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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) has secured an exclusive license for a gene therapy candidate, CAPN-3, aimed at treating Limb-girdle muscular dystrophy type 2A (LGMD2A). This type is the most prevalent LGMD, comprising nearly a third of cases. The CAPN-3 program follows promising preclinical studies conducted by Nationwide Children's Hospital, demonstrating early proof of concept. Sarepta's gene therapy portfolio is robust, addressing six LGMD subtypes, which together account for over 70% of known LGMDs. The AAVrh74 vector used in these therapies can effectively deliver treatments to key muscle groups.
Sarepta Therapeutics (NASDAQ:SRPT) announced the granting of equity awards to 10 new employees as a part of their employment incentives on July 30, 2021. These awards include options to purchase 20,450 shares and 10,400 restricted stock units (RSUs), approved under the 2014 Employment Commencement Incentive Plan and Nasdaq Rule 5635(c)(4). The options have an exercise price of $67.78 per share, and the vesting schedule spans four years. Sarepta continues to lead in precision genetic medicine for rare diseases, with over 40 programs in development.
Sarepta Therapeutics (NASDAQ:SRPT) will announce its second quarter 2021 financial results after market close on August 4, 2021. A conference call is scheduled for 4:30 p.m. E.T. to discuss these results and provide a corporate update. Investors can access the call via phone or through the company's website, which will also archive the event for 90 days. Sarepta is focused on developing precision genetic medicine for rare diseases, particularly in Duchenne muscular dystrophy, with a robust pipeline of over 40 programs.
Sarepta Therapeutics (NASDAQ:SRPT) has granted equity awards to two new employees as part of its compensation strategy. Approved on June 30, 2021, these awards include options to purchase 2,450 shares and 1,250 restricted stock units (RSUs). The options have an exercise price of $77.74 per share, equivalent to the closing price on the grant date. The vesting schedule includes a one-year cliff, with full vesting over four years, contingent on continued employment. This move aligns with Nasdaq Listing Rule 5635(c)(4) and highlights Sarepta's commitment to attracting top talent.
Sarepta Therapeutics (NASDAQ:SRPT) announced participation in two virtual investor conferences. The management will take part in:
- Goldman Sachs 42nd Annual Global Healthcare Conference on June 10 at 1:20 p.m. E.T.
- BofA Securities 2021 Napa BioPharma Virtual Conference on June 16 at 10:30 a.m. E.T.
Both presentations will be webcast live on Sarepta's website and archived for 90 days. Sarepta focuses on precision genetic medicine and has over 40 programs targeting rare diseases, notably Duchenne muscular dystrophy.
Sarepta Therapeutics (NASDAQ:SRPT) announced on May 28, 2021, that it granted equity awards to six new employees as part of its 2014 Employment Commencement Incentive Plan. The awards include options to purchase 6,295 shares at an exercise price of $75.65 per share and a total of 2,950 restricted stock units (RSUs). The options will vest over four years, while the RSUs will also fully vest by the fourth anniversary of the grant date. This step aligns with Nasdaq Listing Rule 5635(c)(4), emphasizing Sarepta's commitment to attracting talent in precision genetic medicine for rare diseases.
Sarepta Therapeutics (NASDAQ:SRPT) announced positive results from the first 11 participants in Study SRP-9001-103 ENDEAVOR. The study shows robust transduction with mean vector genome copies of 3.87 per nucleus and micro-dystrophin expression levels at 55.4% of normal. Proper localization of micro-dystrophin was confirmed, with 70.5% dystrophin-positive fibers. The safety profile remains consistent with previous studies, with no new safety signals. These findings support the potential of SRP-9001 as a transformative therapy for Duchenne muscular dystrophy, leading to plans for an FDA meeting to start a registrational study.
Sarepta Therapeutics (NASDAQ:SRPT) will host a conference call on May 18, 2021, at 8:30 am ET, to share results from the SRP-9001-103 clinical trial, also known as ENDEAVOR. This trial evaluates SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) for treating Duchenne muscular dystrophy. It marks the first use of commercially representative material in a clinical setting. The event will be webcast live and archived for a year.
Sarepta Therapeutics (NASDAQ:SRPT) reported first-quarter 2021 net product sales of $124.9 million, a 24% increase from the previous year. The company launched its third RNA therapy for Duchenne, AMONDYS 45™, shortly after FDA approval. Significant advancements in pipeline development were noted, particularly with SRP-5051, which showed an 18x increase in exon skipping compared to a standard treatment. However, the company reported a GAAP net loss of $167.3 million, reflecting increased R&D expenses of $195.1 million. Cash reserves decreased from $1.9 billion to $1.7 billion.
Sarepta Therapeutics (NASDAQ:SRPT) announced its participation in two upcoming virtual investor conferences. Management will engage in fireside chats at the BofA Securities Virtual Healthcare Conference on May 11, 2021, at 2:00 p.m. E.T., and the RBC 2021 Global Healthcare Conference on May 18, 2021, at 2:30 p.m. E.T. Both presentations will be webcast live and archived on Sarepta’s website for 90 days. Sarepta is dedicated to precision genetic medicine for rare diseases, holding a leadership position in Duchenne muscular dystrophy and limb-girdle muscular dystrophies.
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