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…]
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. [Read more…]
The AASCP has set up safety guidelines for Physicians who use regenerative therapies in their practice.
MIAMI, September 6, 2019 – The American Academy of Stem Cell Physicians (AAOSCP), strives to provide an industry standard for Physicians who use regenerative therapies in their practice. The group has recently created guidelines which are current safety recommendations given to Physicians who are using biologics in their medical practice.
According to AASCP, If you are using biologics in your practice, including but not limited to SVF, PRP, bone marrow, UCB, amniotic products, exosomes, xenografts, or peptides, there are key considerations to take into account to achieve safety for your patients. The AASCP also recommends communication with the Chief Scientific Officer (CSO) from the laboratory with whom you work, and advises that talking to a sales agent is insufficient to determine the quality of regenerative medicine (RM) products for your patients. Unfortunately, sales agents frequently do not have a medical or scientific background and are commercially motivated to promote specific products.
Furthermore, if you are dealing with a company who cultures and expands their cells through the use of growth factors in the media, it is critical that you raise certain questions with the Chief Scientific Officer.
AASCP has outlines the recommendations below to assist Physicians.
Example of questions recommended by AASCP are the following:
( a ) How many passages do they do when culturing their cells? What is the source of the biologics?
* It is well known that culture expansion beyond a certain threshold increased incidences of mutagenesis. Mutation of DNA is fundamental to cancer pathogenesis. Lab cultures expanding beyond 5 passages risk passing along exosomes with cancer stemness. Furthermore, as highlighted in the Tamanako article, neural stimulating factors used in culture media can trigger pancreatic cancer stemness.
( b ) Have you had any adverse effects over the past year? Past five years?
( c ) What is your method of sterilization?
( d ) What is your method of shipping?
( e ) What kind of disease testing are you doing on donors?
( f ) Are you testing for bacteria or other contaminants before sending out your product?
( g ) Have you done any biological profiling on your product?
( h ) Have you done an immunoassay to verify immunomodulatory effects?
( i ) Are your products processed in an aseptic environment?
( j) What is your current lab certification?
( k ) Who processes your products? What are their qualifications?
The spokesman for the AASCP, Dr. AJ Farshchian, said earlier; ”Safety is first. Over the past few months, we have seen several Biotech companies who put greed before safety, and Physicians and consumers need to beware of unregulated practices. Hopefully, the safety guidelines we have developed will set the tone and the bar high enough for our health care system which is number one in the world. Currently, our safety committee meets weekly and is in the process of evaluating some of the laboratories that are questionable.”
Among members of the AASCP’s Safety Committee is Anette Mnabhi, DO, NBOPASFP, NBOPASNMM, CAAOM, RN, MSN. Dr. Mnabhi is an osteopathic physician who utilizes the principles of regenerative medicine as a foundation in her clinical practice.
About the American Academy of Stem Cell Physicians (AASCP)
The American Academy of Stem Cell Physicians (AASCP) is an organization created to advance research and the development of therapeutics in regenerative medicine, including diagnosis, treatment, and prevention of disease related to or occurring within the human body. Secondarily, the AASCP aims to serve as an educational resource for physicians, scientists, and the public in diseases that can be caused by physiological dysfunction that are ameliorable to medical treatment.
For further information, please contact Marie Barba at AASCP 305-891-4686. To request assistance from the AASCP in evaluating regenerative therapies, email [email protected].
The U.S. FDA also offers valuable resources pertaining to its Cellular & Gene Therapy Guidances at this link.
Do you have questions for the AASCP? Ask them in the comments below.
Three-Year Registrational Data Set to be Presented by Year End 2019
Regulatory Submissions Planned in the U.S. and Europe for 2021
BOSTON and LONDON, July 29, 2019 — Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that it has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for OTL-103, Orchard’s ex vivo autologous hematopoietic stem cell (HSC)-based gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS) developed at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. [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.