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.
AavantiBio was co-founded by renowned gene therapy researchers Barry Byrne, M.D., Ph.D., and Manuela Corti, P.T., Ph.D., from University of Florida's renowned Powell Gene Therapy Center. The Company’s lead program is focused on Friedreich’s Ataxia (FA), a rare inherited genetic disease that causes cardiac and central nervous system dysfunction. In October 2020, Sarepta, along with a syndicate of highly regarded life sciences investors, participated in a $107 million Series A financing for AavantiBio. Sarepta’s head of gene therapy, Dr. Louise Rodino-Klapac, serves as an AavantiBio Board Observer.
Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experimentation to gene therapy. AI and machine learning technologies have the potential to deliver enhanced vectors for gene therapies and Dyno’s proprietary platform uses AI technology for the design of novel capsids, the cell-targeting protein shell of viral vectors, that could offer improved muscle targeting and immune-evading properties, in addition to advantages in packaging and manufacturing. Through this agreement, Dyno’s platform will be used to develop next-generation adeno-associated virus (AAV) vectors for muscle diseases. Dyno is responsible for the design and discovery of novel AAV capsids with improved functional properties and Sarepta is responsible for conducting preclinical, clinical and commercialization activities for any gene therapy product candidates identified through the collaboration.
Genethon, a non-profit organization, has one of the largest clinical and research groups dedicated to developing biotherapies for rare genetic diseases. Sarepta and Genethon are working jointly to develop treatments for Duchenne muscular dystrophy (DMD), using a micro-dystrophin gene therapy approach. Unique among biotech companies, Genethon was launched in 1990 with funding from the French Muscular Dystrophy Association (AFM Telethon), which continues to provide significant financial support to the organization towards developing biotherapies for rare neuromuscular diseases.
Genevant Sciences is a leading nucleic acid delivery company with world-class platforms and the industry’s most robust and expansive lipid nanoparticle (LNP) patent estate. LNPs offer the potential for a non-viral approach to gene editing and can provide both optimal uptake into desired cells and efficient release, resulting in functional delivery of gene editing cargo, such as CRISPR-Cas, to target tissues. Under the terms of the agreement, Genevant will design and collaborate with Sarepta in the development of muscle targeted LNPs to be applied to gene editing targets in early stage development. Sarepta has options for an exclusive license to Genevant’s LNP technology for up to four neuromuscular indications, including Duchenne muscular dystrophy.
Hansa Biopharma is the leader in immunomodulatory enzyme technology for rare immunoglobulin G (IgG) mediated diseases and the developer of imlifidase, an antibody-cleaving enzyme that specifically cleaves IgG and inhibits an IgG-mediated immune response. This agreement aims to evaluate whether pre-treatment with imlifidase will allow for administration of Sarepta’s AAV-based gene therapies to treat patients with Duchenne and LGMD who would otherwise have been ineligible for treatment because of pre-existing neutralizing antibodies.
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.
Selecta Biosciences, Inc. is a clinical-stage biotechnology company leveraging its clinically validated platform to develop tolerogenic therapies that selectively mitigate unwanted immune responses which has generated strong preclinical evidence to support the potential for redosing patients receiving adeno-associated virus (AAV) based gene therapies. This agreement allows Sarepta to proactively explore the potential of using Selecta’s platform to inhibit immune responses to the AAV vectors used to deliver investigational gene therapies in Duchenne and certain Limb-girdle muscular dystrophies and pave the path to redosing, if it is needed.
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.
In this strategic collaboration Sarepta is working with UF Innovate, the technology commercialization arm of the University of Florida (UF), to enable leading-edge research for novel genetic medicines. Through the agreement, Sarepta will fund multiple research programs at UF, and will have an exclusive option to further develop any new therapeutic compounds that result from the funded research programs. Under this agreement, funding has been allocated for projects that include exploratory research in novel gene therapy vectors, next-generation capsids and gene editing technologies as well as work in new therapeutic areas in degenerative genetic diseases.
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.
Through a collaboration with researchers in Harvard University’s Department of Stem Cell and Regenerative Biology, Sarepta has strengthened its commitment to developing a durable gene editing therapy that would be available to a majority of DMD patients. Pre-clinical research led by Prof. Amy Wagers at Harvard, supported by Sarepta, combines cutting-edge CRISPR/Cas9 genome editing technologies with a deep knowledge of stem cell science and regenerative biology. The approach pursued by her lab, if successful, might offer a therapeutic strategy to permanently restore muscular function.
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.
Sarepta has a long-standing collaboration with Murdoch University in Perth, Western Australia, which has led to the establishment of the Sarepta Translational Laboratory. The collaboration is led by Murdoch University professors, Steve Wilton, PhD, and Sue Fletcher, PhD. The work in the Translational Laboratory includes exploring the applicability of the Sarepta's phosphorodiamidate morpholino oligomer (PMO) technology for treating rare genetic diseases. As part of the collaborative agreement, Sarepta will have exclusive option to license technology and/or products resulting from research on the PMO technology in the Translational Laboratory.
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 partnered with a renowned group of investigators at the University of Minnesota with the goal of advancing the understanding of dystrophin structure and function. Dr. Jim Ervasti, a leading expert on the structure and cellular function of the dystrophin glycoprotein complex, is collaborating with Dr. Joseph Muretta to leverage a novel fluorescence-based high throughput screening method developed by Dr. David Thomas. Combining their respective expertise in cell biology and biophysics could lead to new insights relevant to the critical functional domains within the dystrophin protein.
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.