Biogen

SAGE Therapeutics is a biopharmaceutical company focused on developing and commercializing innovative treatments for central nervous system (CNS) disorders. Its lead product, ZULRESSO, is an intravenous formulation of brexanolone designed for postpartum depression. The company's pipeline includes SAGE-217, a neuroactive steroid in Phase III trials for various depressive disorders and anxiety, and SAGE-324, currently in Phase II trials for essential tremors and previously tested for other neurological conditions. Additionally, SAGE-718, which targets multiple CNS disorders, has completed Phase I trials, while SAGE-904 and SAGE-689 are in early clinical stages for NMDA-related disorders. SAGE Therapeutics has formed strategic collaborations with Shionogi & Co., Ltd. and Biogen Inc. for the development and commercialization of its key product candidates. Founded in 2010 and headquartered in Cambridge, Massachusetts, the company aims to address the unmet needs of patients affected by debilitating CNS disorders through scientific innovation and strategic partnerships.

Autobahn Therapeutics, Inc. is a biopharmaceutical company based in San Diego, California, dedicated to developing small molecule therapies for central nervous system (CNS) disorders. The company focuses on leveraging its expertise in brain-targeting chemistry to create innovative treatments that harness the regenerative capabilities of the human body. Its primary product candidate, ABX-002, is a thyroid hormone receptor beta agonist aimed at treating multiple sclerosis and adrenomyeloneuropathy, a rare genetic disorder. Founded in 2017, Autobahn Therapeutics is committed to addressing significant unmet medical needs in areas such as neuropsychiatry, neurodegeneration, and neuroinflammation, utilizing validated clinical and biological targets to guide its research and development efforts.

CAMP4 Therapeutics Corporation is a bioinformatics company based in Cambridge, Massachusetts, founded in 2015. The company specializes in gene regulation circuitry, utilizing a 4-D gene circuitry platform to codify the activation of the over 24,000 genes in the human body into a set of combinatorial rules. This approach allows CAMP4 to manipulate gene production through existing cellular signaling pathways, directly addressing the fundamental causes of diseases. By leveraging machine learning algorithms, the platform simplifies the drug discovery process, enabling the identification of druggable targets and facilitating a quicker and less risky path to developing new medicines. This innovative methodology significantly accelerates the initiation of disease treatment, ultimately benefiting patients by reducing the time required to bring new therapies to market.

Nightstar Therapeutics plc is a clinical-stage gene therapy company based in London, focused on developing innovative one-time treatments for rare inherited retinal diseases. The company’s lead product candidate, NSR-REP1, is currently in phase 3 clinical development for choroideremia, while NSR-RPGR is undergoing phase 2/3 trials for X-linked retinitis pigmentosa. Additionally, Nightstar is developing NSR-BEST1 and NSR-ABCA4, with the latter in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address the unmet medical needs of patients suffering from these conditions.

Samsung Bioepis Co., Ltd. is a biopharmaceutical company based in Incheon, South Korea, that specializes in the development and production of biosimilar medicines. Founded in 2012, the company aims to enhance patient access to high-quality treatment options through its extensive pipeline of biosimilar candidates. Samsung Bioepis focuses on innovative therapies for various medical conditions, including neurodegenerative diseases, hemophilia, and autoimmune disorders. The company has established a strategic collaboration with Takeda Pharmaceutical Company, which further strengthens its position in the healthcare industry by leveraging process innovation to deliver effective therapies.

Neurimmune is a biopharmaceutical company dedicated to the development of immunotherapeutics for the treatment and prevention of human disorders with high unmet medical needs. With its unique Reverse Translational Medicine™ platform, Neurimmune creates recombinant human-derived monoclonal antibodies with biophysical characteristics closely resembling those occurring in healthy elderly. These antibodies display unique properties such as target selectivity, superior pharmacodynamics and low immunogenicity, resulting in superior risk profiles and excellent efficacy. Neurimmune’s pipeline comprises programs for a broad variety of disease related targets and specific target conformations. These include misfolded, oligomeric, fibrillar or post-translationally modified forms of disease-associated proteins as well as physiological conformations involved in major disease pathways.

Remedy Pharmaceuticals, Inc. is a clinical stage pharmaceutical company based in New York that specializes in developing small molecule drugs aimed at treating acute central nervous system disorders. Founded in 2004, the company focuses on addressing conditions such as stroke, traumatic brain injury, spinal cord injury, organ ischemia, and complications arising from CNS surgery. One of its key products, RP-1127, is an intravenous formulation of glyburide, which acts as an inhibitor of NCCa-ATP channels by antagonizing the type-1 sulfonylurea receptor. The company's innovative approach targets critical aspects of central nervous system injuries, highlighting its commitment to advancing treatment options in this vital area of healthcare.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic disorder primarily affecting boys. The company's lead candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in muscle tissues. In addition to SGT-001, Solid Biosciences is advancing Anti-LTBP4, a monoclonal antibody designed to mitigate fibrosis and inflammation by targeting the LTBP4 protein. The company also works on developing biomarkers and wearable assistive devices under the Solid Suit program, which aims to provide therapeutic benefits to patients. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to further develop and commercialize gene therapies for DMD. The company’s initiatives address a critical need, as there are currently no effective cures or satisfactory treatments for this progressive and life-threatening condition.

Rodin Therapeutics is a biotechnology company based in Cambridge, Massachusetts, focused on developing therapeutics for cognitive disorders through the application of epigenetic insights. Founded in 2013 with backing from Atlas Venture and Johnson & Johnson Development Corporation, the company leverages advanced structural biology capabilities and a team experienced in central nervous system (CNS) drug development. Rodin's innovative approach centers on the modulation of histone deacetylase (HDAC) complexes to restore synaptic function and promote neuronal health in degenerative brain diseases. This strategy aims to address significant unmet medical needs in the treatment of neurological disorders. As of November 2019, Rodin operates as a subsidiary of Alkermes Inc.

Convergence Pharmaceuticals is an independent biotechnology company focused on the development of novel analgesics with potentially commercially attractive efficacy, responder-rate and side effect profiles. The Company was formed in October 2010 following the acquisition of certain neuroscience clinical assets from GlaxoSmithKline (“GSK”). Convergence Pharmaceuticals has a pipeline of differentiated clinical-stage compounds targeting the points of convergence in chronic pain signaling through modulation of specific ion-channels.

True North Therapeutics, Inc. is a biotechnology company focused on developing therapies that target the complement pathway of the immune system, specifically for rare diseases with high unmet clinical needs. Founded in 2013 and based in South San Francisco, California, the company’s lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway. This approach aims to prevent inflammatory mediators and phagocytosis in conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, and other complement-mediated disorders. True North is also exploring additional treatment options for a variety of diseases in hematology, dermatology, and neurology. The company operates as a subsidiary of Bioverativ Inc.

Ataxion is a discovery-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapies for rare and underserved neurological diseases. Founded in April 2013, Ataxion aims to address conditions such as hereditary ataxia and other degenerative disorders, including multiple sclerosis and Huntington's disease. The company specializes in creating novel small-molecule therapeutics that utilize ion-channel modulators to provide effective treatments for these debilitating conditions. By targeting unmet medical needs in the field of neurology, Ataxion is committed to improving the quality of life for patients affected by these challenging diseases.

Stromedix is a biomedical company that develops therapies targeting fibrosis, organ failure, and cancer. Initially, the company will concentrate on chronic indications, utilizing its expertise to address these conditions effectively. Stromedix aims to expand its therapeutic pipeline into related areas, including acute organ failure and cancer, by remodeling tissues to improve patient outcomes.

TYSABRI is a prescription medication designed to treat adults with relapsing forms of multiple sclerosis (MS), including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease. It is administered as an infusion every four weeks, making it a less frequent option compared to daily or weekly treatments. While TYSABRI can be effective for patients who have not responded to other therapies or whose physical disability has worsened, it carries a risk of progressive multifocal leukoencephalopathy (PML). Therefore, it is crucial for patients to discuss the potential benefits and risks of TYSABRI with their healthcare provider before starting treatment. With nearly 400,000 individuals in the United States affected by MS, TYSABRI represents a significant option in the evolving landscape of MS therapies.

Knopp Biosciences LLC is a biotechnology company based in Pittsburgh, Pennsylvania, focused on discovering and developing innovative treatments for inflammatory and neurological diseases. Founded in 2004, the company is engaged in clinical programs targeting inflammation and hematology, including dexpramipexole, an anti-inflammatory therapeutic that modulates white blood cells. Additionally, Knopp has a hematology discovery program aimed at selectively arresting eosinophil maturation with orally available medications. The company also explores a KCNQ2 preclinical program, which seeks to activate a crucial ion channel in nerve cells, addressing conditions such as neonatal encephalopathy, epilepsy, and neuropathic pain. Furthermore, Knopp is developing small molecule modulators to enhance mitochondrial efficiency as part of its mitochondrial discovery program. Through its focused research and development efforts, Knopp Biosciences aims to provide breakthrough treatments that improve patient outcomes and address significant healthcare challenges.

The Museum of Science, founded in 1830, is one of the largest science centers in the world and the most visited cultural institution in New England, attracting over 1.5 million visitors annually. It offers a comprehensive exploration of science, technology, engineering, and math (STEM) through engaging programs and hundreds of interactive exhibits. Notable attractions include the Thomson Theater of Electricity, Charles Hayden Planetarium, Mugar Omni Theater, Gordon Current Science & Technology Center, 3-D Digital Cinema, and a Butterfly Garden. The Museum plays a significant role in education, reaching 25,000 teens globally through the Intel Computer Clubhouse Network and leading a National Science Foundation-funded initiative to enhance informal science education. Its acclaimed "Science Is an Activity" exhibit plan has received numerous NSF grants and has influenced science centers internationally. Additionally, the Museum's National Center for Technological Literacy® has developed engineering curricula that have benefited over 40,600 teachers and nearly 3 million students across the United States.

Vivoryon Therapeutics is a Germany based biopharmaceutical company engaged in the activities of research and development, preclinical and clinical trials. It is focused on the development of therapeutic products for the treatment of Alzheimer's disease and cancer. The company's pipeline products includes PQ912, PQ1565 and PQ-family.

Portola Pharmaceuticals, Inc. is a biopharmaceutical company headquartered in South San Francisco, California, founded in 2003. It specializes in developing and commercializing innovative therapeutics primarily aimed at treating thrombosis, hematologic disorders, and inflammation. The company's lead product is Andexxa, an antidote for the anticoagulants rivaroxaban and apixaban. Additionally, Portola offers Bevyxxa, a once-daily oral Factor Xa inhibitor designed to prevent venous thromboembolism in adults with acute medical conditions. The firm is also developing several investigational products, including cerdulatinib, an oral dual inhibitor for hematologic cancers, and other compounds targeting chronic inflammatory diseases and cardiovascular conditions. Portola engages in collaborative agreements with various pharmaceutical companies to enhance its research and development efforts. As of July 2020, it operates as a subsidiary of Alexion Pharmaceuticals, Inc.

iPierian is engaged in the research and development of therapies aimed at treating neurodegenerative diseases, including spinal muscular atrophy (SMA), amyotrophic lateral sclerosis (ALS), Parkinson's disease, and Alzheimer's disease. The company utilizes induced pluripotent stem cells to facilitate drug discovery and development, focusing on addressing significant unmet medical needs. In particular, iPierian is developing therapies and monoclonal antibodies that target Tauopathies, which are diseases characterized by the accumulation of tau protein in the brain. These therapies aim to slow the spread of tau throughout the brain, thereby inhibiting the progression of associated diseases.

Knopp Biosciences LLC is a biotechnology company based in Pittsburgh, Pennsylvania, focused on discovering and developing innovative treatments for inflammatory and neurological diseases. Founded in 2004, the company is engaged in clinical programs targeting inflammation and hematology, including dexpramipexole, an anti-inflammatory therapeutic that modulates white blood cells. Additionally, Knopp has a hematology discovery program aimed at selectively arresting eosinophil maturation with orally available medications. The company also explores a KCNQ2 preclinical program, which seeks to activate a crucial ion channel in nerve cells, addressing conditions such as neonatal encephalopathy, epilepsy, and neuropathic pain. Furthermore, Knopp is developing small molecule modulators to enhance mitochondrial efficiency as part of its mitochondrial discovery program. Through its focused research and development efforts, Knopp Biosciences aims to provide breakthrough treatments that improve patient outcomes and address significant healthcare challenges.

Virdante Pharmaceuticals, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, focused on the development and commercialization of drugs for autoimmune and inflammatory diseases. Founded in 2007 and previously known as Centaurus Pharmaceuticals, the company specializes in creating antibody and Fc-fusion therapies aimed at addressing various autoimmune conditions. Through its innovative research and development efforts, Virdante Pharmaceuticals seeks to improve treatment options for patients suffering from these disorders.

Intellikine works in the discovery and development of novel, small molecule therapies targeting the PI3K/Akt/mTOR pathway. Intellikine is committed to building an exceptional team and a powerful discovery platform that rapidly generates small molecule kinase inhibitor drug candidates that will become the next generation of medical breakthroughs.

CalciMedica, Inc. is a biotechnology company based in La Jolla, California, focused on the discovery and development of small molecule drugs aimed at treating autoimmune and inflammatory diseases. Founded in December 2006 by a team of scientists with backgrounds in calcium signaling, the company specializes in the targeted inhibition of calcium release-activated calcium channels, a key mechanism in immune response regulation. The founding team includes individuals who previously collaborated at TorreyPines Therapeutics and the CBR Institute, where they made significant contributions to understanding calcium signaling pathways. Through its innovative research, CalciMedica aims to develop effective therapies for patients suffering from various autoimmune conditions.

Provasculon is a biotechnology company focused on developing novel therapeutic proteins aimed at healing organs damaged by ischemia. Founded based on the discoveries of Richard Lee, MD, and Vincent Segers, MD, both affiliated with Brigham and Women's Hospital, Provasculon specializes in innovative applications of Stromal Cell-Derived Factor-1 (SDF-1). The company's lead therapeutic protein shows promise in restoring the function of hearts severely affected by ischemic conditions, potentially bringing them back to near normal performance. Through its research and development efforts, Provasculon seeks to address critical medical needs in the treatment of ischemic organ damage.

PanGenetics B.V., based in Utrecht, Netherlands, specializes in the development of monoclonal antibodies aimed at treating immune-mediated diseases. The company focuses on advancing antibodies from late-stage research to clinical proof of concept, utilizing a lean business model that outsources manufacturing and clinical development to specialized providers. Among its clinical programs is PG110, along with PG102, a CD40 antagonist currently undergoing evaluation in a clinical study for patients with psoriatic arthritis. Additionally, PanGenetics employs an in-licensing model to expand its portfolio of antibody-based therapeutic products. The company's strategic approach allows it to concentrate on its core competencies while leveraging external expertise for other critical functions.

Escoublac Inc. is a biotechnology company based out of Cambridge, Massachusetts, United States.

Syntonix discovers and develops novel, long-acting, proprietary therapeutic products using the company’s proprietary Fc-fusion proteins and other engineered ligands which bind to specific Fc receptors. These technologies, including Syntonix’ drug delivery platform technology Transceptor™, harness the body’s natural pathways for protecting antibodies against premature destruction and for transporting antibodies across cell barriers such as those in the lungs and intestines. Syntonix focuses on using these technologies to create next generation treatments for devastating chronic diseases such as hemophilia, anemia, and autoimmune disorders. Syntonix develops selected products for specialty indications, such as hemophilia, on its own. Other products will be evaluated and commercialized through partnerships with companies with established market positions. The company is headed by an experienced management team and has focused initially on using its proprietary technology to enhance existing drugs with proven safety, efficacy, and economic return. Syntonix currently occupies a 25,000 square foot state-of-the-art laboratory facility, including a cGMP clinical manufacturing suite, in Waltham, Massachusetts.

Fumapharm is a privately held pharmaceutical company.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, that specializes in the discovery and development of antibody-based therapeutics for cancer treatment. The company's pipeline includes several immuno-oncology candidates, such as Margetuximab, a monoclonal antibody currently in Phase III trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include various monoclonal antibodies targeting immune checkpoints like PD-1 and B7-H3, as well as combination therapies that leverage multiple targets. MacroGenics employs advanced Fc engineering technology to enhance antibody functionality and is also exploring applications for autoimmune disorders and infectious diseases. The company has established strategic collaborations with several partners to advance its research and development efforts. Founded in 2000, MacroGenics is committed to innovating immunotherapeutics to address significant medical needs.

Conforma Therapeutics Corporation designs and develops drugs for the treatment of cancer. It develops drugs that induce tumor cells to degrade the proteins that promote cancer growth. The company offers HSP90 and CNF1010 which are used for the treatment of cancer. The company was incorporated in 1999 and is based in San Diego, California

GlobeImmune, Inc. is a biopharmaceutical company based in Louisville, Colorado, dedicated to developing therapeutic products for cancer and infectious diseases through its proprietary Tarmogen platform. The company’s pipeline includes GS-4774, currently in Phase 2 trials for hepatitis B virus treatment, and various candidates targeting cancer, such as GI-6207 for medullary thyroid cancer, GI-6301 for tumors expressing the brachyury protein, and GI-4000 for resected pancreatic cancer. Additionally, GlobeImmune is advancing several candidates for infectious diseases, including GI-19000 for tuberculosis, GI-2010 for HIV, and GI-18000 for hepatitis D virus, currently in preclinical stages. The Tarmogen platform also includes products designed to address challenges in cancer therapy, such as preventing the emergence of mutated escape variants. The company has established strategic collaborations with Gilead Sciences and Celgene Corporation. Originally founded as Ceres Pharmaceuticals in 1995, GlobeImmune changed its name in 2001 to reflect its focus on innovative immunotherapy solutions.

Raven biotechnologies, Inc., a biotechnology company, focuses on the development of monoclonal antibody therapeutics for treating cancer. Its lead product candidate, RAV12, targets adenocarcinomas and used for the treatment of gastrointestinal, lung, colon, pancreatic, prostate, breast, brain, and ovarian cancer. The company was founded in 1999 and is based in South San Francisco, California. As of July 17, 2008, Raven Biotechnologies, Inc. operates as a subsidiary of MacroGenics, Inc.

Sunesis is an emerging biopharmaceutical company creating new medicines to improve the lives of people with cancer. They are building a pipeline of drugs that selectively block critical mechanisms required for tumor growth and survival. The fight against cancer is an enormous mission that requires an integrated approach fueled by a commitment to innovation.

IDEC Pharmaceuticals develop therapies for the treatment of neurodegenerative, hematologic, and autoimmune diseases. Idec Pharmaceuticals was founded in 1986 by UC-San Diego physician and immunologist Ivor Royston, San Diego bioentrepreneur Howard Birndorf, and Stanford University cancer researchers Ron Levy and Richard Miller. In 2003, Idec Pharmaceuticals was merged with Biogen.