A new state-of-the-art facility dedicated to plasmid manufacturing will be in operation by August 2019 in Gerenzano, Italy. The new site will couple with the existing GMP facility active in the Cell and Gene (C&G) therapy space. With 800m2 (8,000 square feet) of cleanrooms, the two facilities of Anemocyte, the first Biotech Manufacturing Organisation (BMO), will address manufacturing needs of Somatic Cells, Non-Viral Modified Cells, Vesicles and Plasmids for Viral Vectors. [Read more…]
The FDA has granted RMAT designation for Sangamo Therapeutics’ SB-525, an investigational gene therapy being developed in collaboration with Pfizer to treat severe hemophilia A. SB-525 is the first hemophilia A gene therapy to receive RMAT designation.
According to Sangamo Therapeutics:
“We are encouraged by the initial clinical data suggesting safety, tolerability, and efficacy of SB-525 and are beginning preparations, including manufacturing, to potentially advance into a registrational study. We are also encouraged by our interactions with regulators and by the FDA’s recent RMAT designation,” said Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Diseases Research Unit. “If FVIII levels are sustained, and patients continue to have no bleeding episodes and remain off factor replacement therapy, we believe that this gene therapy may potentially represent a transformative treatment paradigm for severe hemophilia A.”
FDA Approved RMAT Designations
To date, 30 RMAT designations have been publicly announced. The most recent additions were Krystal Biotech’s RMAT designation for KB103 on June 24, 2019 and Sangamo Therapeutics’ RMAT designation for SB-525 on July 5, 2019.
As of today, the list of publicly announced RMAT’s has risen to 30. However, the U.S. FDA states it has received 108 total requests for RMAT designations and granted 40.
RMAT stands for “Regenerative Medicine Advanced Therapy” designation. Sponsors of cell and gene therapies are eligible to obtain an RMAT designation from the U.S. FDA if their product is intended to treat serious or life-threatening diseases and there is preliminary clinical evidence that it can address unmet medical needs.
In this article:
- RMAT List
- RMAT Reviews by The FDA
- Regenerative Medicine Advanced Therapy (RMAT)
- RMAT Definition
- Future RMAT Designations
New York, NY, June 27, 2019 — Qrons Inc. (OTC: QRON), an emerging biotechnology company developing advanced stem cell-synthetic hydrogel-based solutions for the treatment of traumatic brain injuries (“TBIs”), including concussions and penetrating injuries, announced today the appointment of John N. Bonfiglio, PhD, MBA as its Chief Operating Officer. Qrons Inc. (OTC: QRON)
Dr. Bonfiglio’s primary responsibility will be to oversee all investigational new drug (“IND”) activities in the United States in coordination with Qrons’ Israeli-based scientific team. [Read more…]
Mesoblast (ASX:MSB; Nasdaq: MESO) announced that the U.S. FDA granted the company’s product candidate, Revascor, Orphan Drug Designation for prevention of gastrointestinal bleeding in patients with left ventricular assist devices.
Revascor, Mesenchymal Precursor Cells
Revascor is Mesoblast’s investigational cell therapy composed of 150 million allogeneic mesenchymal precursor cells (MPCs). [Read more…]