“DIY” Gene Therapy Products Unsafe, Sale Illegal – FDA Repeats

by Kim Boateng Posted on May 16th, 2018

Silver Spring, Maryland, USA: “#FDA is concerned about the safety risks involved in “DIY” gene therapy products intended for self-administration and “do it yourself” kits to produce gene therapies for self-administration. We warned the sale of these products is against the law, Food and Drug Administration, FDA, commissioner, Scott Gottlieb, M.D, said today.

FDA said it is aware that gene therapy products intended for self-administration and “do it yourself” kits to produce gene therapies for self-administration are being made available to the public. The sale of these products is against the law. FDA is concerned about the safety risks involved.

According to the FDA, gene therapy is the administration of genetic material to modify or manipulate the expression of a gene product or to alter the biological properties of living cells for therapeutic use. Gene therapies offer the potential to treat diseases or conditions for which no or few treatments exist. They are being studied to treat cancer as well as genetic, infectious, and other diseases. FDA considers any use of CRISPR/Cas9 gene editing in humans to be gene therapy.

Gene therapy products are regulated by the FDA’s Center for Biologics Evaluation and Research (CBER). Clinical studies of gene therapy in humans require the submission of an investigational new drug application (IND) prior to their initiation in the United States, and marketing of a gene therapy product requires submission and approval of a biologics license application (BLA), the FDA said.

Clinical trials of gene therapies can be found at www.clinicaltrials.gov. FDA has also approved certain gene therapy products.

Consumers are cautioned to make sure that any gene therapy they are considering has either been approved by FDA or is being studied under appropriate regulatory oversight, the FDA said.

FDA Approved Cellular and Gene Therapy Products

Below is a list of licensed products from the FDA’s Office of Tissues and Advanced Therapies (OTAT).

• ANDEXXA (coagulation factor Xa (recombinant), inactivated-zhzo)
Portola Pharmaceuticals, Inc.
• ALLOCORD (HPC Cord Blood)
SSM Cardinal Glennon Children’s Medical Center
• LAVIV (Azficel-T)
Fibrocell Technologies
• MACI (Autologous Cultured Chondrocytes on a Porcine Collagen Membrane)
Vericel Corp.
• CLEVECORD (HPC Cord Blood)
Cleveland Cord Blood Center
• GINTUIT (Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen)
Organogenesis Incorporated
• HEMACORD (HPC, cord blood)
New York Blood Center
• Ducord, HPC Cord Blood
Duke University School of Medicine
• HPC, Cord Blood
Clinimmune Labs, University of Colorado Cord Blood Bank
• HPC, Cord Blood – LifeSouth
LifeSouth Community Blood Centers, Inc.
• HPC, Cord Blood – Bloodworks
• IMLYGIC (talimogene laherparepvec)
BioVex, Inc., a subsidiary of Amgen Inc.
• KYMRIAH (tisagenlecleucel)
Novartis Pharmaceuticals Corporation
Spark Therapeutics, Inc
• Plasma Cryoprecipitate (For Further Manufacturing Use)
OCTAPHARMA Pharmazeutika Produktionsges.m.b.H.
• PROVENGE (sipuleucel-T)
Dendreon Corp.
• Sterile Cord Blood Collection Unit with Anticoagulant Citrate Phosphate Dextrose Solution USP (CPD)
MacoProductions, S.A.S.
• YESCARTA (axicabtagene ciloleucel)
Kite Pharma, Incorporated

About Cord blood

Cord blood, which contains powerful stem cells, comes from a newborn’s umbilical cord and is collected immediately after birth. Once the umbilical cord has been clamped and cut, the remaining blood in the umbilical cord is drawn into a collection bag.

Cord blood stem cells are biologically younger and are more flexible compared to adult stem cells from other sources like bone marrow. When saved, they have unique qualities and advantages:

  • Less risk of complications when used in transplants
  • Ability to use one’s own stem cells for conditions that currently lack medical treatment options, also known as “autologous transplantation”
  • Immediately available and can minimize disease progression in early treatment
  • Preserving them “stops the clock” and protects the cells from aging and being exposed to environmental factors and common viruses that can decrease their function

Stem cells can heal the body, promote recovery, and offer an enormous amount of therapeutic potential.

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