OrbiMed

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.

Leal Therapeutics is a Massachusetts-based company focused on developing innovative therapeutics aimed at addressing significant and unmet needs in central nervous system disorders. By leveraging precision medicine, Leal Therapeutics seeks to provide effective treatment options for patients suffering from various neurological conditions. The company's commitment to advancing therapeutic solutions is reflected in its dedication to improving patient outcomes through targeted interventions.

Founded in 2020 and headquartered in Boston, Massachusetts, Scorpion Therapeutics is a clinical-stage biotechnology company specializing in precision oncology. It develops targeted small-molecule drugs to treat cancer by focusing on three areas: therapies against known oncogenes, agents for currently undruggable targets, and drugs for new targets.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

Q32 Bio is a clinical-stage biotechnology company focused on restoring healthy immune balance in autoimmune and inflammatory diseases by developing antibody-based therapies that modulate regulators of the innate and adaptive immune systems. Its development program includes ADX-914, a human anti-IL-7R antibody aimed at rebalancing adaptive immune function, and ADX-097, which demonstrates tissue-targeted distribution and durable pharmacokinetics/pharmacodynamics. Founded in 2017 and based in Cambridge, Massachusetts, the company emphasizes a team of scientists and immunology experts pursuing novel biologics to address diseases with limited or inadequate treatment options.

BiomX develops bacteriophage-based therapies targeting bacteria linked to skin conditions and chronic diseases such as inflammatory bowel disease, liver disorders, and cancer. It collaborates with renowned institutions for research and development, aiming to create innovative microbiome therapeutics.

Pinnacle Medicines is a biotechnology company based in Doylestown, Pennsylvania. It specializes in peptide drug discovery, utilizing AI-based tools to develop safe and effective therapeutics for unmet medical needs.

BiomX develops bacteriophage-based therapies targeting bacteria linked to skin conditions and chronic diseases such as inflammatory bowel disease, liver disorders, and cancer. It collaborates with renowned institutions for research and development, aiming to create innovative microbiome therapeutics.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

Founded in 2014, Compass Therapeutics is a clinical-stage biopharmaceutical company developing antibody therapeutics to treat solid tumors and hematological malignancies. Its pipeline includes CTX-471, an agonistic antibody of CD137 for immune cell activation, currently in Phase I trials; CTX-8371, a bispecific antibody targeting PD-1 and PD-L1, in IND-enabling studies; and CTX-009, a bispecific antibody inhibiting DLL4 and VEGF-A signaling, completed Phase I and is in Phase Ib combination with chemotherapy.

PIC Therapeutics, Inc. is a biotechnology company dedicated to advancing cancer treatment through the selective modulation of oncogene translation. Founded in 2016 and headquartered in Boston, Massachusetts, the company develops precision-based therapeutics that target the master switch of cancer signaling pathways. By modulating the pre-initiation complex responsible for messenger RNA translation, PIC Therapeutics aims to selectively block the production of oncogene proteins. This innovative approach has the potential to simultaneously address multiple oncogenic drivers, paving the way for a new generation of cancer therapies that could significantly enhance patient outcomes.

Treeline Biosciences is a biotechnology company focused on developing precision medicines for cancer and other serious diseases. Founded in 2021 and based in Stamford, Connecticut, the company uses a platform that integrates pathophysiology, genomics, and pattern recognition to identify therapeutic approaches that address unmet patient needs.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

X4 Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing novel oral small molecule therapeutics targeting chemokine receptor CXCR4. Its lead drug candidate, mavorixafor (X4P-001), is in Phase III trials for the treatment of WHIM syndrome and has shown promising results in other indications such as severe congenital neutropenia and clear cell renal cell carcinoma.

Leal Therapeutics is a Massachusetts-based company focused on developing innovative therapeutics aimed at addressing significant and unmet needs in central nervous system disorders. By leveraging precision medicine, Leal Therapeutics seeks to provide effective treatment options for patients suffering from various neurological conditions. The company's commitment to advancing therapeutic solutions is reflected in its dedication to improving patient outcomes through targeted interventions.

Scout Bio, Inc. is a biotechnology company focused on advancing veterinary medicine through innovative therapies for chronic pet health conditions. Founded in 2016 and headquartered in Philadelphia, Pennsylvania, the company specializes in developing one-time treatments utilizing veterinary biotechnology and gene therapy. Scout Bio's offerings include therapies for conditions such as anemia associated with chronic kidney disease in cats, as well as a range of small molecular drugs, protein therapies, and gene therapies. Employing adeno-associated viral vector technology, Scout Bio aims to transform veterinary care by providing effective protein and monoclonal antibody therapeutics delivered through single intra-muscular injections, thereby enhancing the quality of life for pets and supporting veterinary professionals.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

Carrot Fertility, Inc. offers comprehensive fertility benefits services for employers, supporting employees throughout their fertility healthcare journey on a global scale. Founded in 2015 and headquartered in San Francisco, California, the company provides a range of services including egg freezing, in vitro fertilization, adoption, donor and gestational carrier services. It also features Carrot Rx, a premium pharmacy experience for fertility medications, and Carrot Pregnancy, which offers virtual access to experts and on-demand doctor-approved content. Additionally, Carrot Card is a flexible debit card that employees can use to pay for fertility care. Carrot Fertility aims to help companies customize their fertility benefits, ensuring that employees receive the necessary financial, medical, and emotional support as they pursue parenthood. The company has expanded its presence with additional offices in major cities including Chicago, New York, Dublin, Geneva, Tokyo, and Seoul.

GentiBio, Inc. is a biotherapeutics company that specializes in developing engineered regulatory T cells, known as EngTregs, aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

XtalPi Inc. is a pharmaceutical technology company that focuses on digital drug development solutions, leveraging quantum mechanics and artificial intelligence to enhance the efficiency of drug research and development. Founded in 2014 by a group of quantum physicists at MIT, the company has developed its Intelligent Digital Drug Discovery and Development (ID4) platform, which integrates advanced cloud computing algorithms to aid pharmaceutical firms in critical stages of drug R&D. XtalPi offers various tools, including XtalForc for visual force field calculations, XtalVision for crystal structure predictions, and Renova for platform support of its AI research initiatives. Its comprehensive services encompass crystal structure determination, solid-state testing, small molecule drug design, and collaborations in antibody and peptide research. Based in Cambridge, Massachusetts, with additional offices in China, XtalPi has established strategic partnerships with leading international pharmaceutical companies, demonstrating the broad applicability and recognition of its innovative technologies across the pharmaceutical value chain.

Turnstone Biologics is a clinical-stage biotechnology company focused on developing viral immunotherapies to improve cancer survival. Its lead candidate, RIVAL-01, combines three immunomodulators to stimulate immune activity and reprogram the tumor microenvironment.

Biocytogen specializes in developing gene-targeted animal models for life science research, offering customized mouse models, phenotype analysis, and extreme genome editing platforms. It also provides integrated solutions for antibody drug development, including animal model generation, therapeutic antibody discovery, and preclinical validation studies.

Magenta Therapeutics, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on advancing stem cell transplantation and related therapies. Founded in 2015, the company aims to enhance the patient experience in transplant medicine by developing innovative treatments that make stem cell transplants safer and more accessible. Its pipeline includes several key candidates, such as C100, C200, and C300 targeted antibody-drug conjugates designed for transplant conditioning; MGTA-145, a stem cell mobilization product; and MGTA-456, an allogeneic stem cell therapy. Additionally, Magenta is exploring E478, a small molecule aimed at expanding gene-modified stem cells, and G100, an antibody-drug conjugate to mitigate acute graft-versus-host disease. The company has established research collaborations with other biotechnology firms to further investigate the potential of its therapies in treating blood cancers, genetic disorders, and autoimmune diseases, ultimately striving to transform stem cell transplantation from a last-resort option into a more viable and effective treatment for patients.

Treeline Biosciences is a biotechnology company focused on developing precision medicines for cancer and other serious diseases. Founded in 2021 and based in Stamford, Connecticut, the company uses a platform that integrates pathophysiology, genomics, and pattern recognition to identify therapeutic approaches that address unmet patient needs.

X4 Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing novel oral small molecule therapeutics targeting chemokine receptor CXCR4. Its lead drug candidate, mavorixafor (X4P-001), is in Phase III trials for the treatment of WHIM syndrome and has shown promising results in other indications such as severe congenital neutropenia and clear cell renal cell carcinoma.

Century Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies derived from induced pluripotent stem cells (iPSCs) to treat hematologic and solid malignancies. Founded in 2018 and headquartered in Philadelphia, Pennsylvania, the company utilizes a specialized platform that integrates gene editing and protein engineering to create allogeneic therapies. This platform allows for the generation of master cell banks of modified immune effector cells, specifically engineered to optimize performance and reduce the risk of rejection by the host immune system. By employing advanced technologies such as CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to address significant unmet medical needs in cancer and other diseases.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

Founded in 2020 and headquartered in Boston, Massachusetts, Scorpion Therapeutics is a clinical-stage biotechnology company specializing in precision oncology. It develops targeted small-molecule drugs to treat cancer by focusing on three areas: therapies against known oncogenes, agents for currently undruggable targets, and drugs for new targets.

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.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

Q32 Bio is a clinical-stage biotechnology company focused on restoring healthy immune balance in autoimmune and inflammatory diseases by developing antibody-based therapies that modulate regulators of the innate and adaptive immune systems. Its development program includes ADX-914, a human anti-IL-7R antibody aimed at rebalancing adaptive immune function, and ADX-097, which demonstrates tissue-targeted distribution and durable pharmacokinetics/pharmacodynamics. Founded in 2017 and based in Cambridge, Massachusetts, the company emphasizes a team of scientists and immunology experts pursuing novel biologics to address diseases with limited or inadequate treatment options.

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

Novellus is a biotechnology company working to develop engineered cellular medicines. The company uses its non-immunogenic mRNA molecules to reprogram and gene-edit cells safely, and with high efficiency. They engineer precision medicines for patient segments and select from its technology toolbox for in vivo, ex vivo, allogeneic and autologous delivery. Novellus platform enables the high-level expression of gene-editing and cell-reprogramming proteins for high-specificity gene repair and both autologous and allogeneic cell therapies. The company is advancing its lead program, a breakthrough therapy for a rare dermatologic disease, through pre-clinical studies. Novellus was founded in 2015 and is headquartered in Cambridge, Massachusetts, United States.

CoreMap, Inc. is a medical technology company based in Winooski, Vermont, focused on developing advanced diagnostic solutions for electrophysiologists treating atrial fibrillation. Incorporated in 2016, the company offers a micro-electrode solution that allows for sequential mapping of tissue properties, addressing significant challenges in the treatment of atrial fibrillation, such as poor spatial resolution and inadequate sample density. Additionally, CoreMap's ultra-high resolution electrodes provide detailed insights into cardiac activation, even during episodes of fibrillation. By enhancing the diagnostic capabilities of electrophysiologists, CoreMap aims to improve patient outcomes in the management of this complex condition.

MobileODT develops optical diagnostic devices and software for early cancer detection, enabling health care providers to perform visual examinations with portable, internet-connected tools. The Enhanced Visual Assessment system includes the FDA-cleared EVA COLPO portable colposcope and accompanying software to securely store and manage patient data via HIPAA-compliant cloud services such as EVA SANE, and a digital visualization tool for gynecologic exams, EVA WELL. The company also offers a portfolio of aids for screening, including a mobile app for Android that enables remote image capture and patient information tracking, an online portal for collaboration and reporting, and cloud storage solutions for documented patient data. Founded in 2012 and based in Tel Aviv with an additional location in New York, MobileODT aims to extend expert-level diagnostic capabilities to diverse healthcare settings and geographies, including applications in cervical cancer screening and other visual-based diagnostics.

GentiBio, Inc. is a biotherapeutics company that specializes in developing engineered regulatory T cells, known as EngTregs, aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, Washington, GentiBio employs a proprietary platform that combines autologous and allogeneic technologies. This platform enhances the ability to restore immune tolerance and addresses significant limitations found in existing regulatory T-cell therapeutics. By focusing on the underlying mechanisms of immune system disorders, GentiBio aims to provide innovative therapeutic solutions that tackle the root causes of diseases caused by dysregulated immune responses. The company is co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of advanced immunotherapy development.

PMV Pharmaceuticals, Inc. is a precision oncology company focused on the discovery and development of small molecule therapies targeting p53 mutations in cancer. Its lead candidate, PC14586, aims to correct and restore the function of the p53 protein, which is critical in regulating cell growth and preventing tumor formation. In addition to PC14586, the company is developing therapies for various p53 hotspot mutations, including the R273H mutation. Founded in 2013 and headquartered in Cranbury, New Jersey, PMV Pharmaceuticals leverages extensive expertise in p53 biology to advance its innovative treatment options for cancer patients.

Founded in 2014, Compass Therapeutics is a clinical-stage biopharmaceutical company developing antibody therapeutics to treat solid tumors and hematological malignancies. Its pipeline includes CTX-471, an agonistic antibody of CD137 for immune cell activation, currently in Phase I trials; CTX-8371, a bispecific antibody targeting PD-1 and PD-L1, in IND-enabling studies; and CTX-009, a bispecific antibody inhibiting DLL4 and VEGF-A signaling, completed Phase I and is in Phase Ib combination with chemotherapy.

Q32 Bio is a clinical-stage biotechnology company focused on restoring healthy immune balance in autoimmune and inflammatory diseases by developing antibody-based therapies that modulate regulators of the innate and adaptive immune systems. Its development program includes ADX-914, a human anti-IL-7R antibody aimed at rebalancing adaptive immune function, and ADX-097, which demonstrates tissue-targeted distribution and durable pharmacokinetics/pharmacodynamics. Founded in 2017 and based in Cambridge, Massachusetts, the company emphasizes a team of scientists and immunology experts pursuing novel biologics to address diseases with limited or inadequate treatment options.

TytoCare develops handheld devices and software that enable users to perform self-examinations, facilitating remote diagnosis by healthcare professionals. The company's tools allow examination of various body areas and diagnose common conditions. TytoCare offers solutions for consumers (TytoHome), professionals (TytoPro), and remote clinics (TytoClinic). Incorporated in 2011, the company is headquartered in Netanya, Israel with an additional office in New York.

Pandion Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, focused on developing innovative therapeutics for patients with autoimmune diseases. The company’s lead product candidate, PT101, is an engineered interleukin-2 variant currently undergoing Phase 1a clinical trials for various autoimmune and inflammatory conditions, including moderate-to-severe ulcerative colitis and systemic lupus erythematosus. In addition to PT101, Pandion's pipeline includes PT627, a systemic PD-1 agonist in preclinical studies, and two bifunctional molecules, PT001 and PT002, designed to provide tissue-selective immunomodulation by targeting specific adhesion molecules in the gastrointestinal tract. Pandion employs a network-based approach to immune system conceptualization through its TALON drug design platform and collaborates with Astellas Pharma to develop locally acting immunomodulators for pancreatic autoimmune diseases. The company aims to create a new generation of therapeutics that offer enhanced efficacy and safety for the treatment of autoimmune and inflammatory diseases.

Keros Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Lexington, Massachusetts, dedicated to the discovery, development, and commercialization of innovative treatments for hematological and musculoskeletal disorders with significant unmet medical needs. The company's lead product candidate, KER-050, targets low blood cell counts, or cytopenias, including anemia and thrombocytopenia in patients with myelodysplastic syndromes and myelofibrosis. Additionally, Keros is developing KER-047, a small molecule aimed at treating anemia and fibrodysplasia ossificans progressiva, which is currently in Phase 1 clinical trials. Another candidate, KER-012, is being explored for its potential in treating bone loss disorders such as osteoporosis and osteogenesis imperfecta, as well as pulmonary arterial hypertension. Keros Therapeutics emphasizes understanding the role of the Transforming Growth Factor-Beta family of proteins, which are crucial for the regulation of blood cell production and the maintenance of muscle and bone health.

Harmony Biosciences Holdings is a commercial-stage pharmaceutical company based in Plymouth Meeting, Pennsylvania, founded in 2017. It develops and commercializes therapies for rare neurological disorders with unmet medical needs. Its lead product is WAKIX (pitolisant), used to treat narcolepsy, and the company maintains a pipeline that includes candidates such as HBS-102, BP1.15205, ZYN002, EPX-100 and EPX-200.

PMV Pharmaceuticals, Inc. is a precision oncology company focused on the discovery and development of small molecule therapies targeting p53 mutations in cancer. Its lead candidate, PC14586, aims to correct and restore the function of the p53 protein, which is critical in regulating cell growth and preventing tumor formation. In addition to PC14586, the company is developing therapies for various p53 hotspot mutations, including the R273H mutation. Founded in 2013 and headquartered in Cranbury, New Jersey, PMV Pharmaceuticals leverages extensive expertise in p53 biology to advance its innovative treatment options for cancer patients.

Passage Bio is a gene therapy company dedicated to developing transformative treatments for rare, monogenic central nervous system disorders. It has an integrated pipeline of five AAV-delivered therapeutics and collaborates with the University of Pennsylvania's Gene Therapy Program for preclinical work.

Foamix Pharmaceuticals is a specialty pharmaceutical company based in Bridgewater, New Jersey, that focuses on the development and commercialization of proprietary topical drugs for dermatological conditions, as well as for ophthalmologic and gynecologic applications. Founded in 2014, the company utilizes its Molecule Stabilizing Technology platform to create innovative foam-based formulations. Its lead product, a first-in-class Topical Minocycline Foam, targets significant markets in dermatology and ophthalmology, having shown high efficacy and favorable safety in clinical trials for conditions such as acne and impetigo. Foamix's pipeline includes multiple drug candidates aimed at treating various skin conditions, including FMX101, FMX102, FMX103, and FDX104. The company is also engaged in development and licensing agreements with other pharmaceutical firms, enhancing its reach in the healthcare market.

Athenex, Inc. is a biopharmaceutical company focused on the discovery, development, and commercialization of innovative therapies for cancer and related conditions. Founded in 2003 and headquartered in Buffalo, New York, Athenex operates across North America and Asia, with a clinical pipeline that includes several promising product candidates. The company’s Orascovery platform features oral formulations of well-known chemotherapeutic agents such as Paclitaxel, Irinotecan, Docetaxel, Topotecan, and Eribulin, which are in various stages of clinical trials targeting multiple cancer types. Additionally, Athenex is developing Src Kinase inhibitors for skin cancers and psoriasis, as well as advanced immunotherapies and arginine deprivation therapies. Through its commitment to innovative research and collaborations, Athenex aims to become a leader in delivering effective cancer treatments and improving patient health outcomes globally.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company applying precision medicine to develop medicines for severe rare diseases and cancer. It maintains a differentiated targeted oncology portfolio with lead candidates nirogacestat, a gamma secretase inhibitor, and mirdametinib, a MEK inhibitor, both in late-stage trials; the pipeline also includes BGB-3245, an oral inhibitor of activating BRAF mutations, in Phase I, and several combination programs. The company pursues collaborations with BeiGene, GlaxoSmithKline, and Allogene and has license agreements with Pfizer for nirogacestat and mirdametinib. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks aims to advance potentially registrational studies in rare tumor types and genetically defined cancers through partnerships that expand its portfolio and patient access.

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York, specializing in the development and commercialization of gene therapies targeting neurodegenerative diseases. Founded in 2017, the company focuses on adeno-associated virus (AAV)-based therapies, with its lead candidate, PR001, currently undergoing Phase I/II clinical trials for Parkinson's disease associated with GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is advancing PR006 for frontotemporal dementia linked to GRN mutations and PR004 for various synucleinopathies. The company's mission is to provide disease-modifying treatments for patients suffering from these challenging conditions.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that specializes in developing and commercializing therapeutics for patients with rare genetic disorders of hemoglobin. Founded in 2016, the company is advancing IMR-687, an oral, once-daily therapeutic designed to treat sickle cell disease and beta-thalassemia. IMR-687 is a highly selective small molecule inhibitor of PDE9, engineered to have a multimodal mechanism of action that impacts various cell types, including red blood cells and white blood cells, as well as adhesion mediators. Through its innovative approach, Imara aims to provide disease-modifying treatments for individuals affected by hemoglobinopathies.

BiomX develops bacteriophage-based therapies targeting bacteria linked to skin conditions and chronic diseases such as inflammatory bowel disease, liver disorders, and cancer. It collaborates with renowned institutions for research and development, aiming to create innovative microbiome therapeutics.

Passage Bio is a gene therapy company dedicated to developing transformative treatments for rare, monogenic central nervous system disorders. It has an integrated pipeline of five AAV-delivered therapeutics and collaborates with the University of Pennsylvania's Gene Therapy Program for preclinical work.

Founded in 2014, Compass Therapeutics is a clinical-stage biopharmaceutical company developing antibody therapeutics to treat solid tumors and hematological malignancies. Its pipeline includes CTX-471, an agonistic antibody of CD137 for immune cell activation, currently in Phase I trials; CTX-8371, a bispecific antibody targeting PD-1 and PD-L1, in IND-enabling studies; and CTX-009, a bispecific antibody inhibiting DLL4 and VEGF-A signaling, completed Phase I and is in Phase Ib combination with chemotherapy.

Avedro, Inc. is a commercial-stage ophthalmic pharmaceutical and medical device company headquartered in Waltham, Massachusetts. The company specializes in developing and commercializing innovative products for treating corneal disorders, primarily focusing on corneal ectatic conditions and refractive issues. Avedro's proprietary Corneal Remodeling Platform includes the KXL and Mosaic systems, which utilize ultraviolet A (UVA) light in conjunction with riboflavin drug formulations for corneal cross-linking. This technology is aimed at strengthening and reshaping the cornea through minimally invasive and non-invasive outpatient procedures. Avedro has also introduced the Keraflex refractive correction procedure, a non-invasive technique for flattening the cornea without tissue removal. While Avedro primarily serves ophthalmologists, hospitals, and ambulatory surgery centers in the United States, it also markets its products internationally through medical device distributors. The company, originally named ThermalVision, Inc., was incorporated in 2002 and rebranded as Avedro in 2005. As of November 2019, Avedro operates as a subsidiary of Glaukos Corporation.

Foamix Pharmaceuticals is a specialty pharmaceutical company based in Bridgewater, New Jersey, that focuses on the development and commercialization of proprietary topical drugs for dermatological conditions, as well as for ophthalmologic and gynecologic applications. Founded in 2014, the company utilizes its Molecule Stabilizing Technology platform to create innovative foam-based formulations. Its lead product, a first-in-class Topical Minocycline Foam, targets significant markets in dermatology and ophthalmology, having shown high efficacy and favorable safety in clinical trials for conditions such as acne and impetigo. Foamix's pipeline includes multiple drug candidates aimed at treating various skin conditions, including FMX101, FMX102, FMX103, and FDX104. The company is also engaged in development and licensing agreements with other pharmaceutical firms, enhancing its reach in the healthcare market.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.

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.

Prevail Therapeutics Inc. is a biotechnology company headquartered in New York, specializing in the development and commercialization of gene therapies targeting neurodegenerative diseases. Founded in 2017, the company focuses on adeno-associated virus (AAV)-based therapies, with its lead candidate, PR001, currently undergoing Phase I/II clinical trials for Parkinson's disease associated with GBA1 mutations and neuronopathic Gaucher disease. Additionally, Prevail is advancing PR006 for frontotemporal dementia linked to GRN mutations and PR004 for various synucleinopathies. The company's mission is to provide disease-modifying treatments for patients suffering from these challenging conditions.

TytoCare develops handheld devices and software that enable users to perform self-examinations, facilitating remote diagnosis by healthcare professionals. The company's tools allow examination of various body areas and diagnose common conditions. TytoCare offers solutions for consumers (TytoHome), professionals (TytoPro), and remote clinics (TytoClinic). Incorporated in 2011, the company is headquartered in Netanya, Israel with an additional office in New York.

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.

resTORbio, Inc., founded in 2016 and headquartered in Boston, Massachusetts, was a clinical-stage biopharmaceutical company focused on developing medicines to treat aging-related diseases. Its primary approach involved selectively inhibiting the target of rapamycin complex 1 (TORC1), a pathway linked to age-related decline in organ functions. The company's lead drug candidate, RTB101, an oral and potent TORC1 inhibitor, was being evaluated in Phase 1b/2a clinical trials for Parkinson's disease and other neurodegenerative diseases. Prior to its acquisition by Adicet Bio in September 2020, resTORbio collaborated with TrialSpark to investigate RTB-101 as a potential treatment for COVID-19.

RDD Pharma, Ltd. is a pharmaceutical company based in Tel-Aviv, Israel, with an additional office in New York, New York. Founded in 2008, RDD Pharma focuses on the development and commercialization of targeted therapeutics for anorectal diseases and gastrointestinal disorders. The company specializes in creating innovative pharmacological treatments for conditions such as chronic anal fissures, fecal incontinence, and pruritus ani, particularly in patients with spinal cord injuries. RDD Pharma addresses the unique challenges of treating these conditions by employing specialized formulation and delivery techniques that cater to the anatomical and physiological variations in this sensitive area.

TELA Bio is a medical technology company specializing in soft tissue reconstruction. It designs, develops, and markets the OviTex Reinforced Tissue Matrix portfolio for hernia repair and abdominal wall reconstruction, as well as OviTex PR Reinforced Tissue Matrix for plastic and reconstructive surgery and OviTex for laparoscopic and robotic procedures. The reinforced matrices are derived from ovine rumen with polypropylene fiber and are intended to address unmet needs in soft tissue repair while emphasizing preservation of anatomy and minimizing long-term exposure to permanent synthetic materials. The company markets its products through a single direct sales force, principally in the United States. Founded in 2012 and headquartered in Malvern, Pennsylvania.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

SpringWorks Therapeutics is a clinical-stage biopharmaceutical company applying precision medicine to develop medicines for severe rare diseases and cancer. It maintains a differentiated targeted oncology portfolio with lead candidates nirogacestat, a gamma secretase inhibitor, and mirdametinib, a MEK inhibitor, both in late-stage trials; the pipeline also includes BGB-3245, an oral inhibitor of activating BRAF mutations, in Phase I, and several combination programs. The company pursues collaborations with BeiGene, GlaxoSmithKline, and Allogene and has license agreements with Pfizer for nirogacestat and mirdametinib. Founded in 2017 and headquartered in Stamford, Connecticut, SpringWorks aims to advance potentially registrational studies in rare tumor types and genetically defined cancers through partnerships that expand its portfolio and patient access.

Founded in 2014, Compass Therapeutics is a clinical-stage biopharmaceutical company developing antibody therapeutics to treat solid tumors and hematological malignancies. Its pipeline includes CTX-471, an agonistic antibody of CD137 for immune cell activation, currently in Phase I trials; CTX-8371, a bispecific antibody targeting PD-1 and PD-L1, in IND-enabling studies; and CTX-009, a bispecific antibody inhibiting DLL4 and VEGF-A signaling, completed Phase I and is in Phase Ib combination with chemotherapy.

MobileODT develops optical diagnostic devices and software for early cancer detection, enabling health care providers to perform visual examinations with portable, internet-connected tools. The Enhanced Visual Assessment system includes the FDA-cleared EVA COLPO portable colposcope and accompanying software to securely store and manage patient data via HIPAA-compliant cloud services such as EVA SANE, and a digital visualization tool for gynecologic exams, EVA WELL. The company also offers a portfolio of aids for screening, including a mobile app for Android that enables remote image capture and patient information tracking, an online portal for collaboration and reporting, and cloud storage solutions for documented patient data. Founded in 2012 and based in Tel Aviv with an additional location in New York, MobileODT aims to extend expert-level diagnostic capabilities to diverse healthcare settings and geographies, including applications in cervical cancer screening and other visual-based diagnostics.

LogicBio Therapeutics, Inc. is a genome editing company dedicated to developing innovative treatments for rare diseases, particularly for pediatric patients with significant unmet medical needs. Utilizing its proprietary GeneRide technology platform, LogicBio aims to integrate corrective genes into patients' genomes to achieve therapeutic effects. The company's lead product candidate, LB-001, targets Methylmalonic Acidemia, a serious condition that manifests at birth. Additionally, LogicBio is advancing other product candidates, including LB-101 and LB-301, the latter developed in collaboration with Takeda Pharmaceutical Company to address Crigler-Najjar syndrome. The company also partners with the Children's Medical Research Institute to create new viral vectors, enhancing the potential of its gene therapy approaches. Founded in 2014, LogicBio is headquartered in Lexington, Massachusetts.

Avitide, Inc. specializes in the discovery, manufacture, and supply of molecule-specific affinity purification solutions tailored for the biopharmaceutical industry. Founded in 2012 and based in Lebanon, New Hampshire, the company focuses on developing customized affinity purification products that improve the efficiency and effectiveness of bioprocessing. Avitide's technology enhances the purification of therapeutic proteins, including enzymes, antibodies, gene therapies, and vaccines, by employing rapid development of chromatography resins through chemical synthesis. This advanced platform enables clients to accelerate bioprocess development timelines, mitigate program risks, and achieve predictable commercial scalability while lowering manufacturing costs in both batch and continuous operations.

Tisbury Pharmaceuticals, Inc. is a biotechnology company focused on the discovery, development, and commercialization of therapies for primary open-angle glaucoma. Established in 2017 and located in Beverly, Massachusetts, the company is advancing its clinical candidate, R-801. This innovative treatment works by enhancing fluid flow through the trabecular meshwork, utilizing mechanisms such as redox catalysis and K+-ATP channel activation. The company’s objective is to effectively reduce intraocular pressure, thereby providing significant relief for patients suffering from glaucoma.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

BiomX develops bacteriophage-based therapies targeting bacteria linked to skin conditions and chronic diseases such as inflammatory bowel disease, liver disorders, and cancer. It collaborates with renowned institutions for research and development, aiming to create innovative microbiome therapeutics.

Avedro, Inc. is a commercial-stage ophthalmic pharmaceutical and medical device company headquartered in Waltham, Massachusetts. The company specializes in developing and commercializing innovative products for treating corneal disorders, primarily focusing on corneal ectatic conditions and refractive issues. Avedro's proprietary Corneal Remodeling Platform includes the KXL and Mosaic systems, which utilize ultraviolet A (UVA) light in conjunction with riboflavin drug formulations for corneal cross-linking. This technology is aimed at strengthening and reshaping the cornea through minimally invasive and non-invasive outpatient procedures. Avedro has also introduced the Keraflex refractive correction procedure, a non-invasive technique for flattening the cornea without tissue removal. While Avedro primarily serves ophthalmologists, hospitals, and ambulatory surgery centers in the United States, it also markets its products internationally through medical device distributors. The company, originally named ThermalVision, Inc., was incorporated in 2002 and rebranded as Avedro in 2005. As of November 2019, Avedro operates as a subsidiary of Glaukos Corporation.

Arsanis, Inc. is a clinical-stage biopharmaceutical company headquartered in Waltham, Massachusetts, specializing in monoclonal antibody (mAb) immunotherapies for serious infectious diseases. Founded in 2010, Arsanis focuses on developing innovative therapies targeting various bacterial and viral pathogens. Its lead clinical program, ASN100, is currently in a Phase 2 trial aimed at preventing Staphylococcus aureus pneumonia in high-risk, mechanically ventilated patients. The company's preclinical pipeline includes mAbs directed against respiratory syncytial virus and Gram-negative bacteria, such as Klebsiella pneumoniae and Escherichia coli. Arsanis leverages advanced mAb discovery techniques to create a diverse range of therapeutic candidates with multiple mechanisms of action, addressing the urgent need for effective treatments in infectious disease management. The company also has research and preclinical development operations in Vienna, Austria, under Arsanis Biosciences GmbH.

PMV Pharmaceuticals, Inc. is a precision oncology company focused on the discovery and development of small molecule therapies targeting p53 mutations in cancer. Its lead candidate, PC14586, aims to correct and restore the function of the p53 protein, which is critical in regulating cell growth and preventing tumor formation. In addition to PC14586, the company is developing therapies for various p53 hotspot mutations, including the R273H mutation. Founded in 2013 and headquartered in Cranbury, New Jersey, PMV Pharmaceuticals leverages extensive expertise in p53 biology to advance its innovative treatment options for cancer patients.

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

Turnstone Biologics is a clinical-stage biotechnology company focused on developing viral immunotherapies to improve cancer survival. Its lead candidate, RIVAL-01, combines three immunomodulators to stimulate immune activity and reprogram the tumor microenvironment.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for eye diseases using its proprietary Mucus Penetrating Particles (MPP) technology. This technology enhances drug distribution and pharmacokinetics by coating mucosal surfaces with biocompatible, drug-loaded particles, allowing for improved local drug concentrations while minimizing systemic exposure. Kala's lead product candidate, KPI-121 0.25%, has completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS has also completed two Phase III trials for managing inflammation and pain after ocular surgery. The company is advancing KPI-285, a receptor tyrosine kinase inhibitor, in preclinical studies targeting retinal diseases. Established in 2009 and headquartered in Watertown, Massachusetts, Kala Pharmaceuticals continues to explore diverse product opportunities while focusing on internal development and research collaborations to address significant clinical needs.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

LogicBio Therapeutics, Inc. is a genome editing company dedicated to developing innovative treatments for rare diseases, particularly for pediatric patients with significant unmet medical needs. Utilizing its proprietary GeneRide technology platform, LogicBio aims to integrate corrective genes into patients' genomes to achieve therapeutic effects. The company's lead product candidate, LB-001, targets Methylmalonic Acidemia, a serious condition that manifests at birth. Additionally, LogicBio is advancing other product candidates, including LB-101 and LB-301, the latter developed in collaboration with Takeda Pharmaceutical Company to address Crigler-Najjar syndrome. The company also partners with the Children's Medical Research Institute to create new viral vectors, enhancing the potential of its gene therapy approaches. Founded in 2014, LogicBio is headquartered in Lexington, Massachusetts.

Avedro, Inc. is a commercial-stage ophthalmic pharmaceutical and medical device company headquartered in Waltham, Massachusetts. The company specializes in developing and commercializing innovative products for treating corneal disorders, primarily focusing on corneal ectatic conditions and refractive issues. Avedro's proprietary Corneal Remodeling Platform includes the KXL and Mosaic systems, which utilize ultraviolet A (UVA) light in conjunction with riboflavin drug formulations for corneal cross-linking. This technology is aimed at strengthening and reshaping the cornea through minimally invasive and non-invasive outpatient procedures. Avedro has also introduced the Keraflex refractive correction procedure, a non-invasive technique for flattening the cornea without tissue removal. While Avedro primarily serves ophthalmologists, hospitals, and ambulatory surgery centers in the United States, it also markets its products internationally through medical device distributors. The company, originally named ThermalVision, Inc., was incorporated in 2002 and rebranded as Avedro in 2005. As of November 2019, Avedro operates as a subsidiary of Glaukos Corporation.

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.

Selecta Biosciences, Inc. is a clinical-stage biopharmaceutical company focused on developing nanoparticle immunomodulatory drugs for the treatment and prevention of human diseases. The company's proprietary platform utilizes advances in immunobiology and nanotechnology to create targeted immunomodulatory nanoparticles. Selecta's lead product, SEL-212, has successfully completed Phase II clinical trials for chronic refractory gout. Additionally, the company is advancing gene therapy candidates in preclinical development for rare metabolic disorders, including SEL-302 for methylmalonic acidemia and SEL-313 for ornithine transcarbamylase deficiency. Founded in 2007 and headquartered in Watertown, Massachusetts, Selecta Biosciences has established collaborations with various organizations, including Spark Therapeutics and the Massachusetts Institute of Technology, to enhance its research and development capabilities.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

Syndax Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Waltham, Massachusetts, specializing in the development of therapies for cancer treatment. The company's lead candidate, entinostat, is a class I HDAC inhibitor currently in Phase III trials for advanced hormone receptor-positive, HER2-negative breast cancer. Additionally, Syndax is advancing SNDX-5613, a selective inhibitor targeting the Menin-MLL binding interaction, in Phase I/II trials for acute myeloid leukemia with specific mutations. The company also explores entinostat in combination with immunotherapies such as Keytruda and Tecentriq for various cancers, including non-small cell lung cancer and metastatic breast cancer. Furthermore, Syndax is developing axatilimab, a monoclonal antibody targeting the CSF-1 receptor, currently in trials for chronic graft-versus-host disease. The company maintains collaborative relationships with several major pharmaceutical entities and research organizations to enhance its drug development efforts. Founded in 2005, Syndax Pharmaceuticals is committed to advancing innovative treatments for patients with challenging malignancies.

Rhythm Metabolic is the subsidiary of Rhythm Pharmaceuticals that develops setmelanotide (RM-493), a novel peptide MC4 agonist for the treatment of obesity caused by genetic deficiencies in the MC4 pathway. It was established in 2013 and is headquartered in Boston, Massachusetts.

Avitide, Inc. specializes in the discovery, manufacture, and supply of molecule-specific affinity purification solutions tailored for the biopharmaceutical industry. Founded in 2012 and based in Lebanon, New Hampshire, the company focuses on developing customized affinity purification products that improve the efficiency and effectiveness of bioprocessing. Avitide's technology enhances the purification of therapeutic proteins, including enzymes, antibodies, gene therapies, and vaccines, by employing rapid development of chromatography resins through chemical synthesis. This advanced platform enables clients to accelerate bioprocess development timelines, mitigate program risks, and achieve predictable commercial scalability while lowering manufacturing costs in both batch and continuous operations.

TytoCare develops handheld devices and software that enable users to perform self-examinations, facilitating remote diagnosis by healthcare professionals. The company's tools allow examination of various body areas and diagnose common conditions. TytoCare offers solutions for consumers (TytoHome), professionals (TytoPro), and remote clinics (TytoClinic). Incorporated in 2011, the company is headquartered in Netanya, Israel with an additional office in New York.

Symbiomix Therapeutics, LLC is a biopharmaceutical company focused on developing innovative treatments for serious women's health infections, particularly those that have been historically overlooked. Founded in 2012 and headquartered in Newark, New Jersey, with additional offices in Baltimore, Maryland, and Hamden, Connecticut, the company specializes in antibiotics for gynecologic infections. Its lead product, Solosec (secnidazole), is a next-generation 5-nitroimidazole antibiotic designed for the one-time oral treatment of bacterial vaginosis. This drug is characterized by its enhanced pharmacokinetic properties, allowing for effective and well-tolerated delivery in a single dose. Symbiomix Therapeutics operates as a subsidiary of Lupin Inc.

Dimension Therapeutics, Inc. is a gene therapy company based in Cambridge, Massachusetts, specializing in the development of innovative treatments for rare and metabolic diseases associated with the liver. Founded in 2013, the company has a robust pipeline of gene therapy programs, including DTX101 and DTX201 for hemophilia B and A, respectively. Additionally, its portfolio encompasses DTX301 for ornithine transcarbamylase deficiency, DTX401 for glycogen storage disease type Ia, DTX501 for phenylketonuria, DTX701 for Wilson disease, and DTX601 for citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, gaining access to a significant portfolio of patents related to adeno-associated virus (AAV) therapeutics. This collaboration supports the company's aim to advance its gene therapy platform and enhance its product development capabilities in the treatment of rare diseases. Dimension Therapeutics operates as a subsidiary of Ultragenyx Pharmaceutical Inc. since November 2017.

GENEWIZ is a global genomics service company offering DNA sequencing, gene synthesis, molecular biology, next-generation sequencing, bioinformatics, and GLP regulatory services. Established in 1999, it serves pharmaceutical, biotechnology, academic, agricultural, government, and clinical institutions worldwide.

Pieris Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on the development of anticalin-based therapeutics for treating various medical conditions, including cancer and severe asthma. Anticalins are engineered proteins derived from lipocalins, naturally occurring human proteins that can bind and transport a variety of molecules. The company's lead programs include PRS-060, a drug candidate in Phase I clinical trials targeting IL-4Ra for asthma and inflammatory diseases, and PRS-343, a bispecific protein in Phase I trials for oncology. Pieris is also advancing PRS-344, a bispecific anticalin-antibody fusion protein in preclinical development, and PRS-080, currently in Phase IIa trials for targeting hepcidin in patients with functional iron deficiency. Pieris collaborates with strategic partners and research institutions to enhance its development efforts, showcasing a robust pipeline aimed at addressing significant unmet medical needs. The company was founded in 2000 and is recognized for its innovative approach in biotherapeutics.

PMV Pharmaceuticals, Inc. is a precision oncology company focused on the discovery and development of small molecule therapies targeting p53 mutations in cancer. Its lead candidate, PC14586, aims to correct and restore the function of the p53 protein, which is critical in regulating cell growth and preventing tumor formation. In addition to PC14586, the company is developing therapies for various p53 hotspot mutations, including the R273H mutation. Founded in 2013 and headquartered in Cranbury, New Jersey, PMV Pharmaceuticals leverages extensive expertise in p53 biology to advance its innovative treatment options for cancer patients.

Selecta Biosciences, Inc. is a clinical-stage biopharmaceutical company focused on developing nanoparticle immunomodulatory drugs for the treatment and prevention of human diseases. The company's proprietary platform utilizes advances in immunobiology and nanotechnology to create targeted immunomodulatory nanoparticles. Selecta's lead product, SEL-212, has successfully completed Phase II clinical trials for chronic refractory gout. Additionally, the company is advancing gene therapy candidates in preclinical development for rare metabolic disorders, including SEL-302 for methylmalonic acidemia and SEL-313 for ornithine transcarbamylase deficiency. Founded in 2007 and headquartered in Watertown, Massachusetts, Selecta Biosciences has established collaborations with various organizations, including Spark Therapeutics and the Massachusetts Institute of Technology, to enhance its research and development capabilities.

Dimension Therapeutics, Inc. is a gene therapy company based in Cambridge, Massachusetts, specializing in the development of innovative treatments for rare and metabolic diseases associated with the liver. Founded in 2013, the company has a robust pipeline of gene therapy programs, including DTX101 and DTX201 for hemophilia B and A, respectively. Additionally, its portfolio encompasses DTX301 for ornithine transcarbamylase deficiency, DTX401 for glycogen storage disease type Ia, DTX501 for phenylketonuria, DTX701 for Wilson disease, and DTX601 for citrullinemia type I. Dimension Therapeutics collaborates with REGENX Biosciences, gaining access to a significant portfolio of patents related to adeno-associated virus (AAV) therapeutics. This collaboration supports the company's aim to advance its gene therapy platform and enhance its product development capabilities in the treatment of rare diseases. Dimension Therapeutics operates as a subsidiary of Ultragenyx Pharmaceutical Inc. since November 2017.

Loxo Oncology, Inc. is a biopharmaceutical company based in Stamford, Connecticut, focused on developing and commercializing targeted therapies for genetically defined cancers in the United States. Founded in 2013, the company aims to bring innovative cancer treatments to the clinic by identifying actionable "driver mutations" in tumors. Its lead product candidate, larotrectinib, is an oral selective inhibitor of tropomyosin receptor kinase (TRK) and is undergoing various clinical trials for multiple cancer types, including lung, melanoma, and breast cancer. Loxo Oncology is also advancing several preclinical programs, including candidates targeting RET fusion proteins and FGFR isoforms. The company has established collaborations with several organizations to enhance its drug discovery efforts and optimize its clinical development strategies. As a subsidiary of Eli Lilly and Company, Loxo Oncology is dedicated to creating effective therapies that address specific genetic vulnerabilities in cancer patients.

TytoCare develops handheld devices and software that enable users to perform self-examinations, facilitating remote diagnosis by healthcare professionals. The company's tools allow examination of various body areas and diagnose common conditions. TytoCare offers solutions for consumers (TytoHome), professionals (TytoPro), and remote clinics (TytoClinic). Incorporated in 2011, the company is headquartered in Netanya, Israel with an additional office in New York.

SAGE Therapeutics, Inc. is a clinical-stage biopharmaceutical company headquartered in Cambridge, Massachusetts, dedicated to developing innovative treatments for central nervous system (CNS) disorders. The company's lead product, ZULRESSO, is an intravenous formulation of brexanolone specifically designed for the treatment of postpartum depression. SAGE Therapeutics is advancing a diverse product pipeline, including SAGE-217, a novel neuroactive steroid in Phase III trials for various depressive disorders and anxiety, and SAGE-324, currently in Phase II trials for essential tremors and other neurological conditions. Additionally, SAGE-718, an oxysterol-based positive allosteric modulator, has completed Phase I trials targeting multiple CNS-related disorders. The company also explores other compounds, such as SAGE-904 and SAGE-689. SAGE Therapeutics has established strategic collaborations with Shionogi & Co., Ltd. and Biogen Inc. for the development and commercialization of its key product candidates, emphasizing its commitment to addressing the challenges of brain health.

Loxo Oncology, Inc. is a biopharmaceutical company based in Stamford, Connecticut, focused on developing and commercializing targeted therapies for genetically defined cancers in the United States. Founded in 2013, the company aims to bring innovative cancer treatments to the clinic by identifying actionable "driver mutations" in tumors. Its lead product candidate, larotrectinib, is an oral selective inhibitor of tropomyosin receptor kinase (TRK) and is undergoing various clinical trials for multiple cancer types, including lung, melanoma, and breast cancer. Loxo Oncology is also advancing several preclinical programs, including candidates targeting RET fusion proteins and FGFR isoforms. The company has established collaborations with several organizations to enhance its drug discovery efforts and optimize its clinical development strategies. As a subsidiary of Eli Lilly and Company, Loxo Oncology is dedicated to creating effective therapies that address specific genetic vulnerabilities in cancer patients.