Sarepta Therapeutics

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legal form Incorporated
founding 1980 (as AntiVirals ), 2012 (renamed Sarepta )
Seat Cambridge , Massachusetts , United States
United StatesUnited States 
management Douglas S. Ingram, CEO
sales $ 155.0 million (2017)
Branch pharmacy

Sarepta Therapeutics is an American medical research company that was originally founded in 1980 under the company name AntiVirals and renamed Sarepta in 2012. Sarepta is headquartered in Cambridge , MA . The European headquarters are in Zug , Switzerland . Subsidiaries were also established in Germany, Italy, Great Britain, Ireland, Sweden and Brazil in 2018. Sarepta is listed on the American technology exchange NASDAQ under the label SRPT.

Sarepta is dedicated to the discovery and development of precisely effective gene therapeutics ("precision genetic medicines") for the treatment of rare neuromuscular diseases . First and foremost, Sarepta is driving the development of its drug candidates for the treatment of Duchenne muscular dystrophy (DMD).

Field of activity

A central area of ​​indications for the gene therapeutics developed by Sarepta is currently DMD, a hereditary muscular disease in which the muscle structure protein dystrophin is missing.

Some diseases - such as B. Many neuromuscular diseases - can be caused by missing or defective proteins . Proteins are important components e.g. B. the cell structure or of enzymes . The protein synthesis takes place intracellularly to the ribosomes . Here, the information stored in the DNA is first “transcribed” into the corresponding mRNA templates (mRNA: messenger RNA ) if necessary . These are then processed by the ribosomes and “translated” into the respective proteins. A defective DNA or mRNA can lead to the production of defective proteins or even to a breakdown of the respective protein synthesis - such as B. at the DMD. The underlying therapeutic goal here is to (re) enable the corresponding protein biosynthesis.

Medication / pipeline

Sarepta's pipeline currently has the most drug candidates in the DMD area. The most advanced are mRNA-targeted drugs. mRNA-targeted drugs are one way to at least partially remedy the lack of the protein dystrophin in patients with DMD. These can e.g. For example, they can be designed in such a way that they can partially or even completely correct the faulty production or the lack of a protein involved in a disease and thus contribute to a delay in the progression of the disease. Due to their effect on the genetic level, mRNA-targeted active substances have the potential to treat certain diseases that could otherwise not be treated in this form with conventional low-molecular or biological drugs. A typical example of this is the treatment of DMD, in which the synthesis of a shortened form of dystrophin is made possible via mRNA-targeted active substances.

The most advanced mRNA-targeted active ingredients in the Sarepta pipeline are based on a proprietary phosphorodiamidate-morpholino-oligomer (PMO) chemistry platform, which is performed by means of "exon skipping" (= skipping or "skipping" of certain, mostly defective sections) of the mRNA) is aimed at an individual correction of the respective mRNA. In the case of DMD, this approach is intended to achieve at least partial production of the missing protein dystrophin. In contrast to earlier approaches of exon skipping in DMD, PMOs have a neutral phosphate bond, which, according to previous findings, results in only low toxicity and only minor side effects associated with it.

The following PMO drugs and active ingredients are currently in the late stage of clinical development at Sarepta:

  • Eteplirsen / Exondys 51 / AVI-4648 ( Phase III / IV ): approved in September 2016 under the trade name Exondys 51 in the USA in an accelerated approval process ( fast track ) as the first drug for the causal treatment of DMD for patients with a corresponding mutation , which includes exon 51. Approval was refused in Europe.
  • Golodirsen / Yvondis53 / SRP-4053 (phase III or IV in the USA): intended for exon 53 skipping; an application in the USA for approval for the treatment of DMD in the case of mutations including exon 53 was granted in December 2919.
  • Casimersen / SRP-4045 (Phase III): intended for exon 45 skipping; under development for the treatment of DMD in the presence of mutations including exon 45.

The protein-phosphorodiamidate-morpholino-oligomers (PPMOs) represent a further development of the PMOs. These are PMOs linked to an additional protein, which thereby achieve an improved affinity for certain substances.

In addition to PMOs and PPMOs, Sarepta develops numerous other substances in the field of gene therapy (e.g. AAVrh74.MHCK7.Micro-dystrophin, rAAVrh74. MCK.GALGT2, AAV2 / 8-Micro-dystrophin-1) and genome editing (e.g. . CRISPR / CAS9 ) - mostly in cooperation with well-known academic research institutions and organizations.

In June 2018, on its first R&D Day , Sarepta presented a detailed overview of its pipeline as well as initial, preliminary, but promising data on gene therapy for DMD with AAVrh74.MHCK7.Micro-Dystrophin in the treatment of three boys (phase I / II) Prof. Jerry Mendell's research group at Nationwide Children's Hospital. At the 23rd meeting of the World Muscle Society in Mendoza in September 2018, further results from this study were presented, which now also included a fourth patient. In December 2019 Roche announced that it would purchase the rights to SRP-9001 micro-dystrophin.

Sarepta's pipeline also includes drug candidates for the indications of limb girdle dystrophy , Charcot-Marie-Tooth neuropathies and mucopolysaccharidosis .


There is an intensive collaboration between Sarepta in the area of ​​gene therapy for DMD and the research group of Prof. Jerry Mendel from the Nationwide Children's Hospital in Columbus OH, USA.

In addition, Sarepta is working with the Skip-NMD patient organization on the development of Golodirsen (SRP-4053).

In June 2017, Sarepta Therapeutics also signed a collaboration with Genethon , a non-profit R&D organization from France dedicated to developing biotherapies for rare genetic diseases from research to clinical validation.

In June 2018, a cooperation agreement with the company Myonexus followed, which, under the direction of Prof. Louise Rodino-Klapac, deals with the development of therapy options for limb girdle muscular dystrophy (LGMD). Further collaborations were concluded for the indications Pompe , Charcot-Marie-Tooth neuropathies and mucopolysaccharidosis.

See also

Web links

Individual evidence

  1. a b c d e f g FAQs , Company WebSite, accessed on June 29, 2018
  2. Senior Leadership , Company WebSite, accessed June 29, 2018
  3. ^ Revenue & Earnings , Company WebSite, accessed June 29, 2018
  4. ^ For Investors , Company WebSite, accessed June 29, 2018
  5. Sarepta Therapeutics, Inc. Real-Time Quotes, NASDAQ WebSite, accessed June 29, 2018
  6. a b c Our Pipeline , Company WebSite, accessed January 2, 2019
  7. FDA grants accelerated approval to first drug for Duchenne muscular dystrophy , PM FDA, September 19, 2016, accessed January 2, 2019
  8. Rejected human medicinal products in the EU Community Register , accessed on March 5, 2019.
  9. Exondys , EMA - EPAR, accessed January 2, 2019
  10. FDA grants accelerated approval to first targeted treatment for rare Duchenne muscular dystrophy mutation , PM FDA dated December 12, 2019, accessed January 15, 2020
  11. Sarepta Therapeutics Announces Plan to Submit a New Drug Application (NDA) for Accelerated Approval of Golodirsen (SRP-4053) in Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Skipping Exon 53 , PM Sarepta, March 12, 2018, accessed March 11, 2018 January 2019
  12. 2018: A Year of Transformation , presentation January 8, 2018, accessed January 2, 2019
  13. Sarepta Therapeutics Announces that at its First R&D Day, Jerry Mendell, MD Presented Positive Preliminary Results from the First Three Children Dosed in the Phase 1 / 2a Gene Therapy Micro-dystrophin Trial to Treat Patients with Duchenne Muscular Dystrophy , PM Sarepta vom 19. June 2018, accessed July 4, 2018
  14. Roche enters licensing agreement with Sarepta Therapeutics to improve the lives of patients living with Duchenne muscular dystrophy , PM Roche of December 23, 2019, accessed on January 15, 2020
  15. Partnerships , Company WebSite, accessed January 11, 2019