Sarepta Therapeutics is at the forefront of developing innovative genetic medicines aimed at treating rare diseases, particularly those affecting muscle function. The company employs a multifaceted approach that includes gene therapy, RNA technologies, and gene editing. Their gene therapy engine is designed to create targeted therapies for conditions such as Duchenne muscular dystrophy and limb-girdle muscular dystrophies. This engine utilizes adeno-associated virus (AAV) vectors to deliver therapeutic genes, which are essential for producing functional proteins that are often deficient or absent in patients with these genetic disorders. By carefully selecting the appropriate vectors, promoters, and transgenes, Sarepta aims to optimize the expression of these proteins in specific tissues, thereby enhancing the efficacy of their treatments.

In addition to gene therapy, Sarepta leverages RNA technologies, particularly phosphorodiamidate morpholino oligomers (PMOs) and their next-generation counterparts, peptide PMOs (PPMOs). These RNA-based therapies are designed to address genetic mutations by facilitating exon skipping, which allows for the production of shortened but functional proteins, such as dystrophin in the case of Duchenne muscular dystrophy. The company is committed to advancing its pipeline of investigational therapies through rigorous clinical trials, with the goal of transforming the lives of patients affected by rare diseases. By continuously innovating and expanding their therapeutic portfolio, Sarepta Therapeutics is dedicated to making significant strides in the field of precision genetic medicine.

United States

Sarepta Therapeutics, headquartered in Cambridge, Massachusetts, is a global biotechnology company on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing.

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Genetic Diseases

Musculoskeletal Diseases

Sarepta Therapeutics

Duchenne muscular dystrophy

SRP-9001 is an investigational gene transfer therapy intended to deliver &nbsp;the micro-dystrophin-encoding gene to muscle tissue for the targeted &nbsp;production of the micro-dystrophin protein. Sarepta is responsible for &nbsp;global development and manufacturing for SRP-9001 and plans to &nbsp;commercialize SRP-9001 in the United States upon receiving FDA approval. &nbsp;In December 2019, Roche partnered with Sarepta to combine Roche’s &nbsp;global reach, commercial presence and regulatory expertise with &nbsp;Sarepta’s gene therapy candidate for Duchenne to accelerate access to &nbsp;SRP-9001 for patients outside the United States. Sarepta has exclusive &nbsp;rights to the micro-dystrophin gene therapy program initially developed &nbsp;at the Abigail Wexner Research Institute at Nationwide Children’s &nbsp;Hospital.
Gene therapy

ELEVIDYS (SRP-9001)

Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein, which was evaluated in Study 2 and Study 3.&nbsp;

EXONDYS 51 (eteplirsen)

Golodirsen is designed to bind to exon 53 of dystrophin pre-mRNA resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon 53 skipping is intended to allow for production of an internally truncated dystrophin protein in patients with genetic mutations that are amenable to exon 53 skipping.&nbsp;

VYONDYS 53 (golodirsen) 

AMONDYS 45 (casimersen) (SRP-4045)

Limb-Girdle Muscular Dystrophy

SRP-9003 (bidridistrogene xeboparvovec) is an investigational gene therapy that uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat peripheral neuromuscular diseases. SRP-9003 also uses the MHCK7 promoter, chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary or cardiac complications.
Gene therapy

SRP-9003 (bidridistrogene xeboparvovec) 

SRP-5051 is an investigational agent using Sarepta’s PPMO chemistry and &nbsp;exon-skipping technology to skip exon 51 of the dystrophin gene. &nbsp;SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA, &nbsp;resulting in exclusion of this exon during mRNA processing in patients &nbsp;with genetic mutations that are amenable to exon 51 skipping. Exon &nbsp;skipping is intended to allow for production of an internally truncated &nbsp;dystrophin protein. PPMO is Sarepta’s next-generation chemistry platform &nbsp;designed around a proprietary cell-penetrating peptide conjugated to &nbsp;the PMO backbone, with the goal of increasing tissue penetration, &nbsp;increasing exon skipping and significantly increasing dystrophin &nbsp;production. Around 13% of DMD patients have mutations which make them &nbsp;amenable to skipping exon 51. If successful, the PPMO offers the &nbsp;potential for improved efficacy and less frequent dosing for patients.

SRP-5051 (vesleteplirsen)

Dysferlinopathy

SRP-6004 is Sarepta’s investigational dual-vector gene therapy approach for treating dysferlinopathy and is designed to express the full-length protein dysferlin that is missing in individuals with LGMD2B/R2.

Sarepta Therapeutics

Company Details