The FDA New Guidelines to Speed the Approval of Gene Therapy. See Also: The Gene Editing Effect

Important news has come from the FDA, which issued new guidelines to speed the introduction of treatments with human cells and tissues, including gene therapy. Cracking down on rogue clinics offering dangerous or unproven versions of gene therapy or stem cell therapy is also included in the report.

Scott Gottlieb, the commissioner of the FDA stated that regenerative medicine and other important gene therapy treatments are no longer science fiction, but rather, real-life science where cells and tissues can be engineered to grow healthy functional organs to replace diseased ones with new genes to be introduced into the body to combat disease and where adult stem cells can generate replacements for cells that are lost to injury or illness.

Gene and cell therapies that demonstrate the potential to treat unmet medical needs and serious illnesses may now qualify for expedited review to get the products to market more quickly, the guidelines say. The FDA will still require clinical trials, but it is promising a faster process, as required by Congress under the 2016 21st Century Cures Act.

Prohost Observations

Genetically engineered cell therapy mentioned in the report is the CAR T immuno-oncology products that two of them are already FDA approved. These are Kymriah CAR T from Novartis (NVS)  and  Yescarta created and developed by Kite Pharma, and owned now by Gilead (GILD) after it acquired Kite. The genetic engineering in these products is conducted on the patients’ cells in vitro so the engineered cells turn into immunotherapy treatments for leukemia, lymphoma, or multiple myeloma.

Another more typical gene therapy is that which corrects defective patients’ genes was recommended approval by the FDA advisory panel belongs to Spark Therapeutics (ONCE). This gene therapy is used to correct a hereditary gene defect that causes the patients loss of sight. Many other gene therapies with different technologies are in clinical trials promising to treat acquired as well as hereditary gene mutations that cause intractable and life-threatening diseases.

The Gene Editing Effect

The number of gene therapy treatments will be increased with the advancement of gene editing, including, Clustered Regulatory Interspaced Short Palindromic Repeats/Cas9 systems (CRISPR/Cas9); Transcription Activator-Like Effector Nucleases (TALEN) created by Cellectis (CLLS); and Zinc finger nucleases (ZFNs) created and used by Sangamo Therapeutics (SGMO).

Cellectis has used its TALEN gene editing technology to genetically engineer a chimeric antigen receptor T cell (CAR T) treatments for Acute Lymphoblastic Leukemia (ALL) and multiple myeloma (MM).

Recently, for the first time in history, Sangamo’s zinc finger nuclease (ZFN) gene-editing technology enabled a patient with mucopolysaccharidosis type II (MPS II), also known as Hunter receive a treatment to edit the DNA of his cells directly inside the body. (Read the story posted on November 15, 2017, under Today’s Highlights at the Prohost Website).

The biotechnology industry is leading an unprecedented revolution that left no space for argument. The good news is that the FDA is putting its weight behind this revolution, trying to guide and help speeding its path towards conquering diseases.

We are optimistic.

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