{"id":10958,"date":"2021-10-04T10:35:59","date_gmt":"2021-10-04T10:35:59","guid":{"rendered":"https:\/\/laforcedmd.com\/?p=10958"},"modified":"2022-05-03T17:57:29","modified_gmt":"2022-05-03T17:57:29","slug":"embark-gene-therapy-for-the-treatment-of-dmd","status":"publish","type":"post","link":"https:\/\/laforcedmd.com\/en\/embark-gene-therapy-for-the-treatment-of-dmd\/","title":{"rendered":"EMBARK, gene therapy for the treatment of DMD"},"content":{"rendered":"

Sarepta Therapeutics announces initiation of EMBARK, a global p0ivotal study of SRP-9001, gene therapy for the treatment of DMD (Duchenne Muscular Dystrophy)<\/h4>\n

Original press release > CAMBRIDGE, Mass., Oct. 04, 2021 (GLOBE NEWSWIRE)<\/a><\/span><\/p>\n

Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, today announced the initiation, in partnership with Roche, in the U.S. and countries around the world, of study SRP-9001-301, also known as EMBARK, a pivotal study of SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin) for the treatment of Duchenne muscular dystrophy. SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.<\/strong><\/p>\n

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Doug Ingram, president and chief executive officer, Sarepta –<\/strong> \u201cWe are delighted to announce the initiation of EMBARK, representing the first pivotal double-blind gene therapy trial in Duchenne which will be initiated in the US, Europe and Asia.\u201d<\/p><\/blockquote>\n

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\u201cThe initiation of EMBARK represents the culmination of enormous effort and success from a research, development and manufacturing perspective and is an extraordinarily important moment for the patient community and a leap forward in our effort to change the course of Duchenne. In addition to our team, our investigators and the families who have participated in our trials, I want to thank the professionals within FDA\u2019s Office of Tissues and Advanced Therapies for their collaborative guidance and insight as we finalized our EMBARK study and advance the SRP-9001 program. We look forward to sharing the particulars of EMBARK and additional functional data from our prior studies in our upcoming \u2018Micro-dystrophin Day\u2019.\u201d<\/p><\/blockquote>\n

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Webcast live<\/h4>\n

The Company will host an SRP-9001 Micro-dystrophin R&D Day on Monday, Oct. 11, 2021, at 8:30 am Eastern Time.<\/strong><\/p>\n

The presentation will be webcast live under the investor relations section of Sarepta’s website here<\/a>, <\/span>and slides will be archived there following the call for one year. Please connect to Sarepta’s website several minutes before the start of the broadcast to ensure adequate time for any software download that may be necessary. The conference call may be accessed by dialling (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The passcode for the call is 3878397. Please specify to the operator that you would like to join the “SRP-9001 Micro-dystrophin R&D Day” call.<\/p>\n

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About SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)<\/h4>\n

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 upon receiving FDA approval. In December 2019, Roche partnered with Sarepta to combine Roche’s global reach, commercial presence and regulatory expertise with Sarepta’s gene therapy candidate for Duchenne to accelerate access to SRP-9001 for patients 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.<\/p>\n

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Community letter from Sarepta<\/h4>\n

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October 4, 2021, <\/em><\/p>\n

Dear Duchenne Community, <\/em><\/p>\n

Today, Sarepta Therapeutics announced the initiation, in partnership with Roche, in the United States, and countries around the world, of study SRP-9001-301, also known as EMBARK, a pivotal study of SRP-9001 (rAAVrh74.MHCK7. micro-dystrophin) for the treatment of Duchenne muscular dystrophy. SRP-9001 is an investigational gene transfer therapy intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the microdystrophin protein. <\/em><\/p>\n

Initiating a clinical trial means that the study sponsor(s) and clinical trial sites take the necessary steps required to enable dosing of the investigational therapy. These initiating steps happen prior to the first patient being recruited into a study. <\/em><\/p>\n

We have received many inquiries in the past months, and we recognize the interest and urgency expressed by the Duchenne community. Parent Project Muscular Dystrophy (PPMD)<\/a><\/span> has invited us to share details of the EMBARK clinical trial on a community webinar, which we are targeting for the week of October 11th. Please stay tuned for an announcement from PPMD. <\/em><\/p>\n

Many individuals with Duchenne, families, and Patient Advocacy Organizations have contributed time and effort to support early studies of this investigational therapy. We thank you for your dedication, without which this milestone in therapeutic development efforts would not be possible. <\/em><\/p>\n

With our kind regards, <\/em><\/p>\n

Siobhan Fitzgerald<\/em><\/p><\/blockquote>\n

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A link to the community letter may be found here.<\/a><\/span><\/p>\n

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About Sarepta<\/h4>\n

Sarepta Therapeutics, Inc., a biopharmaceutical company, is working to unlock the potential of RNA-based and gene therapy technologies for the treatment of severe and life-threatening diseases like Duchenne muscular dystrophy (DMD). Sarepta’s primary focus is to advance new treatments for DMD rapidly.<\/p>\n

More interesting links<\/h4>\n