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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) will announce its first quarter 2021 financial results after market close on May 5, 2021. A conference call to discuss these results and provide a corporate update will follow at 4:30 p.m. E.T. Interested parties can access the call via domestic and international numbers provided in the release, with a live webcast available on Sarepta's website. Sarepta focuses on precision genetic medicine for rare diseases, notably Duchenne muscular dystrophy, and has over 40 development programs in its pipeline.
Sarepta Therapeutics (Nasdaq:SRPT) received notice of an unsolicited mini-tender offer from TRC Capital to purchase up to 2 million shares at $68.50 each, about 4.6% below the stock's closing price on April 16, 2021. Sarepta does not endorse this offer and advises shareholders against tendering their shares, citing the below-market price and lack of affiliation with TRC Capital. The offer represents 2.52% of Sarepta's outstanding shares and is set to expire on May 18, 2021. Sarepta emphasizes caution and recommends consulting with financial advisors.
Sarepta Therapeutics (NASDAQ:SRPT) has launched the Route 79, The Duchenne Scholarship Program, now accepting applications for the 2021-2022 academic year. Up to 15 scholarships, each worth $5,000, will be awarded to individuals diagnosed with Duchenne muscular dystrophy, assessed by an independent committee based on various criteria. Over 50 scholarships have been granted in previous years, showcasing the resilience and ambition of the recipients. Applications are open until May 11, 2021, with recipients notified by August.
Sarepta Therapeutics (SRPT) reported promising two-year data from its ongoing study of SRP-9003, a gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). Key findings include a mean beta-sarcoglycan protein expression of 54% at 24 months in the low-dose cohort, up from 36% at Day 60, and a sustained mean NSAD score improvement of 5.7 points from baseline. Results indicate continued safety and tolerability. Both cohorts showed significant functional improvements, suggesting SRP-9003’s potential as an effective treatment for this severe genetic disorder.
Sarepta Therapeutics (NASDAQ:SRPT) presented ten abstracts, including four podium presentations, at the MDA Annual Clinical and Scientific Conference from March 15-18, 2021. The research focuses on advancing genetic medicine for rare neuromuscular diseases like Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD). Key highlights include new data from gene therapies SRP-9001 and SRP-9003, showing promising results in clinical trials. Presentations are available on-demand, emphasizing the company's commitment to improving treatment outcomes for patients with rare diseases.
Sarepta Therapeutics reported strong financial results for Q4 and full-year 2020, with net product sales reaching $122.6 million and $455.9 million, respectively. This marks a 23% increase in Q4 sales compared to 2019 and a nearly 20% increase for the year. A key highlight is the FDA approval of AMONDYS 45 for Duchenne muscular dystrophy, enhancing treatment options for 8% of patients with specific mutations. The firm is advancing its pipeline with significant developments in gene therapy and RNA platforms, aiming to address over 80% of Duchenne mutations.
Sarepta Therapeutics (NASDAQ:SRPT) announced the grant of equity awards to two new employees on February 26, 2021, as per the Compensation Committee's approval. The awards include options for 1,820 shares of common stock at an exercise price of $87.06, equal to the closing price on the grant date, and 680 restricted stock units. Vesting is set to occur over four years, contingent upon continued employment. Sarepta focuses on precision genetic medicine for rare diseases, particularly in Duchenne muscular dystrophy and gene therapies.
Sarepta Therapeutics (NASDAQ: SRPT) has received FDA approval for AMONDYS 45 (casimersen), its third RNA exon-skipping treatment for Duchenne muscular dystrophy (DMD), specifically for patients with exon 45 amenable mutations. This approval is based on statistically significant increases in dystrophin production in treated patients. Commercial distribution will start immediately, with ongoing confirmatory trials expected to conclude in 2024. The treatment carries a potential risk of kidney toxicity, necessitating close monitoring of kidney function.
Sarepta Therapeutics, a leader in precision genetic medicine, announced participation in two upcoming virtual investor conferences. Management will speak at the Cowen 41st Annual Health Care Conference on Mar. 3, 2021, at 2:40 p.m. E.T., and at the Barclays Global Healthcare Conference on Mar. 10, 2021, at 2:25 p.m. E.T.. Both presentations will be available via live webcasts on Sarepta's website and archived for 90 days. The company focuses on genetic treatments for rare diseases, including Duchenne muscular dystrophy, with over 40 development programs.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT) will disclose its fourth quarter and full-year 2020 financial results on March 1, 2021, after the Nasdaq closes. A conference call will follow at 4:30 p.m. E.T. to discuss the results and provide a corporate update. Investors can access the call at (844) 534-7313 for domestic or (574) 990-1451 for international, using passcode 9672289. Sarepta specializes in precision genetic medicine, particularly in DMD and gene therapies for various rare diseases, with over 40 programs in development.