Sarepta Therapeutics Announces Launch of EMBARK, a Global Pivotal Study of SRP-9001, a Gene Therapy for the Treatment of Duchenne Muscular Dystrophy
Category: DNA RNA and cells
Posted on Monday, 04 October 2021 23:18
CAMBRIDGE, MA, USA I 04 October 2021 I Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a leader in precision genetic medicine for rare diseases, today announced the launch, in partnership with Roche, in the United States and countries of the world, of the 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 experimental gene transfer therapy for delivering its gene encoding micro-dystrophin to muscle tissue for the targeted production of the micro-dystrophin protein.
“We are delighted to announce the launch of EMBARK, which represents the first pivotal double-blind gene therapy trial in Duchenne to be launched in the United States, Europe and Asia,” said Doug Ingram, president and chief from the leadership of Zarephath. âThe launch of EMBARK represents the culmination of tremendous efforts and success from a research, development and manufacturing perspective and is an extremely important moment for the patient community and a quantum leap in our efforts to change the course of Duchenne. In addition to our team, investigators, and the families who participated in our trials, I would like to thank the professionals in the Office of Tissues and Advanced Therapies at the FDA for their guidance and collaborative ideas as we finalized our EMBARK study. and were advancing the SRP-9001. program. We look forward to sharing details of EMBARK and additional functional data from our previous studies at our upcoming âMicro-Dystrophin Dayâ.
The company will host an SRP-9001 Micro-Dystrophin R&D Day on Monday, October 11, 2021 at 8:30 a.m. Eastern Time.
The presentation will be webcast live in the Investor Relations section of Sarepta’s website at https://investorrelations.sarepta.com/events-presentations and the slides will be archived there after the call for one year. Please log into the Sarepta website several minutes before the start of the broadcast to ensure sufficient time for any software downloads that may be required. The conference call can be accessed by dialing (844) 534-7313 for domestic callers and (574) 990-1451 for international callers. The access code for the call is 3878397. Please let the operator know that you wish to participate in the “SRP-9001 Micro-dystrophin R&D Day” call.
About SRP-9001 (rAAVrh74.MHCK7.micro-dystrophin)
SRP-9001 is an experimental gene transfer therapy for delivering the gene encoding micro-dystrophin to muscle tissue for the targeted production of the micro-dystrophin protein. Sarepta is responsible for the global development and manufacturing of the SRP-9001 and plans to market SRP-9001 in the United States after 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 owns the exclusive rights to the micro-dystrophin gene therapy program initially developed at the Abigail Wexner Research Institute of Nationwide Children’s Hospital.
About Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a rare and fatal genetic neuromuscular disease that occurs in approximately one in 3,500 to 5,000 men 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 walking, climbing stairs, or standing from a seated position. As the disease progresses, muscle weakness in the lower extremities spreads to the arms and other areas. Most patients require a full-time wheelchair in their early teens and then gradually lose the ability to independently perform activities of daily living such as using the toilet, bathing, and sleeping. food. Eventually, increasing breathing difficulties due to dysfunction of the respiratory muscles require ventilatory assistance, and cardiac dysfunction can lead to heart failure. The disease is universally fatal, and patients typically succumb to the disease in their twenties.
About Sarepta Therapeutics
Sarepta has an urgent mission: to design precision genetic medicine for rare diseases that devastate lives and cut short the future. We occupy leading positions in Duchenne muscular dystrophy (DMD) and girdle muscular dystrophies (LGMD) and currently have over 40 programs in various stages of development. Our vast pipeline is powered by our multi-platform precision genetic medicine engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.
THE SOURCE: Therapeutic Zarephath