SRPT Sarepta Therapeutics Inc.

140.43
-0.85  -1%
Previous Close 141.28
Open 141.65
52 Week Low 73.72
52 Week High 175
Market Cap $11,024,515,428
Shares 78,505,415
Float 66,342,019
Enterprise Value $9,947,224,428
Volume 387,396
Av. Daily Volume 724,107
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Upcoming Catalysts

Drug Stage Catalyst Date
SRP-5051
Duchenne muscular dystrophy
Phase 1/2
Phase 1/2
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Casimersen
Duchenne muscular dystrophy
PDUFA priority review
PDUFA priority review
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SRP-9001
Duchenne muscular dystrophy
Phase 2
Phase 2
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Drug Pipeline

Drug Stage Notes
SRP-9003
Limb-girdle Muscular Dystrophy Type 2E (LGMD2E)
Phase 1/2
Phase 1/2
Phase 1/2 18-month functional results from Cohort 1 presented September 28, 2020.
LYS-SAF302
Mucopolysaccharidosis type IIIA (MPS IIIA)
Phase 2/3
Phase 2/3
Phase 2/3 dosing to be completed mid-2020.
rAAVrh74.MCK.GALGT2
Duchenne muscular dystrophy
Phase 1/2
Phase 1/2
Phase 1/2 ongoing.
MYO-102
Duchenne muscular dystrophy - LGMD2D
Phase 1/2
Phase 1/2
Phase 1/2 complete.
Golodirsen - Exon 53
Duchenne muscular dystrophy
Approved
Approved
FDA Approval announced December 12, 2019.
MYO-201
Duchenne muscular dystrophy - (LGMD2B)
Phase 1
Phase 1
Phase 1 trial ongoing.
Eteplirsen - Exon 51
Duchenne muscular dystrophy
Approved
Approved
Approved September 19, 2016.

Latest News

  1. CAMBRIDGE, Mass., Sept. 30, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, granted equity awards on September 30, 2020 that were previously approved by the Compensation Committee of its Board of Directors under Sarepta's 2014 Employment Commencement Incentive Plan, as a material inducement to employment to 37 individuals hired by Sarepta in September 2020. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received, in the aggregate, options to purchase 38,960 shares of Sarepta's common stock, and in the aggregate, 15,460 restricted stock units ("RSUs"). The options have an exercise price of $140.43 per share, which…

    CAMBRIDGE, Mass., Sept. 30, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, granted equity awards on September 30, 2020 that were previously approved by the Compensation Committee of its Board of Directors under Sarepta's 2014 Employment Commencement Incentive Plan, as a material inducement to employment to 37 individuals hired by Sarepta in September 2020. The equity awards were approved in accordance with Nasdaq Listing Rule 5635(c)(4).

    The employees received, in the aggregate, options to purchase 38,960 shares of Sarepta's common stock, and in the aggregate, 15,460 restricted stock units ("RSUs"). The options have an exercise price of $140.43 per share, which is equal to the closing price of Sarepta's common stock on September 30, 2020 (the "Grant Date"). One-fourth of the shares underlying each employee's option will vest on the one-year anniversary of the Grant Date and thereafter 1/48th of the shares underlying each employee's option will vest monthly, such that the shares underlying the option granted to each employee will be fully vested on the fourth anniversary of the Grant Date, in each case, subject to each such employee's continued employment with Sarepta on such vesting dates.

    One-fourth of the RSUs will vest yearly on each anniversary of the Grant Date, such that the RSUs granted to each employee will be fully vested on the fourth anniversary of the Grant Date, in each case, subject to each such employee's continued employment with Sarepta on such vesting date.

    About Sarepta Therapeutics

    At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company's programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

    Internet Posting of Information

    We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.  

    Source: Sarepta Therapeutics, Inc.

    Sarepta Therapeutics, Inc.

    Investors:

    Ian Estepan, 617-274-4052

    Media:

    Tracy Sorrentino, 617-301-8566

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  2. --Results demonstrate continued safety and tolerability of SRP-9001 in four participants with Duchenne --

    --All four participants demonstrated improvements in NSAA scores compared to baseline and showed a durable response two years after administration of SRP-9001 --

    CAMBRIDGE, Mass., Sept. 28, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced two-year follow up results from four Duchenne muscular dystrophy (DMD) clinical trial participants who received SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted…

    --Results demonstrate continued safety and tolerability of SRP-9001 in four participants with Duchenne --

    --All four participants demonstrated improvements in NSAA scores compared to baseline and showed a durable response two years after administration of SRP-9001 --

    CAMBRIDGE, Mass., Sept. 28, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced two-year follow up results from four Duchenne muscular dystrophy (DMD) clinical trial participants who received SRP-9001 (AAVrh74.MHCK7.micro-dystrophin). SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of micro-dystrophin protein. Results presented at the 25th International Annual Congress of the World Muscle Society demonstrated that two years after a one-time infusion of SRP-9001, trial participants exhibited a mean 7.0 point improvement on the North Star Ambulatory Assessment (NSAA) compared to baseline.

    "We continue to be encouraged by the safety profile and enduring treatment response that we have seen to date with SRP-9001 gene transfer therapy," said Doug Ingram, President and CEO, Sarepta. "The consistent results and functional improvements sustained over two years give us added confidence as we prepare for the results from Study 102, our randomized, double-blind, placebo-controlled study of SRP-9001. We continue to work with urgency to bring this potentially transformative treatment to patients as quickly as possible."

    In the open-label trial, known as Study 101, four ambulatory participants between the ages of 4 and 7 were treated with an infusion of SRP-9001 at a dose of 2x1014 vg/kg. The therapy was well-tolerated in all participants over the two-year time period. All adverse events were considered mild or moderate, and occurred within 90 days of treatment. There were no serious adverse events or evidence of complement activation.

    At day 90, all participants had confirmed vector transduction and showed functional improvement on the NSAA scale and reduced creatine kinase (CK) levels. Participants demonstrated a mean increase of 5.5 points from baseline one year after treatment and 7.0 points from baseline two years after treatment. The NSAA is a validated scale developed to measure functional motor abilities in ambulant children with Duchenne, with scores ranging from 0-34.

    As previously disclosed, micro-dystrophin protein levels for participants in Study 101 were as follows: 12-weeks post-infusion, a mean of 81.2% muscle fibers expressing micro-dystrophin with a mean intensity at the sarcolemma by immunohistochemistry of 96% compared to normal biopsies. Adjusted for fat and fibrotic tissue, western blot showed a mean expression of 95.8%.

    About SRP-9001

    SRP-9001 is an investigational gene transfer therapy intended to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for global development and manufacturing for SRP-9001 and plans to commercialize SRP-9001 in the United States. In December 2019, the Company announced a licensing agreement granting Roche the exclusive right to launch and commercialize SRP-9001 outside the United States. Sarepta has exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Children's Hospital.

    About Duchenne Muscular Dystrophy

    Duchenne muscular dystrophy (DMD) is a rare, fatal neuromuscular genetic disease that occurs in approximately one in every 3,500-5,000 males worldwide. DMD is caused by a change or mutation in the gene that encodes instructions for dystrophin. Symptoms of DMD usually appear in infants and toddlers. Affected children may experience developmental delays such as difficulty in walking, climbing stairs or standing from a sitting position. As the disease progresses, muscle weakness in the lower limbs spreads to the arms, neck and other areas. Most patients require full-time use of a wheelchair in their early teens, and then progressively lose the ability to independently perform activities of daily living such as using the restroom, bathing and feeding. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and patients usually succumb to the disease in their twenties.

    About Sarepta Therapeutics

    At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company's programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.



    Sarepta Forward-Looking Statement

    This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding SRP-9001's intention to deliver the micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein, the potential benefits of SRP-9001 and its potential to provide a transformative treatment for DMD, the future results from Study 102 and potential market opportunities.

    These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in preclinical trials and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; if the actual number of patients suffering from DMD is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the COVID-19 pandemic; and even if Sarepta's programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading "Risk Factors" in Sarepta's most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

    Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

    Internet Posting of Information

    We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.  

    Source: Sarepta Therapeutics, Inc.

    Investors:

    Ian Estepan, 617-274-4052

    Media:

    Tracy Sorrentino, 617-301-8566



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  3. -- Continued functional improvements were observed at 18 months in the low-dose cohort -- 

    -- First look at functional outcomes in high-dose cohort found improvements 6 months after administration --

    -- Results in both cohorts continue to reinforce safety and tolerability profile of SRP-9003 --

    CAMBRIDGE, Mass., Sept. 28, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from the ongoing study of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Company's investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). Results included 18-month functional data from three clinical trial participants in…

    -- Continued functional improvements were observed at 18 months in the low-dose cohort -- 

    -- First look at functional outcomes in high-dose cohort found improvements 6 months after administration --

    -- Results in both cohorts continue to reinforce safety and tolerability profile of SRP-9003 --

    CAMBRIDGE, Mass., Sept. 28, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced positive results from the ongoing study of SRP-9003 (rAAVrh74.MHCK7.hSGCB), the Company's investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E). Results included 18-month functional data from three clinical trial participants in the low-dose cohort and 6-month functional data from three participants in the high-dose cohort. SRP-9003 is in development for the treatment of LGMD2E (also known as beta-sarcoglycanopathy and LGMDR4), a devastating monogenic neuromuscular disease caused by a lack of beta-sarcoglycan proteins. SRP-9003 is a gene construct that transduces skeletal and cardiac muscle, delivering a gene that codes for the full-length beta-sarcoglycan protein, the absence of which is the sole cause of the progressive degeneration and a shortened lifespan characterized by the disease.

    "There are currently no approved treatments for people with LGMD2E – a disease that causes significant disability in children and often leads to early mortality. It's very encouraging that we continue to see consistent, positive data from our investigational gene therapy SRP-9003 across several measures, as we know the community needs more options," said Louise Rodino-Klapac, Ph.D., senior vice president of gene therapy, Sarepta Therapeutics. "The improvements in functional measures at 18- and 6- months in participants from both cohorts who received SRP-9003 are distinctly different from what an age-matched, natural history group would predict with LGMD2E. This sustained durability of the response in functional outcomes reinforces that SRP-9003 is getting to the muscle and suggestive of improvement against disease-mediated muscle damage. When coupled with the strong expression results and encouraging safety profile seen to date, today's results increase our confidence in the construct and provide additional evidence as we advance the higher dose of SRP-9003 into the next stage of clinical testing."

    Efficient transduction in skeletal muscle and robust beta-sarcoglycan protein expression were seen in both dose cohorts following infusion with SRP-9003, and significant creatine kinase (CK) reductions were observed at 90 days. Cohort-specific results as follows:

    Cohort 1 (low dose), at 18 months:

    • All three participants continued to show improvements from baseline across all functional measures, including the North Star Assessment for Dysferlinopathies (NSAD), time-to-rise, four-stair climb, 100-meter walk test and 10-meter walk test.
    • The mean NSAD improvement from baseline was 3.0 at 6 months and 5.7 at 18 months.
    • There have been no new drug-related safety signals observed since the one-year update in June 2020, and no decreases in platelet counts outside of the normal range or signs of complement activation were observed.

    Cohort 2 (high dose), at 6 months:

    • All three participants demonstrated improvements from baseline across all functional measures, including the NSAD, time-to-rise, four-stair climb, 100-meter walk test and 10-meter walk test.
    • The mean NSAD improvement from baseline was 3.7.
    • There have been no new drug-related safety signals observed since expression results were shared in June 2020, and no decreases in platelet counts outside of the normal range or signs of complement activation were observed.

    About SRP-9003 and the study

    SRP-9003 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. AAVrh74 has lower immunogenicity rates than reported with other human AAV vectors. The MHCK7 promoter has been 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.

    This first-in-human study is evaluating a single intravenous infusion of SRP-9003 among children with LGMD2E between the ages of 4 and 15 years with significant symptoms of disease. The SRP-9003 study has two cohorts, each studying a different dose-per-kilogram based on the weight of the patient. Three participants in the low-dose cohort (Cohort 1) were treated with a one-time infusion of SRP-9003 dosed at 5x1013 vg/kg and an additional three participants in the high-dose cohort (Cohort 2) received a one-time infusion dosed at 2x1014 vg/kg. The six participants were between the ages of 4 and 13. Post-treatment biopsies were taken at 60 days.

    Sarepta has exclusive rights to the LGMD2E gene therapy program initially developed at the Abigail Wexner Research Institute at Nationwide Children's Hospital.

    About Limb-Girdle Muscular Dystrophy

    Limb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

    Patients with limb-girdle muscular dystrophy Type 2E (LGMD2E) begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality. There is currently no treatment or cure for LGMD2E.

    Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A.

    About Sarepta Therapeutics

    At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company's programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on TwitterLinkedInInstagram and Facebook.



    Forward-Looking Statements

    This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding future clinical testing for SRP-9003, SRP-9003 being the ideal candidate to treat peripheral neuromuscular diseases, the potential benefits of SRP-9003 and potential market opportunities.

    These forward-looking statements involve risks and uncertainties, many of which are beyond our control. Known risk factors include, among others: success in preclinical trials and clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications we utilize to assess particular safety or efficacy parameters may yield different statistical results, and even if we believe the data collected from clinical trials of our product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; if the actual number of patients suffering from LGMD is smaller than estimated, our revenue and ability to achieve profitability may be adversely affected; we may not be able to execute on our business plans and goals, including meeting our expected or planned regulatory milestones and timelines, clinical development plans, and bringing our product candidates to market, due to a variety of reasons, some of which may be outside of our control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates and the COVID-19 pandemic; and even if Sarepta's programs result in new commercialized products, Sarepta may not achieve the expected revenues from the sale of such products; and those risks identified under the heading "Risk Factors" in Sarepta's most recent Annual Report on Form 10-K for the year ended December 31, 2019, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

    Any of the foregoing risks could materially and adversely affect the Company's business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

    Internet Posting of Information

    We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

    Source: Sarepta Therapeutics, Inc.

    Sarepta Therapeutics, Inc.

    Investors:

    Ian Estepan, 617-274-4052



    Media:

    Tracy Sorrentino, 617-301-8566

    Primary Logo

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  4. CAMBRIDGE, Mass., Sept. 15, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced 21 recipients of Route 79, The Duchenne Scholarship Program. This is the third year of the scholarship program, which was created to recognize exceptional individuals with Duchenne muscular dystrophy as they pursue their post-secondary education. Recipients were chosen by an independent selection committee composed of Duchenne community members, who consider each applicant's community involvement and a personal essay. Each student will receive a scholarship of up to $5,000.

    "It is our great privilege to announce and congratulate the 2020 recipients of Route 79, The Duchenne…

    CAMBRIDGE, Mass., Sept. 15, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced 21 recipients of Route 79, The Duchenne Scholarship Program. This is the third year of the scholarship program, which was created to recognize exceptional individuals with Duchenne muscular dystrophy as they pursue their post-secondary education. Recipients were chosen by an independent selection committee composed of Duchenne community members, who consider each applicant's community involvement and a personal essay. Each student will receive a scholarship of up to $5,000.

    "It is our great privilege to announce and congratulate the 2020 recipients of Route 79, The Duchenne Scholarship Program. With each new class of applicants, we are reminded of the varied interests, skills, and goals of these bright young people. Their perseverance and resiliency, particularly in the face of the challenges and uncertainties brought on by COVID, set a powerful example for others living with Duchenne," said Diane Berry, Sarepta's Senior Vice President of Global Health Policy, Government and Patient Affairs. "It is an honor to play a role in supporting their journey in higher education and we wish them great success. Additionally, I want to extend gratitude and appreciation to the selection committee for generously giving of their time to review the applications and essays."

    2020 Named Recipients - Route 79, The Duchenne Scholarship Program

    Praise Akintola, Farmingdale State College

    Porter Aydelotte, Saddleback College

    Donovan Carlson, University of Illinois at Urbana-Champaign

    Tyler Cooley, Arizona State University

    Lucas Currier, Great Bay Community College

    Benjamin Dupree, The University of Texas at Arlington

    Cole Dutton, West Texas A&M University

    Aiden Fecteau, Eastern Connecticut State University

    Yuvaraj Gambhir, University of Pennsylvania

    Justin Gibbons, Saint Bonaventure University

    William Hancock, Merrimack College

    Joshua Jurack, James Madison University

    Brian Le, Stanford University

    Brian Madura, New Jersey Institute of Technology

    Alice McConnell, University of Idaho

    Nicholas O'Neill, Morrisville State College

    Spencer Poole, Saint Joseph's College - Suffolk Campus

    Jordan Reidenberg, University of Delaware

    Nathan Rothe, Texas Christian University

    Christian Tumminello, Pennsylvania College of Technology

    Jack Willis, Syracuse University

    Scholarship recipients are chosen by an independent committee of Duchenne community members based on each applicant's essay and demonstrated level of community involvement. Submissions are de-identified for the voting panel with no indication of whether the candidate has received, or plans to receive, a Sarepta therapy.

    About Route 79, The Duchenne Scholarship Program

    Route 79, The Duchenne Scholarship Program is designed to help students diagnosed with Duchenne muscular dystrophy (Duchenne) pursue their post-high school educational goals. There are 79 exons in the dystrophin gene impacted by Duchenne, and the route traveled by every person with Duchenne is distinct. Sarepta's goal through this program is to acknowledge and support individuals with Duchenne who are mapping out their future via educational pursuits. Additional information is available at https://www.sarepta.com/route79.

    About Sarepta Therapeutics

    At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company's programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on TwitterLinkedInInstagram and Facebook.

    Internet Posting of Information

    We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

    Source: Sarepta Therapeutics, Inc.

    Sarepta Therapeutics, Inc.

    Investors: Ian Estepan, 617-274-4052,

    Media: Tracy Sorrentino, 617-301-8566,

    Primary Logo

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  5. -- Webcast conference call to be held on Monday, Sept. 28, 2020 at 8:30 a.m. Eastern Time --

    -- Additional poster presentations at WMS will highlight data from Sarepta's RNA and gene therapy programs --

    CAMBRIDGE, Mass., Sept. 14, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that new data from its most advanced gene therapy programs will be presented at the WMS25 Virtual Congress, the 25th International Annual Congress of the World Muscle Society, being held Sept. 28 – Oct. 2.

    Sarepta will host a webcast and conference call on Monday, Sept. 28, 2020 at 8:30 a.m. ET, to discuss the results, which include two-year functional data from Study 101…

    -- Webcast conference call to be held on Monday, Sept. 28, 2020 at 8:30 a.m. Eastern Time --

    -- Additional poster presentations at WMS will highlight data from Sarepta's RNA and gene therapy programs --

    CAMBRIDGE, Mass., Sept. 14, 2020 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that new data from its most advanced gene therapy programs will be presented at the WMS25 Virtual Congress, the 25th International Annual Congress of the World Muscle Society, being held Sept. 28 – Oct. 2.

    Sarepta will host a webcast and conference call on Monday, Sept. 28, 2020 at 8:30 a.m. ET, to discuss the results, which include two-year functional data from Study 101 of SRP-9001 for Duchenne muscular dystrophy and 18-month functional results from Cohort 1 in the study of SRP-9003 for Limb-girdle muscular dystrophy Type 2E.

    This will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and will be archived there following the call for one year. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary. The conference call may be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 6793650. Please specify to the operator that you would like to join the "Long-term Functional Data from Sarepta's Gene Therapy Programs" call.

    In total, Sarepta will present 16 abstracts at this year's meeting. All posters will be available on-demand throughout the Congress beginning on Monday, Sept. 28 at 7:00 a.m. EST. The full WMS25 Virtual Congress program is available here: https://www.wms2020.com/programme/.

    Gene Therapy:

    TITLEPROGRAMPOSTER #
    Treatment of Aged Mice and Long-term Durability of AAV-Mediated Gene Therapy in Two Mouse Models of Limb Girdle Muscular DystrophySRP-9003,

    SRP-9004

    P.137
    Expression-Functional Correlation and Validation of a Surrogate Marker for DAPC Restoration in LGMD2E Mouse ModelSRP-9003P.139
    Systemic Gene Transfer with rAAVrh74.MHCK7.SGCB Increased β-sarcoglycan Expression in Patients with Limb Girdle Muscular Dystrophy Type 2ESRP-9003P.140
    Evaluation of the Lipid-Binding and Stability Properties of Recombinant Dystrophin Spectrin-Like Repeat ConstructsSRP-9001P.206
    Systemic Gene Transfer with rAAVrh74.MHCK7.micro-dystrophin in Patients with Duchenne Muscular DystrophySRP-9001P.280
    Systemic Dose-Finding Study with AAV-Mediated γ-Sarcoglycan Gene Therapy for Treatment of Muscle Deficits in LGMD2C MiceSRP-9005P.138

    RNA Platform:

    TITLEPROGRAMPOSTER #
    Long-term Safety and Efficacy of Golodirsen in Male Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 SkippingGolodirsenP.283
    Casimersen Treatment in Eligible Patients with Duchenne Muscular Dystrophy: Safety, Tolerability, and Pharmacokinetics Over 144 Weeks of TreatmentCasimersenP.288
    Open-Label Evaluation of Eteplirsen in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI TrialEteplirsenP.289
    Delay in Duchenne Muscular Dystrophy Progression with Eteplirsen: Attenuation of Pulmonary Decline and Projected Freedom from Continuous VentilationEteplirsenP.290
    Real-world Evidence of Eteplirsen Treatment Effects on Duchenne Muscular Dystrophy Related Health Outcomes Using Claims Data in the United StatesEteplirsenP.291

    Natural history and other presentations:

    TITLEPOSTER #
    Development of Cardiomyopathy, Respiratory Insufficiency and Loss of Ambulation in Becker Muscular Dystrophy: A Systematic Literature ReviewP.41
    Disease Attributes Most Important from a Societal Perspective: A Case Study Involving Duchenne Muscular DystrophyP.56
    Identification of Disease Progression Stages in Patients with Duchenne Muscular Dystrophy Using Administrative Claims Data in the United StatesP.119
    The Age at Loss of Ambulation Among Patients with Limb-Girdle Muscular Dystrophy (LGMD) Subtype 2: A Systematic ReviewP.142
    Rasch Analysis of the Pediatric Quality of Life Inventory 4.0 Generic Core Scales Administered to Patients with Duchenne Muscular DystrophyP.345

    Presentations will be archived under the events and presentations section of the Sarepta Therapeutics website at www.sarepta.com for one year following their presentation at WMS25. 

    About Sarepta Therapeutics

    At Sarepta, we are leading a revolution in precision genetic medicine and every day is an opportunity to change the lives of people living with rare disease. The Company has built an impressive position in Duchenne muscular dystrophy (DMD) and in gene therapies for limb-girdle muscular dystrophies (LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth (CMT), and other CNS-related disorders, with more than 40 programs in various stages of development. The Company's programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

    Internet Posting of Information

    We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us. 

    Source: Sarepta Therapeutics, Inc.

    Sarepta Therapeutics, Inc.

    Investors: Ian Estepan, 617-274-4052,

    Media: Tracy Sorrentino, 617-301-8566,  

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