Adage Capital Management

MedinCell S.A. is a pharmaceutical company focused on developing innovative therapeutic solutions through its proprietary BEPO technology, which facilitates long-acting injectable products utilizing biodegradable polymers and a biocompatible solvent. Based in Jacou, France, the company is engaged in ongoing research and development to optimize the efficiency and accessibility of medical treatments across various therapeutic areas. MedinCell's product pipeline includes several candidates, such as mdc-IRM, a subcutaneous injection for schizophrenia currently in Phase III trials, and mdc-CWM, an intra-articular injection aimed at treating pain and inflammation. Additionally, the company is exploring formulations like mdc-WWM for contraception and mdc-ANG for central nervous system disorders, alongside other candidates targeting pain management and organ transplant applications. Through its advancements in controlled, long-acting injectables, MedinCell aims to enhance treatment outcomes for patients.

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.

Monopar Therapeutics Inc. is a clinical-stage biopharmaceutical company based in Wilmette, Illinois, focused on developing proprietary therapeutics to improve outcomes for cancer patients. The company is advancing several key products, including Validive, a clonidine mucobuccal tablet in Phase III trials aimed at preventing severe oral mucositis caused by chemoradiotherapy in oropharyngeal cancer patients. Another significant compound is camsirubicin, a targeted analog of doxorubicin designed to treat soft tissue sarcoma while minimizing cardiac toxicity. Additionally, Monopar is developing MNPR-101, a humanized monoclonal antibody targeting advanced solid tumors, and MNPR-202, an early-stage analog for various cancers. Monopar collaborates with organizations such as Grupo Español de Investigación en Sarcomas and NorthStar Medical Radioisotopes, focusing on treatments for both cancer and severe COVID-19. Founded in 2014, the company emphasizes the development of orphan oncology compounds to extend life and enhance the quality of life for patients.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, established in 2011. The company specializes in the development and commercialization of Haduvio, an oral extended-release formulation of nalbuphine. Haduvio is currently undergoing a Phase IIb/III clinical trial, known as the PRISM trial, aimed at treating chronic pruritus associated with prurigo nodularis. Additionally, Trevi is exploring the use of nalbuphine ER for other serious neurologically mediated conditions, including chronic cough in patients with idiopathic pulmonary fibrosis and levodopa-induced dyskinesia in individuals with Parkinson’s disease. The treatment targets conditions that share a common pathophysiology mediated through opioid receptors in the central and peripheral nervous systems.

Olema Oncology is a preclinical biotechnology company dedicated to developing innovative therapies for the treatment and prevention of estrogen receptor-positive breast cancer. The company specializes in discovering and commercializing targeted treatments specifically designed for women's cancers. By leveraging its extensive understanding of endocrine-driven cancers, nuclear receptors, and mechanisms of acquired resistance, Olema aims to create compounds that surpass existing therapies. The company's focus is on transforming the standard of care for both pre- and post-menopausal women with cancer, with an emphasis on developing more effective and convenient treatment options. Olema's pipeline includes drug candidates such as OP-1250 and OP-3136, which have progressed through discovery and preclinical studies.

Cidara Therapeutics is a biotechnology company dedicated to developing novel anti-infectives. Its primary focus is rezafungin acetate, an antifungal for treating serious invasive fungal infections. Additionally, it advances the Cloudbreak platform to develop antiviral therapies using Fc-conjugates.

Aclaris Therapeutics is a United States-based clinical-stage biopharmaceutical company focused on developing novel therapies for immuno-inflammatory diseases and dermatology, aiming to address unmet medical and aesthetic needs where treatment options are limited. The company operates two segments, therapeutics and contract research, with the therapeutics business pursuing innovative treatments for immuno-inflammatory conditions while the contract research segment provides laboratory services. Its pipeline includes Zunsemetinib, an oral MK2 inhibitor, and ATI-2138, among other drug candidates.

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.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing and delivering gene therapy-based treatments for severe and life-threatening rare genetic diseases. Its pipeline includes therapies for Sanfilippo syndrome types A and B, epidermolysis bullosa, Batten disease, cystic fibrosis, and genetic eye disorders.

Benitec Ltd is a biotechnology company that specializes in developing innovative genetic medicines using its proprietary technology platform, which combines RNA interference with gene therapy. The company focuses on creating therapeutics for chronic and life-threatening diseases, aiming to provide long-lasting gene silencing through a single administration. Among its key product candidates is BB-301, an adeno-associated virus-based gene therapy designed to treat oculopharyngeal muscular dystrophy and chronic hepatitis B virus infection. Benitec's technology, known as ddRNAi, selectively targets and silences specific disease-related genes, offering potential cures for conditions that currently require ongoing treatment or have limited therapeutic options. The company holds over 40 global patents protecting its technology and has an active pipeline of drug development programs addressing various diseases, including hepatitis and cancer-related conditions. Established in 1995, Benitec is headquartered in Hayward, California.

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.

Geron Corporation is a late-stage clinical biopharmaceutical company based in Menlo Park, California, specializing in the development and commercialization of therapeutics for hematologic malignancies. Established in 1990, the company is primarily focused on its investigational drug, imetelstat, a first-in-class telomerase inhibitor currently undergoing Phase 2/3 clinical trials. Imetelstat targets the uncontrolled proliferation of malignant progenitor cells, aiming to improve blood cell production in patients with conditions such as myelodysplastic syndromes and myelofibrosis. Geron generates revenue through collaboration agreements, milestones, royalties, and licensing arrangements, positioning itself as a key player in oncology therapeutics.

Fate Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing programmed cellular immunotherapies for cancer and immune disorders. The company specializes in NK- and T-cell immuno-oncology programs, with a diverse pipeline that includes candidates such as FT516 and FT596 for treating various hematologic malignancies, FT538 for acute myeloid leukemia and multiple myeloma, and FT500 for advanced solid tumors. Fate Therapeutics employs advanced techniques involving induced pluripotent stem cells (iPSCs) to create engineered product candidates, which are designed to be off-the-shelf solutions. The company has established strategic collaborations with notable partners, including Ono Pharmaceutical Co. Ltd. and Janssen Biotech, to enhance its research and development capabilities. Founded in 2007, Fate Therapeutics aims to leverage cutting-edge science to create innovative therapies that restore health by modulating the properties of stem cells.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies to transform the treatment of inflammatory bowel disease (IBD).

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in the discovery and development of microRNA-targeted therapies for various diseases. Founded in 2007 and headquartered in San Diego, California, the company focuses on innovative treatments for conditions such as kidney diseases and viral infections. Its lead product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, and RGLS4326, an anti-miR targeting miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. Additionally, Regulus is advancing a pipeline of preclinical drug candidates, including RGLS5579 to inhibit miR-10b, as well as programs aimed at treating Hepatitis B virus and Non-Alcoholic Steatohepatitis. The company's work leverages the recent scientific advancements in microRNA research, which play a crucial role in regulating gene expression.

Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.

Crinetics Pharmaceuticals is a clinical-stage company focused on discovering, developing, and commercializing therapeutics targeting peptide hormone receptors for treating rare endocrine diseases and related cancers. Its lead product candidate, paltusotine, is an oral selective nonpeptide somatostatin receptor type 2 biased agonist in Phase I trials for acromegaly and neuroendocrine tumors.

Applied Therapeutics is a biopharmaceutical company focused on developing transformative drugs targeting fatal and debilitating diseases with high unmet medical needs. Its pipeline includes novel product candidates such as AT-007 for rare metabolic diseases like galactosemia, AT-001 for diabetic cardiomyopathy, and AT-003 for diabetic retinopathy.

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).

PepGen Ltd. is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing the next generation of nucleic acid therapeutics, particularly in the treatment of severe neuromuscular and neurologic diseases. Incorporated in 2018, PepGen has developed its Enhanced Delivery Oligonucleotide (EDO) platform, which is designed to improve the uptake and activity of conjugated oligonucleotide therapeutics. The company's innovative EDO peptides enhance tissue penetration, cellular uptake, and nuclear delivery, demonstrating their ability to transport oligonucleotides effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. PepGen aims to unlock the clinical potential of these transformative therapeutics, with a particular emphasis on the effective delivery of antisense oligonucleotides, and is advancing its novel conjugate therapeutics toward clinical application.

Astria Therapeutics is a biopharmaceutical company focused on developing therapies for rare and niche allergic and immunological diseases. It is advancing monoclonal antibody programs such as STAR-0215, a plasma kallikrein inhibitor being developed for hereditary angioedema, and STAR-0310, an OX40 antagonist for atopic dermatitis, both in preclinical development to address immune-mediated inflammatory conditions.

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.

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.

Syros Pharmaceuticals, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead product candidates, including SY-1425, a selective retinoic acid receptor alpha agonist currently in Phase II clinical trials for specific patient populations with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor under investigation in a Phase I clinical trial for advanced solid tumors. Syros is also engaged in collaborative research, including a partnership with Incyte Corporation aimed at identifying therapeutic targets for myeloproliferative neoplasms and a licensing agreement with TMRC Co. Ltd. for the development of tamibarotene. With a focus on mapping gene regulatory circuits, Syros leverages its proprietary platform and expertise to address significant unmet medical needs in oncology. Founded in 2011, the company aims to establish new standards of care for patients with hematologic malignancies.

Soleno Therapeutics is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for rare diseases. Its lead candidate, Diazoxide Choline Controlled Release (DCCR), an extended-release formulation of diazoxide, is being evaluated in Phase III trials for the treatment of Prader-Willi Syndrome.

AN2 Therapeutics is a biopharmaceutical company dedicated to researching, developing, and commercializing innovative medicines targeting infectious diseases. Established in 2017, the company focuses on advancing a clinical-stage antibacterial compound and has a strategic partnership with Brii Biosciences.

Benitec Ltd is a biotechnology company that specializes in developing innovative genetic medicines using its proprietary technology platform, which combines RNA interference with gene therapy. The company focuses on creating therapeutics for chronic and life-threatening diseases, aiming to provide long-lasting gene silencing through a single administration. Among its key product candidates is BB-301, an adeno-associated virus-based gene therapy designed to treat oculopharyngeal muscular dystrophy and chronic hepatitis B virus infection. Benitec's technology, known as ddRNAi, selectively targets and silences specific disease-related genes, offering potential cures for conditions that currently require ongoing treatment or have limited therapeutic options. The company holds over 40 global patents protecting its technology and has an active pipeline of drug development programs addressing various diseases, including hepatitis and cancer-related conditions. Established in 1995, Benitec is headquartered in Hayward, California.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

Karyopharm Therapeutics Inc. is a clinical-stage pharmaceutical company based in Newton, Massachusetts, focused on the discovery and development of innovative drug therapies for cancer and other significant diseases. The company specializes in small molecule selective inhibitors of nuclear export (SINE) compounds, targeting the nuclear export protein exportin 1. Its lead product, XPOVIO, is approved in the U.S. for treating multiple myeloma in various settings. Karyopharm is advancing several clinical trials, including BOSTON and STORM for multiple myeloma, SADAL for diffuse large B-cell lymphoma, and SEAL for liposarcoma. Additionally, it is exploring the treatment of endometrial cancer through the SIENDO trial and glioblastoma multiforme in the KING trial. The company maintains collaborations to facilitate clinical trials and research efforts, including projects related to COVID-19. Karyopharm continues to develop various investigational programs aimed at expanding its therapeutic portfolio.

BioAtla, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing innovative antibody-based therapeutics for cancer treatment. The company utilizes its proprietary platforms, Conditionally Active Biologics (CAB) and Comprehensive Integrated Antibody Optimization (CIAO!), to create highly selective and effective drugs. Its lead product candidate, BA3011, is a conditionally active biologic antibody-drug conjugate targeting soft tissue and bone sarcomas, non-small cell lung cancer, and other tumor types. Additionally, BioAtla is developing BA3021, aimed at non-small cell lung cancer and melanoma, as well as BA3071, which targets multiple cancers, including renal cell carcinoma and hepatocellular carcinoma. The company holds over 150 issued patents and applications, emphasizing its commitment to improving drug selectivity and safety while expanding the range of treatable cancers.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing and delivering gene therapy-based treatments for severe and life-threatening rare genetic diseases. Its pipeline includes therapies for Sanfilippo syndrome types A and B, epidermolysis bullosa, Batten disease, cystic fibrosis, and genetic eye disorders.

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.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with classical complement-mediated disorders affecting the body, brain, and eye. The company's pipeline is built on a platform technology that targets well-researched autoimmune and neurodegenerative disease processes triggered by the aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody designed for intravenous administration to treat autoimmune and neurodegenerative disorders. Another key candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration to address neurodegenerative ophthalmic disorders. The company employs a biomarker-driven development strategy to ensure that its product candidates effectively engage the target at therapeutic doses within the intended patient tissue, while also exploring additional orphan and large market indications.

Founded in 2014, Beckley Psytech is a UK-based company dedicated to developing psychedelic medicines for treating mental health disorders. It focuses on rigorous scientific research and clinical-stage drug development, aiming to provide innovative treatments for conditions like depression and anxiety.

ImaginAb, Inc. is a clinical stage biotechnology company specializing in immune oncology imaging and radiopharmaceutical therapy. Founded in 2007 and based in Inglewood, California, the company develops engineered antibody fragments, specifically minibodies, to provide diagnostic imaging solutions and insights into cancer immunotherapy. Its product line includes advanced imaging agents such as the CD8 ImmunoPET™ and PSMA PET, which target specific molecular markers in various cancers, including prostate and immune-oncology. These imaging agents are designed to enhance patient selection and monitor treatment progress, facilitating precision medicine. Currently, ImaginAb's lead candidate, 89Zr crefmirlimab berdoxam, is undergoing Phase II clinical trials and has been licensed by multiple pharmaceutical and biotech companies for use in their immunotherapy clinical trials.

Larimar Therapeutics is a clinical-stage biotechnology company dedicated to developing treatments for complex rare diseases. Its lead compound, CTI-1601, is currently in Phase 1 clinical trials as a potential treatment for Friedreich's ataxia, a rare and progressive genetic disease.

Arch Oncology, Inc. is an immuno-oncology company focused on discovering and developing antibody therapeutics for the treatment of solid tumors and hematologic cancers. Founded in 2006 and headquartered in Brisbane, California, with a scientific research laboratory in St. Louis, Missouri, the company is known for its innovative approach to cancer treatment. Arch Oncology specializes in anti-CD47 antibodies, which represent a new class of checkpoint inhibitors that engage both the adaptive and innate immune responses. These functionally diverse antibodies not only inhibit immune checkpoints but also selectively target and kill tumor cells, providing patients with new therapeutic options in the fight against cancer.

Silence Therapeutics plc is a biotechnology company based in London, specializing in the discovery and development of innovative RNA therapeutics. The company focuses on utilizing short interfering RNA (siRNA) technology to modulate gene expression and address various medical conditions, including hematology, cardiovascular diseases, and rare metabolic disorders. Its pipeline includes several product candidates, such as SLN124, aimed at treating iron overload disorders by silencing the TMPRSS6 gene, and SLN360, which targets the LPA gene to reduce the risk of cardiovascular diseases. Silence Therapeutics has established strategic collaborations with major organizations, including AstraZeneca, to advance the development of siRNA therapeutics across multiple disease areas. The company leverages its proprietary genetic toolkit and delivery systems to optimize therapeutic outcomes, harnessing the body's natural biological mechanisms for effective treatment.

Founded in 2008 and headquartered in Charlottesville, Virginia, HemoShear Therapeutics develops treatments for rare metabolic disorders, including organic acidemias and nonalcoholic steatohepatitis. The company employs its proprietary REVEAL‑Tx platform to create human‑relevant systems that replicate organ biology and disease states, providing detailed insights into disease mechanisms. By generating meaningful human response data, HemoShear aims to reduce the risk of costly failures and accelerate the discovery of effective therapies. It collaborates with pharmaceutical, biotechnology, and medical device partners to advance drug development programs across liver diseases, gout, and other metabolic conditions.

Flame Biosciences is a clinical-stage biotechnology company based in New York, founded in 2017. The company specializes in developing innovative therapies for cancer and other inflammatory conditions. Its portfolio includes FL-101 therapy, designed to treat lung cancers driven by inflammation, and FL-201, aimed at reducing chronic inflammation in patients. Flame Biosciences is dedicated to improving the lives of individuals suffering from debilitating and life-threatening diseases through its transformative therapeutic approaches.

Eledon Pharmaceuticals is a clinical-stage biotechnology company focused on developing treatments for patients undergoing organ or cellular transplantation and those with autoimmune and neurodegenerative diseases. Its lead compound, tegoprubart, is an anti-CD40L antibody targeting the CD40L pathway.

Poseida Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapeutics for patients with significant unmet medical needs. The company specializes in genome engineering technologies and is focused on creating targeted treatments, including a pipeline of autologous and allogeneic chimeric antigen receptor T cell (CAR-T) therapies aimed at addressing hematological malignancies and solid tumors. Additionally, Poseida is advancing gene therapies for rare and life-threatening diseases, such as Ornithine transcarbamylase deficiency and methylmalonic acidemia, along with therapies for genetic liver diseases. Founded in 2014, Poseida Therapeutics is committed to improving patient outcomes through its cutting-edge research and development efforts.

Amplyx Pharmaceuticals, Inc. is a small molecule drug development company focused on creating innovative therapies for debilitating and life-threatening diseases, particularly those affecting individuals with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in the development of oral and injectable anti-infective drugs, including antifungal agents designed to treat invasive fungal infections caused by pathogens such as Candida and Aspergillus. Amplyx employs a unique platform approach that enhances the efficacy and reduces the toxicity of existing drugs by incorporating a second small molecule. This strategy aims to provide effective treatment options for patients undergoing chemotherapy and other immunosuppressive therapies, ensuring they receive appropriate care both during hospitalization and after discharge.

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.

Cabaletta Bio is a clinical-stage biotechnology company focused on developing engineered T cell therapies for patients with B cell-mediated autoimmune diseases. Its proprietary technology, chimeric autoantibody receptor (CAAR) T cells, selectively targets and eliminates disease-causing B cells while sparing healthy ones. Cabaletta's lead product candidate, DSG3-CAART, is in Phase I clinical trials for treating mucosal pemphigus vulgaris.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with classical complement-mediated disorders affecting the body, brain, and eye. The company's pipeline is built on a platform technology that targets well-researched autoimmune and neurodegenerative disease processes triggered by the aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody designed for intravenous administration to treat autoimmune and neurodegenerative disorders. Another key candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration to address neurodegenerative ophthalmic disorders. The company employs a biomarker-driven development strategy to ensure that its product candidates effectively engage the target at therapeutic doses within the intended patient tissue, while also exploring additional orphan and large market indications.

Cabaletta Bio is a clinical-stage biotechnology company focused on developing engineered T cell therapies for patients with B cell-mediated autoimmune diseases. Its proprietary technology, chimeric autoantibody receptor (CAAR) T cells, selectively targets and eliminates disease-causing B cells while sparing healthy ones. Cabaletta's lead product candidate, DSG3-CAART, is in Phase I clinical trials for treating mucosal pemphigus vulgaris.

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.

Engage Therapeutics, Inc. is a biopharmaceutical company based in Summit, New Jersey, focused on developing innovative therapies for individuals experiencing uncontrolled epileptic seizures. Its lead investigational product, Staccato alprazolam, is a small, handheld drug-device combination designed for the rapid cessation of active and acute seizures. Utilizing an FDA-approved delivery system, the Staccato device administers alprazolam, an established benzodiazepine, in a single inhalation. The product has shown promise in a Phase 2a proof of concept study, which indicated potential reductions in seizures within a photosensitivity model. Engage Therapeutics is advancing Staccato alprazolam through clinical development, employing a 505(b)(2) regulatory pathway to facilitate its market entry.

ImaginAb, Inc. is a clinical stage biotechnology company specializing in immune oncology imaging and radiopharmaceutical therapy. Founded in 2007 and based in Inglewood, California, the company develops engineered antibody fragments, specifically minibodies, to provide diagnostic imaging solutions and insights into cancer immunotherapy. Its product line includes advanced imaging agents such as the CD8 ImmunoPET™ and PSMA PET, which target specific molecular markers in various cancers, including prostate and immune-oncology. These imaging agents are designed to enhance patient selection and monitor treatment progress, facilitating precision medicine. Currently, ImaginAb's lead candidate, 89Zr crefmirlimab berdoxam, is undergoing Phase II clinical trials and has been licensed by multiple pharmaceutical and biotech companies for use in their immunotherapy clinical trials.

WellDoc, Inc. is a healthcare technology company focused on chronic disease management, particularly diabetes. Founded in 2005 and headquartered in Baltimore, Maryland, with additional offices in Wilmington, Delaware, and Bengaluru, India, WellDoc develops innovative solutions that integrate into existing healthcare workflows. Its flagship platform, DiabetesManager, provides personalized, real-time coaching and support for patients and healthcare providers, facilitating self-management and adherence to treatment regimens. The platform features tools for medication reminders, alerts for out-of-range metrics, and cloud-based analytics for tracking health outcomes. Additionally, WellDoc offers BlueStar, a mobile prescription therapy solution that guides patients in managing their diabetes. The company serves various stakeholders in the healthcare ecosystem, including payors, employers, pharmaceutical companies, and medical device firms, and has established strategic partnerships to enhance its offerings.

Protagonist Therapeutics, Inc., established in 2006 and headquartered in Newark, California, is a clinical-stage biopharmaceutical company focused on developing peptide-based therapies to address unmet medical needs in hematology and gastroenterology. The company's pipeline includes PTG-300, an injectable hepcidin mimetic in Phase II trials for beta-thalassemia; PTG-200, a completed Phase I antagonist peptide candidate for moderate-to-severe Crohn’s disease; and PN-943, an oral integrin antagonist in Phase I trials for inflammatory bowel disease. Protagonist has a licensing and collaboration agreement with Janssen Biotech for PTG-200's development and commercialization. The company's proprietary platform enables the discovery of novel constrained peptide-based drug candidates targeting protein-protein interactions.

Advanced Accelerator Applications develops and commercializes molecular nuclear medicine products for diagnosis and therapy. Its portfolio includes radiopharmaceuticals like oxodotreotides, dotatate injections, and edotreotide solutions, targeting oncology, cardiology, and neurology. The company also offers theragnostic platforms for precision imaging and targeted therapies.

Aimmune Therapeutics is a biopharmaceutical company focused on developing and commercializing treatments for peanut and other food allergies. The company’s primary product candidate, PALFORZIA, utilizes a therapeutic approach known as Characterized Oral Desensitization Immunotherapy (CODIT) to desensitize patients to food allergens. Aimmune is also engaged in research and development of additional CODIT candidates, including AR201, which is in Phase II clinical trials for treating egg allergy in pediatric and young adult populations, as well as other candidates targeting allergies like cow's milk. The company collaborates strategically with Nestlé Health Science to advance food allergy therapeutics and has clinical agreements with Regeneron and Sanofi to study PALFORZIA in conjunction with dupilumab for peanut-allergic patients. Founded in 2011 and headquartered in Brisbane, California, Aimmune Therapeutics was previously known as Allergen Research Corporation before rebranding in 2015 and is now a subsidiary of Société des Produits Nestlé S.A.

Advaxis, Inc. is a clinical-stage biotechnology company based in Princeton, New Jersey, specializing in the development of immunotherapies for cancer and infectious diseases. The company utilizes a unique platform technology involving live, attenuated bacteria engineered to deliver antigen/adjuvant fusion proteins, effectively redirecting immune responses to target cancer cells. Advaxis is advancing several product candidates, including axalimogene filolisbac for HPV-related cancers, currently undergoing Phase II clinical trials for head and neck cancer. Additionally, it is developing ADXS-PSA for prostate cancer and ADXS-NEO, which personalizes treatment by using whole-exome sequencing to identify tumor neoantigens. Other projects include ADXS-HOT, aimed at generating robust anti-cancer immunity, and ADXS-HER2, designed to target HER2-expressing solid tumors. The company has established collaborations and licensing agreements with various organizations, enhancing its research and development efforts.

Adverum Biotechnologies is a clinical-stage company focused on developing gene therapies to treat ocular diseases. Its pipeline includes ADVM-022 for wet age-related macular degeneration, ADVM-043 for alpha-1 antitrypsin deficiency, and preclinical candidates for other indications.

Nexvet Australia Pty Ltd is a biopharmaceutical company based in Melbourne, Australia, specializing in the development of biological therapies for companion animals. Founded in 2010, Nexvet focuses on creating species-specific monoclonal antibodies to address chronic pain, immune oncology, chronic inflammation, and allergies in pets. The company's key products include Ranevetmab and Frunevetmab, which target nerve growth factor to alleviate pain associated with osteoarthritis in dogs and degenerative joint disease in cats, respectively. Additionally, Nexvet is developing NV-03, a monoclonal antibody for chronic joint pain in horses. Utilizing a unique technology called PETisation, Nexvet customizes therapies to specific species, enhancing the effectiveness and safety of treatments while minimizing immune responses. As a subsidiary of Zoetis Inc., Nexvet aims to transform animal medicine by collaborating with global partners in veterinary and human health to advance its innovative pipeline.

Lumena Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing oral therapeutics for rare liver diseases, particularly cholestatic liver disease. Its lead candidate, LUM001, targets a specific transporter in the intestine, which reduces the risk of systemic toxicities by being minimally absorbed in the body. LUM001 has been extensively studied across 12 clinical trials involving over 1,400 subjects, positioning it for rapid advancement in both pediatric and adult patients. While Lumena's primary objective is to provide novel treatments for rare liver diseases, its therapeutic approach also holds potential for addressing metabolic diseases affecting the liver, which are increasingly recognized as significant health concerns in both children and adults.

ZS Pharma, Inc. is a biopharmaceutical company based in Coppell, Texas, specializing in the development and commercialization of non-absorbed drugs aimed at treating renal, cardiovascular, liver, and metabolic diseases. Founded in 2008, the company focuses on creating highly selective ion-trap therapies using its proprietary zirconium silicate technology, which allows for the reduction of toxic electrolyte levels while maintaining overall electrolyte balance. Its lead product, ZS-9, is an insoluble, non-absorbed zirconium silicate currently in Phase III clinical trials for the treatment of hyperkalemia, a serious condition characterized by elevated potassium levels in the blood, often affecting patients with chronic kidney disease, hypertension, diabetes, or heart failure. As of December 2015, ZS Pharma operates as a subsidiary of Zeneca, Inc.

Calithera Biosciences, Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecule drugs targeting tumor metabolism and immunology for cancer treatment. Headquartered in South San Francisco, California, the company’s lead product candidate, CB-839, is an inhibitor of glutaminase currently in Phase II clinical trials for solid tumors. Additionally, Calithera is developing INCB001158, an oral arginase inhibitor in Phase I/II trials for hematology and oncology, as well as CB-280, another oral arginase inhibitor aimed at treating cystic fibrosis, and CB-708, an orally administered small molecule inhibitor of CD73. The company has established collaborations with Bristol-Myers Squibb to evaluate the combination of nivolumab with CB-839, and with Incyte Corporation for the development and commercialization of INCB001158. Founded in 2010, Calithera Biosciences is dedicated to advancing innovative therapies that specifically target critical pathways in tumor growth and survival.

PTC Therapeutics is a biopharmaceutical company dedicated to discovering, developing, and commercializing treatments for rare genetic disorders. Its portfolio includes approved drugs like Translarna (ataluren) and Emflaza (deflazacort), as well as pipeline candidates targeting various rare diseases such as Duchenne muscular dystrophy, spinal muscular atrophy, and certain cancers.

Puma Biotechnology, Inc. is a biopharmaceutical company dedicated to the development and commercialization of innovative products aimed at improving cancer care. Founded in 2010 and headquartered in Los Angeles, California, the company primarily focuses on the treatment of various forms of cancer, particularly through its lead product, NERLYNX, an oral formulation of neratinib designed for HER2-positive breast cancer patients. Additionally, Puma is developing PB357, an irreversible tyrosine kinase inhibitor that targets the epidermal growth factor receptors HER1, HER2, and HER4. The company operates under a licensing agreement with Pfizer for the development and commercialization of its drug candidates, including oral and intravenous forms of neratinib, as well as PB357. With a commitment to enhancing treatment outcomes, Puma Biotechnology is focused on advancing its clinical programs and bringing effective therapies to market for cancer patients.