HER2 for certain solid tumors. Artiva's approach emphasizes the therapeutic potential of NK cells and aims to overcome challenges in scaling and manufacturing these therapies. The company is committed to delivering safe, effective, and readily accessible cell therapies to benefit cancer patients. Artiva operates as a subsidiary of Green Cross Holdings Corporation.

Biomea Fusion is a preclinical-stage biopharmaceutical company dedicated to advancing precision medicine for cancer treatment. The company's focus is on the discovery and development of irreversible small molecule drugs that target specific genetic alterations associated with tumor growth. Its lead product candidate, BMF-219, is designed as a potent and selective irreversible inhibitor of menin, a key transcriptional regulator involved in oncogenic signaling across various cancers. Biomea Fusion aims to expedite the development of these innovative therapies to provide highly effective treatment options for patients with genetically defined cancers.

Nuance Biotech Inc. is a pharmaceutical development company based in Shanghai, China, founded in 2014. The company focuses on the research and development of innovative drug formulations, offering products in the form of capsules, injections, and tubes. In addition to its core pharmaceutical development activities, Nuance Biotech also provides commercial and academic platforms for drug-related ventures and offers contract sales organization services. Through its diverse capabilities, the company aims to advance healthcare solutions and contribute to the pharmaceutical industry.

NiKang Therapeutics, Inc. is a biotechnology company established in 2017 and located in Wilmington, Delaware. The company is dedicated to discovering and developing innovative small molecule oncology medicines aimed at addressing unmet medical needs in cancer treatment. By employing a discovery approach that leverages target structure biology and structure-based drug design, NiKang Therapeutics aims to facilitate the rapid and efficient development of proprietary drug candidates with optimal pharmacological properties. The company seeks to enhance the treatment options available to healthcare providers, ultimately improving patient outcomes in the fight against cancer.

Encoded Therapeutics, Inc. is a biotechnology company that specializes in precision gene therapies aimed at treating a wide array of severe genetic disorders. Utilizing a unique platform, Encoded identifies sequences within the human genome that regulate gene expression through advanced screening and computational techniques. The company's therapy pipeline targets both genetic and acquired disorders across various disease pathways, including neurocircuitry, liver and metabolic disorders, neurodegeneration, and cardiovascular diseases. Founded in 2014 and based in South San Francisco, California, Encoded Therapeutics is focused on developing innovative solutions that enable medical practitioners to improve patient outcomes and enhance quality of life.

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

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 that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

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

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 a focus on discovering and developing novel therapeutics for cancer, viral diseases, and autoimmune conditions. The company specializes in T cell receptor (TCR) technology, which allows it to create innovative bispecific immunotherapies. Its lead product candidate, IMCgp100, is currently undergoing clinical trials for the treatment of metastatic melanoma. Immunocore's proprietary platform enables efficient manufacturing of TCR-based drugs, addressing significant unmet medical needs. Founded in 1999 and previously known as Avidex Limited, Immunocore has expanded its operations with an additional office in Conshohocken.

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.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

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

Encoded Therapeutics, Inc. is a biotechnology company that specializes in precision gene therapies aimed at treating a wide array of severe genetic disorders. Utilizing a unique platform, Encoded identifies sequences within the human genome that regulate gene expression through advanced screening and computational techniques. The company's therapy pipeline targets both genetic and acquired disorders across various disease pathways, including neurocircuitry, liver and metabolic disorders, neurodegeneration, and cardiovascular diseases. Founded in 2014 and based in South San Francisco, California, Encoded Therapeutics is focused on developing innovative solutions that enable medical practitioners to improve patient outcomes and enhance quality of life.

Pulmonx Corporation is a medical technology company specializing in minimally invasive solutions for the diagnosis and treatment of chronic obstructive pulmonary disease (COPD), particularly severe emphysema. Its primary offerings include the Zephyr Endobronchial Valve, designed for patients with hyperinflation due to emphysema, as well as the Chartis Pulmonary Assessment System, which assesses collateral ventilation, and the StratX Lung Analysis Platform, a cloud-based service that analyzes computed tomography data to identify suitable treatment areas. Pulmonx aims to improve the quality of life for patients who remain symptomatic despite medical management and may not be candidates for surgical interventions. The company has received FDA pre-market approval for the Zephyr Valve and has established a global presence, serving markets in the United States, Europe, the Middle East, Africa, and the Asia-Pacific region. Founded in 1995 and headquartered in Redwood City, California, Pulmonx has become a leader in interventional procedures for COPD, supported by extensive clinical evidence demonstrating the effectiveness of its technologies.

Orchestra BioMed is a biomedical innovation company based in New Hope, Pennsylvania, founded in 2017. The company specializes in developing high-impact therapeutic solutions for major medical conditions, with a primary focus on cardiovascular diseases, which are responsible for approximately 31% of global deaths annually. Orchestra BioMed's business model involves strategic partnerships with medical device companies to accelerate the commercialization of its products. Its key product candidates include BackBeat Cardiac Neuromodulation Therapy for hypertension and Virtue Sirolimus AngioInfusionBalloon for atherosclerotic artery disease.

Magnolia Medical Technologies, Inc. is a medical device company based in Seattle, Washington, focused on enhancing the accuracy of diagnostic blood culture tests. Established in 2011, the company has developed SteriPath, a vacuum-assisted blood collection system designed to prevent contamination of blood samples, which can lead to misdiagnosis of sepsis. This innovative system operates by actively diverting and sequestering contaminated blood, allowing for the collection of uncontaminated samples through a separate flow path. This closed vein-to-bottle collection method aims to improve diagnostic specimen quality, thereby increasing diagnostic accuracy and improving patient outcomes. Magnolia Medical Technologies holds a robust intellectual property portfolio, including over 70 issued patents and more than 50 pending applications, reflecting its commitment to advancing medical technology and addressing challenges such as antibiotic resistance. The company's products are distributed throughout the United States.

Milestone Pharmaceuticals Inc. is a biopharmaceutical company based in Montréal, Canada, focused on developing and commercializing etripamil for various cardiovascular conditions. Etripamil is a novel short-acting calcium channel blocker designed to be administered as a rapid-onset nasal spray, specifically targeting the termination of episodes of paroxysmal supraventricular tachycardia (PSVT). The company is currently conducting Phase III clinical trials for etripamil in the treatment of PSVT and is also exploring its potential applications for atrial fibrillation, angina, and other related cardiovascular disorders. Founded in 2003, Milestone Pharmaceuticals aims to address unmet medical needs in cardiovascular disease management through innovative therapeutic solutions.

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, now known as Novartis Gene Therapies, is a biotechnology company focused on developing and commercializing gene therapies aimed at treating rare and life-threatening neurological genetic disorders. Acquired by Novartis in 2018, the company specializes in motor neuron cell-targeted gene replacement therapies for conditions such as spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. By addressing these severe diseases, which often result in progressive muscle weakness, paralysis, and death, AveXis plays a crucial role in enhancing treatment options for affected patients and their families.

Immunocore Limited is a biotechnology company based in Abingdon, United Kingdom, with a focus on discovering and developing novel therapeutics for cancer, viral diseases, and autoimmune conditions. The company specializes in T cell receptor (TCR) technology, which allows it to create innovative bispecific immunotherapies. Its lead product candidate, IMCgp100, is currently undergoing clinical trials for the treatment of metastatic melanoma. Immunocore's proprietary platform enables efficient manufacturing of TCR-based drugs, addressing significant unmet medical needs. Founded in 1999 and previously known as Avidex Limited, Immunocore has expanded its operations with an additional office in Conshohocken.

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.

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.

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.