Sarepta looks for the best and brightest partners to help us transform 21st century healthcare. We have a long history of collaborative strategic partnerships with top institutions and companies from around the world—all in service to accelerate our mission to rescue lives otherwise impacted or stolen by rare disease.
Created by the AFM-Telethon and located in Evry (France), Genethon is a non-profit organization that has one of the largest clinical and research groups dedicated to developing biotherapies for rare genetic diseases. Genethon specializes in the discovery and development of gene therapy drugs and has several ongoing clinical, preclinical and research programs for genetic diseases of muscle, blood, immune system and liver. Sarepta and Genethon are working jointly to develop treatments for Duchenne muscular dystrophy (DMD), using a micro-dystrophin gene therapy approach.
Lacerta is an AAV gene therapy company focused on developing novel AAV gene therapies for diseases and conditions affecting the central nervous system (CNS). Lacerta’s portfolio comprises fundamental AAV technology developed by seminal investigators at the University of Florida. In addition to Sarepta’s equity investment in Lacerta, this partnership also grants Sarepta exclusive rights to use Lacerta’s proprietary capsids with a scalable AAV manufacturing platform to develop novel gene therapies for certain lysosomal storage diseases.
Lysogene is a Paris-based pioneer in gene therapy focused on rare CNS diseases, including its lead program to treat MPS IIIA, which is partnered with Sarepta. Lysogene’s focus on MPS IIIA is driven by a specific and very compelling purpose: Ornella, the daughter of Lysogene’s founder Karen Aiach, was diagnosed with MPS IIIA in 2005. With no biomedical experience, Karen set out to find a treatment. She funded research, connected with scientists and raised capital leading to the launch of the Company in 2009.
The collaboration between Roche and Sarepta combines Sarepta’s leading gene therapy candidate for Duchenne muscular dystrophy (DMD) with Roche’s global reach, commercial presence and regulatory expertise to support the companies’ collective goal of accelerating access to SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), Sarepta’s investigational gene therapy for DMD, to patients outside the United States.
In addition to financial terms, Roche and Sarepta will equally share global development expenses and Sarepta retains all rights to SRP-9001 in the United States.
StrideBio possesses an innovative and proprietary platform that is enabled by a deep knowledge of AAV structure and a unique approach to engineering capsids with novel functions. The Company was co-founded by Mavis Agbandje-McKenna, PhD, and Aravind Asokan, PhD, leading scientists in the field of AAV biology and gene therapy. StrideBio has created a growing portfolio of engineered AAV capsids evolved in non-human primates that demonstrate novel properties including reduced seroprevalence and improved tropism to targeted tissues. Sarepta and StrideBio’s collaboration and license agreement grants Sarepta an exclusive license to StrideBio’s novel, structure-driven capsid technology for 4 targets (NPC1, UBE3A, SCN1A and MECP2), with an option to select up to 4 additional central nervous system and neuromuscular targets.
Sarepta is collaborating with Howard Worman, MD at Columbia University to develop an AAV gene therapy for X-linked Emery-Dreifuss muscular dystrophy, a life-limiting and often life-ending rare neuromuscular disease that affects skeletal and cardiac muscle. Dr. Worman is a leading expert in the study of the nuclear envelope and its role in the pathology of Emery-Dreifuss muscular dystrophy, and his group will be testing novel gene therapy vectors engineered by Sarepta’s Gene Therapy Center of Excellence in Columbus, Ohio.
Sarepta’s gene editing program for Duchenne muscular dystrophy (DMD) was initiated through a partnership with Duke University’s Department of Biomedical Engineering. The CRISPR/Cas9 program, developed in the lab of Charles A. Gersbach, PhD, focuses on the underlying premise of restoring dystrophin expression by removing or “excising” mutated exons from the dystrophin gene. This strategy could potentially enable a treatment for a majority of patients with DMD, as well as opening the door to restoration of full-length dystrophin protein.
Sarepta has partnered with France Piétri-Rouxel, PhD (U974 INSERM -Sorbonne Université) at the Institute of Myology to explore the potential beneficial effects of combining the Company’s PMO technology and micro-dystrophin gene therapy. Dr. Piétri-Rouxel’s group is focused on characterizing skeletal muscle pathophysiology and development of gene therapies for DMD. She has pioneered the effort toward studying the combined effect of these novel genetic medicines in her lab at the Pitié Salpêtrière Hospital. The Institute of Myology is one of the largest research and clinical centers dedicated to myology and neuromuscular disorders.
The Abigail Wexner Research Institute (AWRI) at Nationwide Children’s Hospital in Columbus, Ohio, is a world leader in gene therapy having developed research programs for Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD), which provide the technological underpinnings for Sarepta’s gene therapy engine. In addition to its micro-dystrophin gene therapy programs for DMD and 5 subtypes of LGMD, Sarepta has rights to gene therapy programs for LGMD type 2A and CMT1A, also developed at AWRI at Nationwide Children’s.
AWRI at Nationwide Children’s is one of the largest pediatric research centers in the United States and one of the leading recipients of funding from the National Institutes of Health.
Sarepta has executed multiple strategic partnerships with preeminent investigators at the University of Florida College of Medicine. Lee Sweeney, PhD, has pioneered the development of a novel gene therapy for the treatment of certain heart diseases, including cardiomyopathies. This cardiomyopathy program, which has shown success in a canine model of Duchenne muscular dystrophy (DMD), has been exclusively partnered with Sarepta. Additionally, Brad Hoffman, PhD, has developed an innovative approach to treat multiple sclerosis (MS), the most common immune-mediated disorder affecting the central nervous system. Dr. Hoffman’s program, which is exclusively partnered with Sarepta, utilizes AAV gene therapy to alter the autoimmune response to reduce the severity of MS disease.
Sarepta has executed multiple strategic partnerships with University of Massachusetts Medical School focused in the field of AAV gene therapy. Given the complex nature of the etiology of Rett Syndrome, Sarepta has partnered with a diverse team of renowned UMass investigators spanning multiple critical disciplines in order to develop an AAV gene therapy for this rare, fatal brain disease. Miguel Sena Esteves, PhD, a leading investigator in developing CNS gene therapies; Guangping Gao, PhD, a world expert in AAV vector discovery and engineering; and Michael Green, MD, PhD, a preeminent leader in understanding the molecular basis of regulating gene expression, are working together with Sarepta to develop a precisely regulated expression cassette to safely and effectively treat Rett syndrome. Sarepta also has an additional collaboration with Dr. Gao focused on characterizing a library of novel, human-derived AAV capsids.
Sarepta has an exclusive, worldwide licensing agreement with the University of Western Australia (UWA) for intellectual property rights to support the development of exon-skipping drug candidates for the treatment of Duchenne muscular dystrophy (DMD). The agreement grants Sarepta rights to UWA's extensive patent portfolio in DMD and enables the Company to expand its exon-skipping pipeline with new candidates to address the majority of patients with DMD worldwide.