Industry Partner News: Capricor Announces Licensing of Additional Patent Applications from Cedars-Sinai Medical Center for Cellular and Exosome-based Technologies

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Capricor Therapeutics announced January 16th, 2018 that it has added seven new patent applications to its existing Exclusive License Agreements with Cedars-Sinai Medical Center for technologies related to cardiosphere-derived cells (CDCs) and CDC-derived extracellular vesicles, including exosomes. 

These new patent applications will pave the way for Capricor to develop new therapeutic platforms for Duchenne muscular dystrophy and other disorders. 

Read their full release by clicking here.

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Industry Partner News: Santhera Announces New Awareness Campaign

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Santhera Pharmaceuticals announces the launch of a disease awareness campaign for the Duchenne muscular dystrophy (DMD) community in the U.S. with a focus on respiratory care.

The "Take a Breath DMD" campaign will underscore the importance of respiratory care for people living with Duchenne muscular dystrophy. The mission of this educational campaign is to help people living with DMD and their families receive information to help manage respiratory complications, including information about breathing, coughing and pulmonary care.

For more information, please see their full release by clicking here.

Industry Partner News: Summit Completes Initial 24 Weeks of Dosing of Ezutromid in Patients with DMD in PhaseOut DMD Clinical Trial

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Summit announces the completion of the initial 24-weeks of dosing of ezutromid in patients with DMD in its Phase 2 clinical trial called PhaseOut DMD. With this milestone, Summit continues to expect to report results from this initial 24-week dosing period in the first quarter of 2018. The 24-week results are expected to include data from muscle biopsies, MRI and functional tests, as well as safety data.

For more information, please see the Summit press release by clicking here.

Industry Partner News: Sarepta Shares Quarterly Update

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This quarterly update contains news regarding Sarepta's clearance from the U.S. Food and Drug Administration (FDA) to start three clinical trials in individuals with DMD – two in gene therapy and one for SRP-5051, its PPMO exon 51 skipping candidate. Sarepta also shares its pursuit of a CRISPR/Cas9 approach through a recently signed collaboration agreement with Duke University. For additional details and more news from Sarepta, please view their newsletter by clicking here.

Industry Partner News: Santhera's SIDEROS trial currently enrolling

The SIDEROS clinical trial, sponsored by Santhera, is currently recruiting boys with DMD ages 10 and older at 24 trial sites in the U.S. The study will compare the efficacy of idebenone to placebo in boys and men currently on steroids. Idebenone is an investigational drug in Phase 3 development for patients with DMD who are in respiratory decline. For more information on the study and enrollment eligibility, please view their newsletter update or visit www.siderosdmd.com.

Industry Partner News: PTC Therapeutics Receives Complete Response Letter for Ataluren's NDA

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The Office of Drug Evaluation I of the U.S. Food and Drug Administration (FDA) has issued a complete response letter stating it cannot approve PTC Therapeutics, Inc.'s New Drug Application (NDA) for the investigational medicine ataluren. Ataluren, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with Duchenne caused by a nonsense mutation.For more, please see PTC Therapeutics, Inc.'s full press release or President and CEO Stuart Peltz, Ph.D.'s community letter.

Industry Partner News: Sarepta Therapeutics Announces Positive Dystrophin Results in Study Evaluating Golodirsen (SRP-4053)

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today announced muscle biopsy results from its 4053-101 study, a Phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of golodirsen in 25 boys with confirmed deletions of the DMD gene amenable to skipping exon 53.

Industry News: FDA Announces Advisory Committee Meeting to Review Ataluren.

The Food and Drug Administration (FDA) has published the date, location, and time of the Central and Peripheral Nervous System (PCNS) Advisory Committee Meeting to review autaluren and request for public comments to the Federal Register. The Advisory Committee will discuss  ataluren's new drug application by PTC Therapeutics, for the treatment of patients with Duchenne muscular dystrophy due to a nonsense mutation in the dystrophin gene.

Industry News: Capricor Announces Results of FDA Meeting on Intravenous CAP-1002 for Duchenne Muscular Dystrophy

Industry News: Capricor Announces Results of FDA Meeting on Intravenous CAP-1002 for Duchenne Muscular Dystrophy

Capricor Therapeutics, Inc.a biotechnology company developing biological therapies for Duchenne muscular dystrophy (DMD) and other rare diseases, today announced that it has received official minutes of the meeting held recently between the U.S. Food and Drug Administration (FDA) and Capricor to discuss the development of intravenous CAP-1002 (allogeneic cardiosphere-derived cells) for the treatment of Duchenne muscular dystrophy (DMD).

Industry News: PTC Announces FDA Advisory Committee Meeting for Ataluren

today announced that the U.S. Food and Drug Administration (FDA) has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna™). today announced that the U.S. Food and Drug Administration (FDA) has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna™).