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Sarepta Therapeutics, Inc. (NASDAQ: SRPT) is a pioneering biopharmaceutical company primarily engaged in the discovery and advancement of RNA-targeted therapeutics. Headquartered in Cambridge, Massachusetts, Sarepta is dedicated to developing innovative treatments for a range of rare, infectious, and other challenging diseases.
Core Focus: Sarepta’s primary focus is on Duchenne Muscular Dystrophy (DMD), a severe type of muscular dystrophy. The company's lead product candidate, eteplirsen, is designed to skip exon 51 and potentially modify the progression of DMD. Alongside eteplirsen, Sarepta is working on other DMD drug candidates that target different exons.
Innovative Technology: The company leverages proprietary RNA-targeted technology platforms to create novel pharmaceuticals. This technology is pivotal in addressing diseases with high unmet medical needs.
Therapeutic Pipeline:
- DMD Program: Focused on rapidly advancing exon-skipping drug candidates.
- Infectious Diseases: Developing treatments for drug-resistant bacteria and other rare infectious diseases.
Financial Condition and Partnerships: Sarepta collaborates with third-party contractors for the manufacturing of its product candidates. This strategic approach allows the company to focus on innovation and development while ensuring efficient production processes. Despite most of its product candidates being in early developmental stages, Sarepta continues to secure funding and partnerships to support its ambitious research and development goals.
For more information, visit their official website at www.sarepta.com.
Sarepta Therapeutics (NASDAQ:SRPT) presented new data from its SRP-9001 program for treating Duchenne muscular dystrophy at 'Micro-dystrophin Day'. Key findings include significant improvements in the North Star Ambulatory Assessment (NSAA) scores: an 8.6-point increase in 4-7 year-olds (Study 101), a 2.9-point increase in 6-7 year-olds (Study 102), and a 3.0-point improvement at six months (Study 103). The EMBARK trial, a pivotal Phase 3 study, aims to enroll 120 patients globally. The safety profile remains consistent, with transient treatment-related adverse events. Sarepta emphasizes the urgency in addressing this degenerative disease.
Sarepta Therapeutics (NASDAQ:SRPT) celebrated the grand opening of its 85,000 square foot Genetic Therapies Center of Excellence in Columbus, Ohio, on October 4, 2021. This facility expands their research capabilities and aims to support their pipeline of genetic medicines, including RNA, gene therapy, and gene editing programs. With 70 employees currently, Sarepta plans to double its workforce by the end of 2022. The center will also enhance collaboration with local partners and bolster Ohio's position in biotechnology.
Sarepta Therapeutics (NASDAQ:SRPT) has announced the initiation of study SRP-9001-301, also known as EMBARK, in collaboration with Roche. This pivotal study aims to evaluate SRP-9001, a gene transfer therapy for Duchenne muscular dystrophy (DMD). The EMBARK study will take place in the US, Europe, and Asia, marking the first pivotal double-blind gene therapy trial for DMD. The SRP-9001 Micro-dystrophin R&D Day is scheduled for October 11, 2021, to present more details about the trial.
Sarepta Therapeutics (NASDAQ:SRPT) announced the granting of equity awards to 43 newly hired employees on September 30, 2021, under its 2014 Employment Commencement Incentive Plan. The total awards include options for 48,350 shares and 24,900 restricted stock units (RSUs), with an exercise price of $92.48 per share. Vesting for options occurs monthly over four years, while RSUs vest annually, both contingent on continued employment. This initiative aims to enhance employee retention and performance within the precision genetic medicine sector.
Sarepta Therapeutics (NASDAQ:SRPT) announced plans to initiate Part B of the MOMENTUM study for SRP-5051, potentially a pivotal trial following positive FDA interactions. This study aims to treat Duchenne muscular dystrophy and will enroll 20-40 patients aged 7 to 21, including both ambulatory and non-ambulatory individuals. Initial results from Part A show SRP-5051's efficacy, boasting significantly higher dystrophin production compared to existing treatments. The protocol for Part B will emphasize safety with magnesium supplementation due to previously noted reversible hypomagnesemia.
Sarepta Therapeutics (NASDAQ:SRPT) will present at the World Muscle Society 2021 Virtual Congress from Sept. 20-24, showcasing its commitment to developing precision genetic medicine for rare diseases. The presentations will cover important data from the company’s gene therapy and RNA platforms and address the prevalence of pre-existing antibodies to the AAVrh74 vector. All presentations will be available on-demand starting Sept. 20, 2021. Sarepta currently leads in therapies for Duchenne muscular dystrophy and has over 40 programs in various development stages.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a leader in precision genetic medicine, announced participation in two upcoming virtual investor conferences. The events include the Morgan Stanley Global Healthcare Conference on Sept. 13, 2021, at 1:15 p.m. E.T., and the Cantor Fitzgerald Virtual Global Healthcare Conference on Sept. 27, 2021, at 1:20 p.m. E.T. Live webcasts will be available on Sarepta's website, and the presentations will be archived for 90 days. The company is committed to developing therapies for rare diseases, focusing on Duchenne muscular dystrophy and limb-girdle muscular dystrophies.
Sarepta Therapeutics (NASDAQ:SRPT) announced the recipients of Route 79, The Duchenne Scholarship Program, on World Duchenne Awareness Day. Fifteen individuals living with Duchenne muscular dystrophy will each receive scholarships of up to $5,000 to support their post-secondary education. The selection was made by an independent committee based on community involvement and personal essays. This program aims to support students with Duchenne in their educational pursuits, reflecting Sarepta's commitment to the Duchenne community.
Sarepta Therapeutics (NASDAQ:SRPT) announced on August 31, 2021, the granting of equity awards to 11 new hires as an employment inducement. The awards consisted of options to purchase 10,600 shares and 5,400 restricted stock units (RSUs). The options have an exercise price of $78.12 per share, matching the closing price on the Grant Date. Vesting for the options and RSUs occurs over four years, subject to continued employment. This initiative aligns with Nasdaq rules and reflects the company’s commitment to attracting talent in the rare disease sector.
Sarepta Therapeutics (NASDAQ:SRPT) reported second-quarter 2021 net product sales of $141.8 million, a 27% increase year-over-year. The company raised its full-year revenue guidance by nearly $30 million to between $565 million and $575 million. Following a successful end-of-phase-2 meeting with the FDA, Sarepta plans to initiate its pivotal trial for SRP-9001 in September 2021. The company also executed a licensing agreement for a gene therapy program targeting limb-girdle muscular dystrophy type 2A, underscoring its ongoing commitment to advancing genetic medicine.
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