Best health care companies in Cambridge, MA

Sarepta Therapeutics is a biopharmaceutical company that focuses on the discovery and development of precision genetic medicine to treat rare diseases. It specializes in gene therapy, RNA-targeted exon skipping, and gene editing.

Sanofi Genzyme is the specialty care global business unit of Sanofi, focused on rare diseases, multiple sclerosis, immunology, and oncology. They help people with debilitating and complex conditions that are often difficult to diagnose and treat. They are dedicated to discovering and advancing new therapies, providing hope to patients and their families around the world. Genzyme became part of Sanofi in 2011. While continuing to focus on rare diseases, they also established ourselves as a leader in the field of multiple sclerosis. In 2016, their focus expanded to include Sanofi's programs in oncology and immunology. Their approach is shaped by a long history of developing highly specialized treatments and forging close relationships with physician and patient communities. Founded as Genzyme in Boston in 1981, they rapidly grew to become one of the world's leading biotechnology companies, recognized for their pioneering development of treatments for rare genetic disorders and their innovative contributions to medical science and biomanufacturing.

Moderna, Inc. is a Cambridge, Massachusetts-based biotechnology company that is focused on drug discovery and drug development based on messenger RNA. The company creates synthetic mRNA that can be injected into patients to help them create their own therapies.

Foundation Medicine plans to create clinical diagnostic tests that can use the latest in genetic sequencing technology to identify personalized cancer therapies for patients.

Tango Therapeutics delivers the next generation of precision medicine to people with cancer that leverages the genetic principle of synthetic lethality to discover and develop therapies that take aim at driver genes in cancer.

Relay therapeutics is a developer of an allosteric drug-discovery platform intended to apply computational techniques to protein motion.The company's platform detects and characterizes interactions that occur anywhere on a protein and combines computational methods with experimental approaches across the fields of structural biology, biophysics and chemistry, enabling pharmaceutical companies to solve and design therapies against complex diseases like cancer and others.

JanusBio has assembled a world's leading drug development team targeting the inhibitions of toll-like receptor 7/8/9. This team contributed to the identification of toll-like receptor 7/8/9 and an understanding of these receptors in auto-immune diseas...

CRISPR Therapeutics is focused on the development of transformative medicines using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. They have licensed the foundational CRISPR/Cas9 patent estate for human therapeutic use from their scientific founder, Dr. Emmanuelle Charpentier, who co-invented the application of CRISPR/Cas9 for gene editing. CRISPR Therapeutics' vision is to cure serious human diseases at the molecular level using CRISPR-Cas9. The company is headquartered in Basel, Switzerland and has operations in London, UK.

Fulcrum Therapectics to develop new medicines to deliver a new future for patients and their families by transforming gene regulation in disease. Above all we are champions for our patients. Trust, Respect, Compassion and Integrity define who we are.

Pandion Therapeutics is a biotechnology company developing bispecific antibody therapeutics to achieve localized immunomodulation at the site of disease for durable, tissue-specific treatment of patients with autoimmune and inflammatory diseases, and organ transplants. Pandion's approach has the potential to more effectively induce and sustain response and remission in patients with many autoimmune and inflammatory conditions. They aim to shift the paradigm and create a new generation of drugs with transformative efficacy and improved safety.

Voyager Therapeutics is a gene therapy company developing life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS). Voyager is committed to advancing the field of AAV (adeno-associated virus) gene therapy through innovation and investment in vector optimization and engineering, dosing techniques, as well as process development and production. The company's initial pipeline is focused on CNS diseases in dire need of effective new therapies, including Parkinson's disease, a monogenic form of amyotrophic lateral sclerosis (ALS), and Friedreich's ataxia. Founded by scientific and clinical leaders in the fields of AAV gene therapy, expressed RNA interference and neuroscience, Voyager Therapeutics was launched in 2014 with funding from leading life sciences investor Third Rock Ventures and is headquartered in Cambridge, Mass.

Semma Therapeutics was founded to develop transformative therapies for Type 1 diabetes patients. Recent work in the laboratory of Professor Douglas Melton led to the discovery of a method to generate billions of functional, insulin-producing beta cells in the laboratory. This breakthrough technology has been exclusively licensed to Semma Therapeutics for the development of a cell-based therapy for diabetes. Ongoing research at Semma Therapeutics is focused on combining these proprietary cells with a state-of-the-art device to provide a true replacement for the missing beta cells in a diabetic patient without immunosuppression. Semma Therapeutics is working to bring this new therapeutic option to the clinic and improve the lives of patients with diabetes.

WaVe Life Sciences is leading a revolution in nucleic acid therapeutics and was founded on the core principle that medicines should possess precisely controlled molecular structures. We and our strategic partners are advancing a portfolio of stereopure drug candidates that target underlying biology across a wide range of human diseases. WaVe maintains R&D facilities in Boston and Japan and was founded by two world-renowned scientific leaders, Gregory Verdine and Takeshi Wada. Dr. Verdine is Erving Professor of Chemistry in the Harvard University Departments of Stem Cell and Regenerative Biology, Chemistry and Chemical Biology, and Molecular and Cellular Biology. Dr. Wada is a professor at Tokyo University of Science, Faculty of Pharmaceutical Sciences and Department of Medicinal and Life Science.

Senior Whole Health, LLC, a health maintenance organization, administers healthcare plans for Medicaid and Medicare eligible seniors in Massachusetts, New York, and Connecticut. Its healthcare package includes in-home support services, physician and hospital care, prescription drugs, and dental services. Senior Whole Health, LLC was founded in 2003 and is based in Cambridge, Massachusetts with additional offices in Cambridge and Raynham.

Rubius Therapeutics is creating RCTs™ as a new class of medicines. Leading applications include programs in cancer, enzyme deficiency diseases and the induction of tolerance to treat autoimmune disease. The company was conceived and launched by Flagship VentureLabs , the innovation foundry where a team of scientific entrepreneurs systematically evolve enterprising ideas based upon previously undiscovered areas of science into real-world inventions and ventures.

PROMETRIKA is a global full-service clinical research organization (CRO) with extensive experience in clinical operations, data management, biostatistics, medical writing, and pharmacovigilance.

Scholar Rock, Inc. is a biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Our newly elucidated understanding of the molecular mechanisms of growth factor activation enabled us to develop a proprietary platform for the discovery and development of monoclonal antibodies that locally and selectively target these signaling proteins at the cellular level. We believe this approach, acting in the disease microenvironment, avoids the historical challenges associated with inhibiting growth factors for therapeutic effect. We believe our focus on biologically validated growth factors may facilitate a more efficient development path. We are advancing our lead product candidate, SRK-015, a selective first-in-class inhibitor of the activation of the growth factor myostatin in skeletal muscle, into clinical development for the treatment of spinal muscular atrophy, or SMA. We expect to initiate a Phase 1 clinical trial in the second quarter of 2018. Utilizing our proprietary platform, we are also creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including other neuromuscular disorders, cancer, fibrosis and anemia.

Blueprint Medicines is driving the development of personalized, highly-selective cancer therapies that harness the growing understanding of the molecular blueprint of cancer. Using its powerful Insights-to-Validation™ Platform and proprietary chemical library, Blueprint Medicines is working to develop new therapeutic compounds and combination therapies that target the molecular aberrations that cause cancer and the emerging resistance mechanisms that make it increasingly difficult to treat. Founded in 2011 by a proven team of scientists and entrepreneurs with world-renowned expertise in the development of targeted cancer therapies, cancer genomics, and rational drug development, Blueprint Medicines is poised to realize the promise of the cancer data “revolution”: truly personalized therapies that improve outcomes and shift cancer to a manageable condition.

Magenta is focused on improving the patient experience in transplant medicine, with the goal of bringing this lifesaving procedure to more patients. Over the past 50 years, stem cell transplantation has been used in more than 1M patients worldwide. It is currently the only treatment with the potential to cure some of the most devastating hematologic cancers and autoimmune diseases. Despite the increased use and clinical advancements over the past several decades, stem cell transplant remains a high-risk last-resort therapeutic option due to the toxicity and potential long-term side effects.