Sanofi Oncology

Granite Bio is a biotechnology company focused on developing novel monoclonal antibodies aimed at treating inflammatory, autoimmune, and fibrotic disorders. The company specializes in creating therapeutic antibodies that deplete pathogenic cells and selectively target key biological processes associated with these diseases. By concentrating on these advanced treatments, Granite Bio contributes to the broader fields of biotechnology, pharmaceuticals, and diagnostics, ultimately seeking to improve patient outcomes in complex medical conditions.

Innate Pharma S.A. is a biotechnology company based in Marseille, France, focused on discovering and developing therapeutic antibodies for oncology and inflammatory diseases. The company is engaged in creating first-in-class immunotherapy drug candidates, leveraging its proprietary platform to harness the innate immune system. Key products in its pipeline include Monalizumab, an immune checkpoint inhibitor in Phase II trials for advanced solid tumors, and Lacutamab, an anti-KIR3DL2 antibody also in Phase II for cutaneous T-cell lymphoma. Additionally, the pipeline features various candidates at different stages, including IPH5201 and IPH5301, which target immunosuppressive pathways. Innate Pharma has established licensing and collaboration agreements with major pharmaceutical companies, enhancing its research and development capabilities. Founded in 1999, the company operates internationally, contributing to the advancement of cancer treatment through innovative therapeutic approaches.

Glycomine, Inc. is a biotech company focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation, which can severely impact patients' health. Established in 2014 and located in San Carlos, California, Glycomine aims to address the significant unmet medical need for effective treatments in the realm of rare diseases, where approximately 95% lack any FDA-approved therapies. The company specializes in creating replacement therapies that utilize a combination of substrates, enzymes, and proteins, delivered through bio-nano materials that target specific cells within clinically relevant organs. By innovating in this area, Glycomine seeks to provide new therapeutic options for patients suffering from disorders of metabolism and protein misfolding.

Latigo Biotherapeutics is a clinical-stage biotechnology company that develops novel, non-opioid therapies for chronic pain. The company aims to target pain at its source to provide effective, rapid-acting pain relief without the risk of addiction. Their lead program targets Nav1.8, a validated human pain target, leveraging internal ion channel expertise.

Curevo is a clinical-stage biotechnology company developing next-generation, non-mRNA vaccines to address unmet medical needs. Its lead candidate, amezosvatein (also known as CRV-101), is an adjuvanted subunit vaccine designed to prevent shingles (herpes zoster) and chickenpox (varicella). The company focuses on improving vaccine efficacy, safety, and tolerability while advancing its pipeline through clinical trials.

AdvanCell is a clinical-stage radiopharmaceutical company focused on developing innovative cancer therapies. The company specializes in alpha-emitting isotopes, specifically 212Pb, which are attached to molecules that target tumors directly. This approach allows for the delivery of cytotoxic radiation at the molecular level, addressing significant challenges in targeted alpha therapy regarding supply reliability and scalability. By providing safe and effective treatments for various forms of cancer, AdvanCell aims to establish its therapies as a standard of care in oncology.

Normunity is a biotechnology company focused on developing precision immuno-oncology medicines, specifically a new class known as immune normalizers. These medicines aim to target novel mechanisms that enhance the body’s natural immune response against cancer. In collaboration with the lab of Dr. Lieping Chen at Yale School of Medicine, Normunity is researching newly-discovered mechanisms of immune disruption in cancer, utilizing proprietary discovery platforms to explore the intricate interactions between cancer and the immune system. The company is advancing a pipeline of immune normalizers, which includes initial drug programs designed to address the exclusion of T-cells from immune-sensitive tumors and other factors that impede normal immune function in cancer. Normunity operates from locations in Boston, Massachusetts, and West Haven, Connecticut.

Nuvig Therapeutics is focused on developing innovative therapies aimed at restoring immune homeostasis and rebalancing immune function, particularly following inflammation. The company is building a pipeline of novel immune therapeutics specifically designed for chronic inflammatory and autoimmune diseases. By leveraging natural mechanisms to modulate immune responses, Nuvig seeks to enhance treatment options available to healthcare providers and improve patient outcomes.

Resalis Therapeutics is a biopharmaceutical company focused on transforming the treatment of metabolic diseases, particularly obesity and fatty liver disease, by developing innovative therapies that target key regulatory pathways. The company's leading program, RES-010, utilizes its expertise in non-coding RNA drug modalities and lipid metabolism to create a treatment that aims for long-term weight loss and reduced liver fat accumulation. With a strong foundation of preclinical evidence, Resalis is progressing RES-010 into clinical trials, offering potential disease-modifying effects and a safe, convenient therapeutic option for patients suffering from various metabolic disorders.

AgomAb Therapeutics N.V., based in Gent, Belgium, specializes in the development of molecular therapies aimed at regenerating damaged tissues. The company focuses on creating agonistic monoclonal antibodies, known as agomAbs, which are designed to stimulate molecular and cellular repair mechanisms. These therapies have the potential to restore organ function in patients suffering from fibrotic, inflammatory, autoimmune, and degenerative diseases. AgomAb targets biologically validated pathways, including Transforming Growth Factor β and Hepatocyte Growth Factor, while employing specialized capabilities in organ-specific small molecules and high-affinity antibodies. The company boasts a diversified clinical pipeline addressing various fibrotic conditions and possesses comprehensive research and development expertise, alongside a strong track record in business development.

Vicore Pharma Holding is a clinical-stage pharmaceutical company that specializes in developing innovative treatments for severe lung disorders, particularly idiopathic pulmonary fibrosis (IPF). The company is advancing its lead drug candidate, C21, a small molecule angiotensin II type 2 receptor agonist, which is currently in phase 2a clinical development for IPF and has received orphan drug designation in both the EU and the US. In addition to C21, Vicore is exploring pre-clinical applications of the drug in other rare diseases associated with the AT2 receptor. The company is also developing Almee, a digital therapeutic that utilizes cognitive behavioral therapy to address the psychological challenges faced by patients with pulmonary fibrosis. Headquartered in Astra Zeneca’s Bioventurehub in Mölndal, Vicore Pharma aims to establish a comprehensive portfolio targeting respiratory diseases.

Nura Bio, Inc. is a clinical-stage biopharmaceutical company based in South San Francisco, California, focused on developing neuroprotective therapies aimed at treating neurological diseases. The company specializes in discovering and developing drugs that prevent axon loss following acute injury or chronic degenerative conditions. Its primary drug candidate, NB-4746, is designed to inhibit the SARM1 protein, which is implicated in axonal degeneration. This candidate has shown promise in preclinical studies for preventing nerve damage and is progressing toward phase 1b/2 clinical trials scheduled for 2025. Nura Bio has successfully raised $140 million in financing to support its drug development initiatives and enhance the immune response of the nervous system following neurological injuries.

MeiraGTx Holdings is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company focuses on addressing serious medical conditions, primarily targeting disorders related to the eye, salivary gland, and central nervous system. Its research and development efforts leverage a portfolio approach, which includes licensing, acquiring, and developing advanced technologies to support a diverse range of product candidates. Key pipeline products include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, with plans to expand its focus to develop additional gene therapy treatments for various serious diseases in the future.

NextPoint Therapeutics is a clinical-stage biotechnology company that develops therapeutics to treat cancer patients. The company offers immunotherapies for a validated checkpoint axis that is independent of PD-1/L1 and modulates both adaptive and innate immune systems to find and destroy cancer cells. It advances therapeutic approaches utilizing the unique upregulation of HHLA2 in cancer as an anchor for tumor-targeting therapeutic modalities.

Areteia Therapeutics is a biotechnology company focused on improving the management of eosinophilic asthma through an innovative oral drug treatment. The company, which emerged from Knopp, aims to provide patients with a novel therapeutic option that inhibits the maturation of eosinophils—cells that can cause airway damage in asthma patients. Its drug candidate has demonstrated promising results in a phase 2 clinical trial, which showed a reduction in blood eosinophil counts, indicating enhanced lung function. This approach seeks to empower patients by offering a potentially effective and accessible treatment for a severe form of asthma, thereby improving their quality of life and disease management.

Inhibrx, Inc. is a clinical-stage biotechnology company based in La Jolla, California, dedicated to developing a diverse pipeline of novel biologic therapeutic candidates. The company employs advanced protein engineering techniques, including its proprietary sdAb platform, to target complex disease biology, with a focus on oncology, orphan diseases, and infectious diseases. Inhibrx's lead candidates include INBRX-109, a multivalent agonist in Phase 1 trials for solid tumors; INBRX-105, an antagonist of PD-L1 also in Phase 1 trials; INBRX-101, an Fc-fusion protein for alpha-1 antitrypsin deficiency; and INBRX-103, a monoclonal antibody targeting CD47. Additionally, the company is developing preclinical programs such as INBRX-106, an agonist of OX40, and INBRX-111, an antibody targeting Pseudomonas aeruginosa. Inhibrx has established collaborations with organizations like Celgene and Bluebird Bio and has received funding from various granting agencies, including the NIH and CARB-X.

Graviton Bioscience is a clinical-stage drug discovery and development firm focused on creating and developing therapies for the treatment of CNS, autoimmune, fibrotic, and other significant disorders in which ROCK2 plays a critical role in pathology.

Eligo Bioscience S.A.S is a biotechnology company based in Paris, France, that specializes in the development of Eligobiotics, a new class of antimicrobials designed to target specific bacteria based on their genetic makeup. Founded in 2014, the company utilizes a combination of CRISPR/Cas technology and engineered phage capsids to create antibiotics capable of addressing microbiome-related diseases and combating resistant pathogens. These programmed antimicrobials aim to eradicate virulent bacteria within the human microbiome, facilitating the creation of advanced therapeutics for conditions caused by superbugs. Eligo Bioscience's innovative approach positions it at the forefront of microbiome precision therapies and next-generation antibiotic solutions.

T-Therapeutics is a biopharmaceutical company specializing in the development of engineered soluble biologics for oncological and immunological conditions. The company utilizes a proprietary T cell receptor (TCR) discovery platform, which is based on a highly humanized mouse model. This innovative approach allows T-Therapeutics to identify TCRs that target human antigens not obtainable from human samples. The TCRs bind specific pMHC targets on target cells, facilitating the recruitment of T cells to either attack cancer cells or modulate immune responses. By leveraging advanced techniques in mouse genome engineering, single cell genomics, machine learning, and structural biology, T-Therapeutics aims to enhance treatment options for patients suffering from chronic and infectious diseases while fostering a culture of creativity and collaboration within its operations.

MeiraGTx Holdings is a clinical-stage gene therapy company dedicated to developing and commercializing innovative gene therapy products aimed at transforming the lives of patients with acquired and inherited disorders. The company focuses on addressing serious medical conditions, primarily targeting disorders related to the eye, salivary gland, and central nervous system. Its research and development efforts leverage a portfolio approach, which includes licensing, acquiring, and developing advanced technologies to support a diverse range of product candidates. Key pipeline products include AAV-CNGB3, AAV-CNGA3, and AAV-RPGR, among others. MeiraGTx operates in the United States, the United Kingdom, and the European Union, with plans to expand its focus to develop additional gene therapy treatments for various serious diseases in the future.

Minervax is focused on developing a vaccine to prevent infections caused by Group B streptococcus (GBS), which can lead to serious health issues in newborns and adverse pregnancy outcomes. The company is researching a specific fusion protein that has demonstrated the ability to stimulate strong protective immunity against various clinically significant GBS strains. By utilizing this innovative approach, Minervax aims to provide medical professionals with an effective tool to protect newborns from potentially life-threatening bacterial infections associated with GBS.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Veralox Therapeutics specializes in the development of small molecule therapeutics aimed at addressing the underlying pathologies of thrombosis and type one diabetes. The company focuses on innovative treatment paradigms informed by a deep understanding of the molecular mechanisms involved in these diseases. By targeting conditions such as heparin-induced thrombocytopenia and thrombosis, Veralox Therapeutics aims to enhance patient outcomes and facilitate complete recovery from diabetes. Through its research and development efforts, the company seeks to provide effective and transformative therapies for individuals affected by these serious health conditions.

Provention Bio, Inc. is a clinical-stage biopharmaceutical company that focuses on the development and commercialization of therapeutics aimed at intercepting and preventing immune-mediated diseases. The company's product candidates include PRV-031, a monoclonal antibody currently in Phase III clinical trials for the interception of type 1 diabetes, and PRV-6527, an oral CSF-1R inhibitor in Phase IIa trials for Crohn's disease. Additionally, PRV-3279 has completed Phase 1b trials for lupus, while PRV-101 is being developed as a vaccine for coxsackie virus B infections, which may prevent the onset of type 1 diabetes. Provention Bio also has a licensing agreement with Amgen for PRV-015, a monoclonal antibody targeting celiac disease. Founded in 2016 and based in Oldwick, New Jersey, Provention Bio aims to address the high morbidity and costs associated with chronic autoimmune and inflammatory diseases through its innovative predictive and preventive therapeutic approaches.

QurAlis Corporation is a clinical-stage biotechnology company focused on discovering and developing therapeutics for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. Based in Cambridge, Massachusetts, the company utilizes proprietary platforms and biomarkers to create precision medicines targeting genetically validated disease-causing alterations. Its pipeline includes various treatments aimed at specific subtypes of ALS, such as therapies designed to restore dysfunctional cellular waste clearance, manage overactive neurons to prevent cell death, and eliminate toxic proteins. QurAlis aims to advance its antisense oligonucleotides and small molecule programs to address the most prevalent forms of ALS, ultimately working to halt disease progression and improve patient outcomes. The company was incorporated in 2016 and operates as a subsidiary of Q-State Biosciences, Inc.

Provention Bio, Inc. is a clinical-stage biopharmaceutical company that focuses on the development and commercialization of therapeutics aimed at intercepting and preventing immune-mediated diseases. The company's product candidates include PRV-031, a monoclonal antibody currently in Phase III clinical trials for the interception of type 1 diabetes, and PRV-6527, an oral CSF-1R inhibitor in Phase IIa trials for Crohn's disease. Additionally, PRV-3279 has completed Phase 1b trials for lupus, while PRV-101 is being developed as a vaccine for coxsackie virus B infections, which may prevent the onset of type 1 diabetes. Provention Bio also has a licensing agreement with Amgen for PRV-015, a monoclonal antibody targeting celiac disease. Founded in 2016 and based in Oldwick, New Jersey, Provention Bio aims to address the high morbidity and costs associated with chronic autoimmune and inflammatory diseases through its innovative predictive and preventive therapeutic approaches.

NextPoint Therapeutics is a clinical-stage biotechnology company that develops therapeutics to treat cancer patients. The company offers immunotherapies for a validated checkpoint axis that is independent of PD-1/L1 and modulates both adaptive and innate immune systems to find and destroy cancer cells. It advances therapeutic approaches utilizing the unique upregulation of HHLA2 in cancer as an anchor for tumor-targeting therapeutic modalities.

Minervax is focused on developing a vaccine to prevent infections caused by Group B streptococcus (GBS), which can lead to serious health issues in newborns and adverse pregnancy outcomes. The company is researching a specific fusion protein that has demonstrated the ability to stimulate strong protective immunity against various clinically significant GBS strains. By utilizing this innovative approach, Minervax aims to provide medical professionals with an effective tool to protect newborns from potentially life-threatening bacterial infections associated with GBS.

Escient Pharmaceuticals is a biotechnology company based in San Diego, California, that develops and manufactures drugs targeting G protein-coupled receptors (GPCRs) for the treatment of neuro-immuno-inflammatory and autoreactive diseases. Founded in 2017, the company specializes in harnessing the therapeutic potential of specific orphan GPCRs, particularly the Mas-Related G-Protein Receptors (Mrgprs), to address various neurosensory-inflammatory disorders. Escient's innovative approach aims to provide first-in-class therapies for conditions associated with mast cell-mediated disorders and cholestatic pruritus, thereby expanding treatment options for healthcare providers and patients facing these challenging diseases.

Normunity is a biotechnology company focused on developing precision immuno-oncology medicines, specifically a new class known as immune normalizers. These medicines aim to target novel mechanisms that enhance the body’s natural immune response against cancer. In collaboration with the lab of Dr. Lieping Chen at Yale School of Medicine, Normunity is researching newly-discovered mechanisms of immune disruption in cancer, utilizing proprietary discovery platforms to explore the intricate interactions between cancer and the immune system. The company is advancing a pipeline of immune normalizers, which includes initial drug programs designed to address the exclusion of T-cells from immune-sensitive tumors and other factors that impede normal immune function in cancer. Normunity operates from locations in Boston, Massachusetts, and West Haven, Connecticut.

Matchpoint Therapeutics is a biotechnology company focused on developing precision covalent medicines aimed at transforming the treatment of immune diseases and other serious conditions. Utilizing its proprietary Advanced Covalent Exploration (ACE) platform, the company combines advanced chemoproteomics, machine learning, and the evolution of covalent chemistry libraries to enhance drug discovery. This innovative approach allows for a deeper exploration of the proteome, enabling the identification and targeting of new binding sites on disease-causing proteins. Matchpoint is building an emerging pipeline of novel covalent medicines, with an initial emphasis on immunology.

Innovent Biologics, Inc. is a prominent biopharmaceutical company based in Suzhou, China, specializing in the discovery, development, manufacturing, and commercialization of monoclonal antibodies. The company focuses on therapeutic areas such as oncology, ophthalmology, immunology, and metabolic diseases. Its flagship product, Tyvyt (sintilimab), is an anti-PD-1 monoclonal antibody approved for treating various cancers, including Hodgkin's lymphoma and esophageal carcinoma. Innovent's pipeline includes several important candidates, such as IBI-301, a rituximab biosimilar for non-Hodgkin's lymphoma, and IBI-303, an adalimumab biosimilar for autoimmune diseases. The company is also developing innovative therapies for conditions like hypercholesterolemia and various cancers. With a robust portfolio of commercialized products and ongoing strategic collaborations with major pharmaceutical companies, Innovent aims to expand its impact in the global biopharmaceutical landscape. Founded in 2011, Innovent is recognized as one of China's leading biotechnology firms.

Areteia Therapeutics is a biotechnology company focused on improving the management of eosinophilic asthma through an innovative oral drug treatment. The company, which emerged from Knopp, aims to provide patients with a novel therapeutic option that inhibits the maturation of eosinophils—cells that can cause airway damage in asthma patients. Its drug candidate has demonstrated promising results in a phase 2 clinical trial, which showed a reduction in blood eosinophil counts, indicating enhanced lung function. This approach seeks to empower patients by offering a potentially effective and accessible treatment for a severe form of asthma, thereby improving their quality of life and disease management.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Nucleai Ltd. is a spatial biology company that has developed an AI-powered pathology platform focused on enhancing drug development and improving patient outcomes. Founded in 2017 and headquartered in Tel Aviv-Yafo, Israel, Nucleai's technology utilizes unique tissue datasets and machine learning algorithms to analyze biopsy samples and detect various cancers, including prostate, breast, and gastrointestinal diseases. The platform aims to improve the interpretation of pathology results, thereby increasing the efficacy of clinical trials and fostering better patient care. Nucleai collaborates with leading pharmaceutical companies, licensing its platform for internal use and participating in biomarker discovery projects. By transforming healthcare through artificial intelligence, Nucleai strives to enable researchers to identify novel biomarkers that predict therapeutic responses and enhance understanding of complex biological phenomena.

Amunix Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to discovering and developing innovative protein and peptide therapeutics aimed at treating cancer. The company employs several proprietary platforms, including XTEN for extending drug half-life, XPAT for analyzing protease activity in the tumor microenvironment, and ProTIA, which focuses on immuno-oncology therapeutics. Amunix's product pipeline includes prodrugs and transformative T-cell engagers designed to activate the immune system against solid tumors. The company collaborates with various academic, biotechnology, and pharmaceutical sectors, maintaining strategic partnerships with organizations such as Janssen and Biogen-Idec. Founded in 2006 and headquartered in Mountain View, California, Amunix strives to deliver breakthroughs that can harness the immune system to combat cancer more effectively.

Gyroscope is a clinical-stage gene therapy company that specializes in developing treatments for retinal diseases, particularly focusing on Age-related Macular Degeneration (AMD), a major cause of blindness. The company leverages advancements in understanding the complement system's role in eye diseases and uses gene therapy as a therapeutic approach. Gyroscope's lead investigational therapy, GT005, is designed to be administered as a one-time treatment under the retina, aiming to restore balance to an overactive complement system by enhancing the production of the Complement Factor I protein. Currently, GT005 is undergoing evaluation in multiple clinical trials, including a Phase I/II trial named FOCUS and two Phase II trials called EXPLORE and HORIZON. Founded in 2016 in Stevenage, Herefordshire, by Andrew Lotery, David Kavanagh, and Peter Lachmann, Gyroscope seeks to provide innovative solutions for patients suffering from retinal diseases.

Click Therapeutics, Inc. is a New York-based company that specializes in the engineering, validation, development, and commercialization of digital therapeutics aimed at addressing unmet medical needs through cognitive and neurobehavioral modification. Founded in 2012, Click Therapeutics focuses on creating software-based medical treatments for various conditions, including smoking cessation, major depressive disorders, schizophrenia, insomnia, and chronic pain. Its innovative Digital Therapeutics™ can be utilized independently or alongside traditional biomedical treatments, enhancing patient engagement and treatment outcomes. The company's Clickometrics® adaptive data science platform personalizes user experiences to optimize results. Notably, Click's smoking cessation program has successfully launched nationwide, and its leading prescription program is currently undergoing a phase III FDA registration trial for treating Major Depressive Disorder in adults.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Kadmon Holdings is a biopharmaceutical company focused on discovering, developing, and commercializing small molecules and biologics for the treatment of inflammatory and fibrotic diseases. Founded in 2010 and headquartered in New York, Kadmon's lead product candidates include Belumosudil (KD025), an orally administered selective inhibitor of rho-associated coiled-coil kinase 2, which is in Phase II clinical trials for chronic graft-versus-host disease and fibrotic diseases; KD045, an oral inhibitor of ROCK for fibrotic diseases; and KD033, an anti-PD-L1/IL-15 fusion protein for cancer treatment. The company also develops Tesevatinib for autosomal dominant polycystic kidney disease, ribavirin for hepatitis, and CLOVIQUE for Wilson’s disease. Kadmon has strategic collaborations with Nano Terra and Dyax Corp.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

Glycomine, Inc. is a biotech company focused on developing nanomedicines for rare genetic disorders, particularly those related to protein and lipid glycosylation, which can severely impact patients' health. Established in 2014 and located in San Carlos, California, Glycomine aims to address the significant unmet medical need for effective treatments in the realm of rare diseases, where approximately 95% lack any FDA-approved therapies. The company specializes in creating replacement therapies that utilize a combination of substrates, enzymes, and proteins, delivered through bio-nano materials that target specific cells within clinically relevant organs. By innovating in this area, Glycomine seeks to provide new therapeutic options for patients suffering from disorders of metabolism and protein misfolding.

Veralox Therapeutics specializes in the development of small molecule therapeutics aimed at addressing the underlying pathologies of thrombosis and type one diabetes. The company focuses on innovative treatment paradigms informed by a deep understanding of the molecular mechanisms involved in these diseases. By targeting conditions such as heparin-induced thrombocytopenia and thrombosis, Veralox Therapeutics aims to enhance patient outcomes and facilitate complete recovery from diabetes. Through its research and development efforts, the company seeks to provide effective and transformative therapies for individuals affected by these serious health conditions.

Tidal Therapeutics is a preclinical biotech company based at LabCentral in Cambridge, MA. The company is focused on developing nanoparticles that deliver mRNA to reprogram immune cells inside the body with applications in oncology and immune diseases.

Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines using its innovative virus-like particle (VLP) technology platform, which allows for the multivalent display of complex viral antigens. This technology aims to provide broad and durable protection against various infectious diseases, with an initial focus on life-threatening respiratory illnesses. Icosavax's pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and SARS-CoV-2. The company is dedicated to advancing its VLP technology to discover, develop, and ultimately commercialize effective vaccines against these infectious diseases.

Kymab Limited is a clinical-stage biopharmaceutical company based in Cambridge, United Kingdom, focused on discovering and developing fully human monoclonal antibody therapeutics. The company utilizes its proprietary Kymouse platform, which is part of the broader IntelliSelect® integrated platforms, to target difficult and novel drug challenges in various therapeutic areas, including immuno-oncology and immune-mediated diseases. Kymab’s technology allows for the generation of a diverse range of human antibodies, enhancing the potential for novel therapies in treating conditions such as cancer and autoimmune disorders. The company has established strategic collaborations, including partnerships with Heptares Therapeutics and LifeArc, to further its research and development efforts. Founded in 2009, Kymab Limited operates as a subsidiary of Kymab Group Limited.

Minervax is focused on developing a vaccine to prevent infections caused by Group B streptococcus (GBS), which can lead to serious health issues in newborns and adverse pregnancy outcomes. The company is researching a specific fusion protein that has demonstrated the ability to stimulate strong protective immunity against various clinically significant GBS strains. By utilizing this innovative approach, Minervax aims to provide medical professionals with an effective tool to protect newborns from potentially life-threatening bacterial infections associated with GBS.

Kiadis Pharma N.V. is a clinical-stage biopharmaceutical company based in Amsterdam, the Netherlands, specializing in the development of cell-based immunotherapy products for blood cancers and inherited blood disorders. The company is currently advancing its product pipeline, which includes K-NK002, an adjunctive immunotherapeutic in Phase II trials for blood cancer patients undergoing haploidentical hematopoietic stem cell transplants, and K-NK003, which is in Phase I/II trials for patients with acute myeloid leukemia who are relapsed or refractory. Additionally, Kiadis Pharma is developing K-NK00X, a preclinical NK-cell therapy candidate aimed at treating various solid tumors. The company is engaged in collaborative efforts with multiple institutions to explore innovative treatments, including a combination of a monoclonal antibody and natural killer cells for COVID-19. Established in 1997, Kiadis Pharma addresses critical unmet medical needs in oncology through its innovative therapeutic approaches.

Lava Therapeutics B.V. is a biotechnology company based in Utrecht, the Netherlands, founded in 2016. The company specializes in developing proprietary bispecific antibodies aimed at engaging gamma-delta T cells for the treatment of both hematological and solid cancers. Its innovative platform, known as the Gammabody platform, is designed to create next-generation bispecific antibodies that activate the immune system to recognize and eliminate tumor cells. By harnessing the potential of γδ T cells, Lava Therapeutics aims to produce potent, safe, and cost-effective biopharmaceuticals that enhance the body's ability to combat cancer, thereby transforming the landscape of cancer treatment.

Escient Pharmaceuticals is a biotechnology company based in San Diego, California, that develops and manufactures drugs targeting G protein-coupled receptors (GPCRs) for the treatment of neuro-immuno-inflammatory and autoreactive diseases. Founded in 2017, the company specializes in harnessing the therapeutic potential of specific orphan GPCRs, particularly the Mas-Related G-Protein Receptors (Mrgprs), to address various neurosensory-inflammatory disorders. Escient's innovative approach aims to provide first-in-class therapies for conditions associated with mast cell-mediated disorders and cholestatic pruritus, thereby expanding treatment options for healthcare providers and patients facing these challenging diseases.

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

Principia Biopharma Inc. is a late-stage biopharmaceutical company based in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead candidate, rilzabrutinib, is an inhibitor currently undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 and Phase 2 trials for immune thrombocytopenia and IgG4-related disease, respectively. Additionally, Principia is advancing PRN2246/SAR442168, aimed at treating multiple sclerosis and other central nervous system disorders, which is in Phase II trials. Another candidate, PRN473, is in Phase I trials for various immune-mediated diseases. Principia Biopharma collaborates with Genzyme Corporation to explore treatments for relapsing and progressive multiple sclerosis, enhancing its commitment to addressing significant unmet medical needs in immunology and oncology. Established in 2008, the company operates as a subsidiary of Sanofi.

Synthorx, Inc. is a biopharmaceutical company based in La Jolla, California, focused on developing innovative cytokine Synthorin programs for the treatment of cancer and autoimmune disorders. The company's lead product candidate, THOR-707, is a variant of recombinant human IL-2 being explored for various solid tumors, both as a standalone treatment and in combination with immune checkpoint inhibitors. Additionally, Synthorx is working on IL-2 Synthorin for autoimmune conditions, IL-10 Synthorin for immuno-oncology, and IL-15 Synthorins to enhance immune regulation. The company employs advanced synthetic biology techniques, utilizing a novel DNA base pair to create proteins with enhanced specificity, safety, and efficacy. These developments enable the manufacturing of improved therapeutics that can better modulate pharmacological properties compared to traditional biologics. Synthorx, which was established in 2014 and previously known as Alinos, Inc., operates as a subsidiary of Sanofi and holds exclusive rights to its innovative synthetic biology technology derived from research at The Scripps Research Institute.

Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines using its innovative virus-like particle (VLP) technology platform, which allows for the multivalent display of complex viral antigens. This technology aims to provide broad and durable protection against various infectious diseases, with an initial focus on life-threatening respiratory illnesses. Icosavax's pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and SARS-CoV-2. The company is dedicated to advancing its VLP technology to discover, develop, and ultimately commercialize effective vaccines against these infectious diseases.

Veralox Therapeutics specializes in the development of small molecule therapeutics aimed at addressing the underlying pathologies of thrombosis and type one diabetes. The company focuses on innovative treatment paradigms informed by a deep understanding of the molecular mechanisms involved in these diseases. By targeting conditions such as heparin-induced thrombocytopenia and thrombosis, Veralox Therapeutics aims to enhance patient outcomes and facilitate complete recovery from diabetes. Through its research and development efforts, the company seeks to provide effective and transformative therapies for individuals affected by these serious health conditions.

BioNTech is a Germany-based biotechnology company that specializes in developing innovative immunotherapies for cancer and other serious diseases, as well as vaccines for infectious diseases, including COVID-19. The company’s oncology pipeline includes a range of drug classes, such as mRNA-based drugs that encode antigens and neoantigens, cell therapies, bispecific antibodies, and antibody-drug conjugates. BioNTech has established partnerships with several major pharmaceutical companies, enhancing its research and development capabilities. Its COVID-19 vaccine, Comirnaty, represents its first commercialized product, demonstrating the company's commitment to addressing urgent health challenges through advanced therapeutic approaches.

Kymera Therapeutics is a biopharmaceutical company based in Watertown, Massachusetts, that specializes in the discovery and development of novel small molecule therapeutics aimed at selectively degrading disease-causing proteins. The company utilizes a proprietary platform for targeted protein degradation, leveraging the body’s natural protein recycling system. Kymera is developing several programs, including the IRAK4 program for immunology-inflammation diseases such as hidradenitis suppurativa and rheumatoid arthritis, the IRAKIMiD program to address MYD88-mutated diffuse large B cell lymphoma, and the STAT3 program targeting hematologic malignancies, solid tumors, and autoimmune diseases. Founded in 2015, Kymera Therapeutics is pioneering new approaches to treat diseases that have previously been considered untreatable, focusing on previously inaccessible therapeutic targets.

Click Therapeutics, Inc. is a New York-based company that specializes in the engineering, validation, development, and commercialization of digital therapeutics aimed at addressing unmet medical needs through cognitive and neurobehavioral modification. Founded in 2012, Click Therapeutics focuses on creating software-based medical treatments for various conditions, including smoking cessation, major depressive disorders, schizophrenia, insomnia, and chronic pain. Its innovative Digital Therapeutics™ can be utilized independently or alongside traditional biomedical treatments, enhancing patient engagement and treatment outcomes. The company's Clickometrics® adaptive data science platform personalizes user experiences to optimize results. Notably, Click's smoking cessation program has successfully launched nationwide, and its leading prescription program is currently undergoing a phase III FDA registration trial for treating Major Depressive Disorder in adults.

Eden Biologics, formerly known as JHL Biotech, is a biopharmaceutical company that specializes in the development of biosimilars and offers contract development and manufacturing services to various clients and partners. The company focuses on producing a diverse range of complex dosage forms, including oral solid, liquid, and semi-solid products, as well as macromolecule biologics. By leveraging its expertise in both global and regional regulatory affairs, Eden Biologics aims to make biopharmaceuticals more affordable and accessible to patients worldwide. Through its comprehensive services, the company supports the entire process from cell line development to commercial manufacturing, enhancing the efficiency and effectiveness of biopharmaceutical development for its partners.

Fulcrum Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing novel therapies for patients with genetically defined diseases that have significant unmet medical needs. The company's lead product candidate, losmapimod, is a small molecule currently undergoing Phase 2b clinical trials for the treatment of facioscapulohumeral muscular dystrophy, a rare condition characterized by progressive muscle wasting and disability. Another key candidate, FTX-6058, aims to upregulate fetal hemoglobin to treat sickle cell disease and beta-thalassemia. Fulcrum is also actively researching drug targets for other genetic disorders, including Duchenne muscular dystrophy and Friedreich ataxia, as well as neurological and pulmonary diseases. The company has established collaborations with Acceleron Pharma to explore biological targets in pulmonary disease and with MyoKardia to develop therapies for genetic cardiomyopathies. Founded in 2015, Fulcrum Therapeutics continues to focus on improving patient outcomes in rare disease contexts.

Imbria Pharmaceuticals, Inc. is a clinical-stage pharmaceuticals company based in Boston, Massachusetts, founded in 2018. The company specializes in developing innovative therapies aimed at addressing cardiometabolic disorders by enhancing or restoring cellular energy production. Imbria's research focuses on the mechanisms underlying these conditions, particularly in areas such as non-obstructive hypertrophic cardiomyopathy, stable angina, and heart failure with preserved ejection fraction. By translating scientific insights into practical treatments, Imbria Pharmaceuticals aims to meet significant unmet medical needs and improve the lives of patients affected by these life-altering disorders. Through its commitment to understanding cellular metabolism and mitochondrial function, the company works to advance healthcare solutions in the cardiometabolic space.

Ablynx is a biopharmaceutical company focused on the discovery and development of Nanobodies®, a unique class of therapeutic proteins derived from single-domain antibody fragments. These innovative proteins are designed to address a variety of serious human diseases, including inflammation, hematology, oncology, and pulmonary conditions. Currently, Ablynx has around 25 projects in its pipeline, with five Nanobodies in clinical development. By combining the advantages of traditional antibodies and small chemical molecules, Ablynx aims to provide new therapeutic options that can significantly improve patient care and outcomes.

Bioverativ Inc. is a biopharmaceutical company focused on developing therapies for hemophilia and other rare blood disorders, including cold agglutinin disease, sickle cell disease, and beta thalassemia. Founded in 2016 and headquartered in Waltham, Massachusetts, the company aims to transform the lives of patients through innovative research and development. Bioverativ's pipeline features hemophilia therapies that offer less-frequent prophylactic dosing for hemophilia A and subcutaneous dosing for hemophilia B, as well as gene therapy programs for both types of hemophilia. Additionally, the company is working on Sutimlimab, a treatment aimed at inhibiting the complement pathway of the immune system to address cold agglutinin disease. As a subsidiary of Sanofi, Bioverativ continues to advance its mission to provide effective treatments for chronic blood disorders.

Thermalin, Inc. is a biotechnology company based in Cleveland, Ohio, founded in 2010, that specializes in developing innovative insulin therapies for individuals with Type 1 and Type 2 diabetes. The company aims to disrupt the insulin and insulin delivery market with its first-in-class, ultra-rapid acting insulin, which is stabilized and highly concentrated. This new insulin formulation is designed for use in a disposable, postage-stamp-sized glucose management system and can also be utilized in existing insulin pumps. In addition to its lead program, Thermalin is exploring partnerships to develop no-cold-chain basal and mix insulins, as well as glucose-responsive insulins. By creating insulin analogs that do not require refrigeration and are rapidly absorbed, Thermalin seeks to reduce the burden of insulin therapy, thereby improving patient adherence and health outcomes.

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

Eden Biologics, formerly known as JHL Biotech, is a biopharmaceutical company that specializes in the development of biosimilars and offers contract development and manufacturing services to various clients and partners. The company focuses on producing a diverse range of complex dosage forms, including oral solid, liquid, and semi-solid products, as well as macromolecule biologics. By leveraging its expertise in both global and regional regulatory affairs, Eden Biologics aims to make biopharmaceuticals more affordable and accessible to patients worldwide. Through its comprehensive services, the company supports the entire process from cell line development to commercial manufacturing, enhancing the efficiency and effectiveness of biopharmaceutical development for its partners.

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

Yumanity Therapeutics is transforming drug discovery for neurodegenerative diseases caused by protein misfolding. Founded in December 2014 by award-winning protein folding expert, Susan Lindquist, and renowned biotech industry leader, Tony Coles, Yumanity is working to identify and develop new, disease-modifying therapies that address several illnesses with critical unmet medical needs. The initial focus of the company is neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS). The company’s proprietary platforms have already identified one potential new target for treating Parkinson’s disease, and Yumanity is actively advancing its new chemical lead series for this condition, as well as identifying additional compounds for Alzheimer’s disease and ALS.

Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.

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.

Ovid Therapeutics Inc. is a biopharmaceutical company based in New York, focused on developing innovative therapies for patients with neurological disorders, particularly rare diseases. Founded in 2014, the company is advancing a promising pipeline of drug candidates aimed at treating conditions such as angelman syndrome and fragile X syndrome. Its lead candidate, OV101, is in Phase III clinical trials for angelman syndrome and has completed Phase II trials for fragile X syndrome. Additionally, Ovid is developing OV935, currently in Phase II trials for cyclin-dependent kinase-like 5 deficiency disorder and Dravet syndrome. The company is also researching OV329 for infantile spasms and OV881 as a microRNA gene therapy for angelman syndrome. Ovid Therapeutics has established collaborations and licensing agreements with various pharmaceutical and research organizations to enhance its development efforts. The company's commitment to addressing unmet medical needs is underpinned by strong intellectual property and a focus on safe, effective treatments for patients facing challenging neurological conditions.

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).

Lysosomal Therapeutics, Inc. is a biotechnology company focused on discovering and developing small-molecule therapies for severe neurological diseases. Founded in 2011 and based in Cambridge, Massachusetts, the company targets disorders such as Gaucher-related neurodegeneration, Parkinson’s disease, and other synucleinopathies. Lysosomal Therapeutics employs a unique molecular platform informed by the clinically validated connection between lysosomal disorders and neurodegeneration. This approach facilitates the development of innovative treatment options aimed at improving the health and quality of life for patients affected by these debilitating conditions.

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).

MyoKardia, Inc. is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing targeted therapies for rare cardiovascular diseases. Founded in 2012 and headquartered in Brisbane, California, the company primarily addresses heritable cardiomyopathies and genetically-driven heart failure caused by biomechanical defects in cardiac muscle contraction. Its lead product candidate, Mavacamten, is an orally administered allosteric modulator of cardiac myosin currently in Phase III trials for hypertrophic cardiomyopathy (HCM). Additionally, MyoKardia is developing Danicamtiv, an orally administered small molecule that has completed Phase I and IIa trials for dilated cardiomyopathy. The company also has several preclinical programs, including MYK-224 for HCM, ACT-1 for diastolic dysfunction, and LUS-1 for diastolic dysfunction. As of late 2020, MyoKardia operates as a subsidiary of Bristol-Myers Squibb Company.

Lysosomal Therapeutics, Inc. is a biotechnology company focused on discovering and developing small-molecule therapies for severe neurological diseases. Founded in 2011 and based in Cambridge, Massachusetts, the company targets disorders such as Gaucher-related neurodegeneration, Parkinson’s disease, and other synucleinopathies. Lysosomal Therapeutics employs a unique molecular platform informed by the clinically validated connection between lysosomal disorders and neurodegeneration. This approach facilitates the development of innovative treatment options aimed at improving the health and quality of life for patients affected by these debilitating conditions.

Immune Design Corp. is a clinical-stage immunotherapy company headquartered in Seattle, Washington, that specializes in developing innovative treatments for cancer through in vivo methods that empower the immune system to combat disease. The company utilizes its ZVex and GLAAS discovery platforms to create a new generation of immune-based products. Its primary product candidate, G100, is currently undergoing Phase II clinical trials for follicular non-Hodgkin lymphoma, while another candidate, CMB305, focuses on enhancing tumor-specific cytotoxic T cells. Immune Design's research aims to overcome limitations of existing oncological therapies, and the company has established collaboration and license agreements with Sanofi Pasteur. Founded in 2008, Immune Design operates as a subsidiary of Merck & Co., Inc.

Edimer Pharmaceuticals focuses on developing EDI200 as a treatment for X-linked Hypohydrotic Ectodermal Dysplasia (XLHED), a rare genetic disorder characterized by symptoms such as insufficient sweat glands, impaired temperature regulation, respiratory difficulties, and abnormal hair and tooth development. The company aims to enhance the health and quality of life for individuals affected by this orphan disease. Edimer is supported by a team of experienced biotechnology professionals and a network of esteemed clinical and scientific advisors, all committed to advancing drug development for XLHED. Through its innovative approach, Edimer seeks to provide significant and lasting improvements in the management of this condition for future generations.

Lumena Pharmaceuticals is focused on developing oral therapeutics for rare liver diseases, aiming to enhance liver function and alleviate disease symptoms. The company's clinical-stage product candidates are designed to selectively target intestinal transporters, minimizing systemic toxicity and absorption in the body. Their lead candidate, LUM001, has undergone extensive evaluation through 12 clinical studies involving over 1,400 subjects, positioning it for rapid advancement in treating cholestatic liver disease in both pediatric and adult patients. In addition to its primary goal of addressing rare liver diseases, Lumena's innovative therapeutic approach shows promise for treating metabolic liver diseases, a significant and rising health concern affecting both children and adults.

Melinta Therapeutics, Inc. is a commercial-stage pharmaceutical company based in Morristown, New Jersey, specializing in the discovery, development, and commercialization of anti-infective therapies for bacterial infections in North America. The company offers a range of antibiotics, including Baxdela, Vabomere, Orbactiv, and Minocin, targeting various serious bacterial infections such as acute bacterial skin and skin structure infections (ABSSSIs) and gram-negative infections. Melinta is actively advancing its investigational antibiotic, delafloxacin, which is currently in Phase 3 development for ABSSSIs. Additionally, the company is engaged in research to develop new classes of antibiotics aimed at combating drug-resistant pathogens, particularly the ESKAPE group responsible for many hospital-acquired infections. Through its innovative approaches, Melinta Therapeutics addresses the urgent need for new therapies against drug-resistant infections, a critical public health challenge today.

Esperance Pharmaceuticals Inc. is a biotechnology company based in Houston, Texas, focused on developing targeted anticancer drugs for women with ovarian and breast cancer. The company's key product, EP-100, is an oncolytic peptide designed to selectively bind to receptors for luteinizing hormone releasing hormone (LHRH) found on cancer cells, enabling it to destroy these cells while sparing normal tissue. This innovative approach allows the drug to effectively target both dividing and non-dividing cancer cells, including those resistant to traditional chemotherapy. Esperance has formed a strategic alliance with MD Anderson Cancer Center to expedite the clinical development of EP-100, further enhancing its commitment to advancing cancer treatment. The company was incorporated in 2005 and is dedicated to improving outcomes for patients through its novel therapeutic strategies.

Sanofi Genzyme is the specialty care global business unit of Sanofi, primarily dedicated to addressing the challenges posed by rare diseases, multiple sclerosis, immunology, and oncology. Established in 1981 as Genzyme in Boston, the company grew to become a major player in biotechnology, known for pioneering treatments for rare genetic disorders. In 2011, Genzyme was integrated into Sanofi, enhancing its capabilities in developing therapies for complex conditions that are often difficult to diagnose and treat. Sanofi Genzyme emphasizes a patient-centered approach, collaborating closely with healthcare providers and patient communities to advance new therapies. Over the years, its focus has expanded beyond rare diseases to include significant contributions in multiple sclerosis, oncology, and immunology, reflecting a commitment to innovation and improving patient outcomes across diverse therapeutic areas.

Regulus Therapeutics Inc. is a clinical stage biopharmaceutical company based in San Diego, California, specializing in the discovery and development of therapies that target microRNAs for various diseases. The company is advancing its lead product candidates, RG-012 and RGLS4326, which are in Phase II and Phase Ib clinical trials, respectively. RG-012 targets miR-21 for the treatment of Alport syndrome, a severe kidney disease, while RGLS4326 targets miR-17 for autosomal dominant polycystic kidney disease. In addition to these candidates, Regulus is developing a pipeline of preclinical drug products, including RGLS5579, aimed at inhibiting miR-10b, as well as programs targeting hepatitis B virus and non-alcoholic steatohepatitis. Founded in 2007, Regulus Therapeutics is at the forefront of leveraging the therapeutic potential of microRNA, a significant scientific breakthrough in recent years.

Bluebird bio, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on researching, developing, and commercializing gene therapies for severe genetic diseases and cancer. The company's gene therapy programs include LentiGlobin, aimed at treating β-thalassemia and sickle cell disease, and Lenti-D for cerebral adrenoleukodystrophy. In oncology, bluebird bio is developing chimeric antigen receptor T (CAR T) cell therapies, specifically bb2121 and bb21217, for multiple myeloma. The company has formed strategic collaborations with several organizations, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to advance gene therapies in oncology and immune cell therapies. Additionally, bluebird bio collaborates with various partners to explore T cell receptor product candidates and in vivo genome editing treatments for genetic disorders. Established in 1992 and originally known as Genetix Pharmaceuticals, the company transitioned to its current name in 2010.

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.

Shantha Biotechnics, established in 1993 by Dr. Varaprasad Reddy and based in Hyderabad, India, specializes in the development, manufacturing, and marketing of vaccines. Operating in a state-of-the-art facility that meets international standards, the company has collaborated with organizations such as UNICEF and PAHO to supply vaccines to major markets across Asia-Pacific, Africa, and Latin America. Shantha is notable for launching SHANVAC-B™, the first recombinant Hepatitis B vaccine produced in India in 1997. Its product line includes SHANVAC-B™, SHANTETRA™ (a combination vaccine for Diphtheria, Pertussis, Tetanus, and Hepatitis B), SHAN5™ (a combination vaccine for Diphtheria, Pertussis, Tetanus, Haemophilus influenza B, and Hepatitis B), and SHANTT™ (a Tetanus toxoid vaccine), all of which are prequalified by the World Health Organization for global distribution to United Nations agencies. Additionally, Shantha has a significant portfolio of vaccines that are currently in development.

BiPar Sciences Inc., based in Brisbane, California, is focused on developing PARP inhibitors as therapies for various cancers. The company's primary experimental product, BSI-201, is undergoing mid-stage trials for advanced breast cancer, ovarian cancer, and other tumors, particularly those that are therapy-resistant or difficult to treat. In addition to BSI-201, BiPar is working on a follow-on inhibitor candidate for pancreatic cancer and an iodothyronine program that possesses anti-tubulin properties, which could enhance the effectiveness of chemotherapy. BiPar has raised approximately $60 million in venture capital from several prominent investment firms and has also secured venture debt. In April 2009, the company announced its acquisition by a French pharmaceutical company for up to $500 million.

Proteostasis Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for cystic fibrosis and other diseases linked to protein homeostasis. The company’s primary product candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. Proteostasis employs theratyping to tailor treatment strategies based on individual responses to CFTR modulators, regardless of specific mutations. The company collaborates with the Cystic Fibrosis Foundation and Genentech to advance its research and development efforts. Founded in 2006 and based in Boston, Massachusetts, Proteostasis was previously known as Proteoguard, Inc. and rebranded in 2007. In December 2020, it was acquired by Yumanity Therapeutics in a reverse merger transaction.

[Sirtris Pharmaceuticals](http://www.sirtrispharma.com) (NASDAQ: SIRT) is a biopharmaceutical company focused on discovering and developing proprietary, orally available, small molecule drugs with the potential to treat diseases associated with aging, including metabolic diseases such as Type 2 Diabetes. via: [Sirtris](http://www.sirtrispharma.com)

Nereus Pharmaceuticals, Inc. engages in the discovery and development of therapeutics for the treatment of oncology, infectious diseases, and inflammation. The company uses its marine microbiology technologies for the discovery and development of drug candidates. Its oncology drug candidates include NPI-2358, a tumor vascular disrupting agent for the treatment of solid tumors; and NPI-0052, a proteasome inhibitor for the treatment of solid tumors, lymphomas, and multiple myeloma. The company was founded in 1998 and is based in San Diego, California.

Vigil Neuroscience Inc. is a therapeutics company focused on microglia, the brain's immune sentinel cells, to address both rare and common neurodegenerative diseases. The company aims to restore the vigilance of microglia to improve patient outcomes. Vigil is advancing its lead candidate, a monoclonal antibody TREM2 agonist, through Phase 1 studies and is also developing an orally available small molecule TREM2 agonist, VG-3927, which is positioned to enter Investigational New Drug (IND) application. Additionally, Vigil is conducting the IGNITE trial, a Phase 2 proof-of-concept study that represents the first interventional trial for patients with adult-onset leukodystrophy. By leveraging modern neuroscience and various therapeutic modalities, Vigil seeks to deliver precision therapies that enhance the lives of patients and their families while expanding its pipeline and supporting the understanding of microglia biology as a critical therapeutic target.