On September 4, 2019, Magenta Therapeutics announced it had received an FDA Regenerative Medicine Advance Therapy (RMAT) designation for MGTA-456, an expanded CD34+ cell therapy treatment used in place of single umbilical cord blood (UCB) transplantation. This one-time stem cell treatment is being investigated for the treatment of multiple inherited metabolic disorders. While there are 36 publicly announced RMAT designations, the FDA states that 40 or more have been awarded to date. [Read more…]
SanBio, a company developing regenerative cell medicines for neurological disorders, has been awarded a regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration (FDA) for SB623.
SB623 is a cell therapy that the company has developed for the treatment of chronic neurological motor deficits that occur as a result of traumatic brain injury (TBI). In addition to this treatment, SanBio is developing regenerative therapies for stroke, diseases of the eye, Parkinson’s disease (PD), and other neurological conditions. [Read more…]
Cynata Therapeutics Ltd (ASX: CYP) is a clinical stage regenerative medicine company specializing in stem cell therapeutics that has a proprietary technology for manufacturing induced pluripotent stem cell (iPSC) derived mesenchymal stem cells (MSCs).
I had the honor of interviewing Dr Ross Macdonald, CEO of Cynata Therapeutics. In this interview, we discuss Cynata’s technology platform, the company’s world first achievements, and its clinical trial underway for GvHD. We also explore the company’s strategic goals and the importance of its partnership with Fujifilm in Japan. [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…]