FDA Hosts Human Genome Editing Public ZoomWebinar, February 29

While the FDA largely looks the other way in preventing endocrine-disrupting chemicals from poisoning America—allowing industry to regulate itself—it is extremely interested in a hands-on role in correcting human maladies through overseeing development, refinement and application of expensive technologies/therapies, like in vitro gametogenesis (IVG) and human genome editing (HGE), the latter first approved as a therapy by FDA in December 2023 in the treatment of Sickle Cell Disease (SCD).

FDA, hoping now to garner public approval for HGE as a therapy, has invited the public to a Zoom webinar on February 29 (1-2pm ET) in which HGE guidelines will be thrashed out. While the deadline for the public to submit questions was February 13, registration is still open for “attending” the Zoom meeting with proceedings recorded and subsequently posted online.

Hosting the event is the agency’s Center for Biologics Evaluation and Research, headed by Peter Marks, a hematologist/oncologist who participated in last year’s controversial National Academies of Sciences meeting: “In Vitro Derived Human Gametes as a Reproductive Technology”—happily predicting that IVG would be realized within 1 or 2 US Presidential cycles.

Click HERE for the FDA’s Feb. 29 webinar invite: “Human Gene Therapy Products Incorporating Human Genome Editing”.

Click HERE for a PDF of the FDA’s working industry guidelines for Human Gene Therapy Products Incorporating Human Genome Editing.

The FDA’s recent landmark approval of the application of HGE as a therapy in the treatment of SCD was given to Big Pharma following small (and ongoing) industry clinical trials that have shown modest results so far in permanent correction by targeting the so-called hemoglobin gene on chromosome 11.

Chemotherapy is first administered to the patient stripping bone marrow of diseased cells; editing the cells comes next, then destroying the diseased bone marrow, and finally infusing the patient with its edited/corrected cells.

SCD afflicts people of color in disproportionate numbers; red blood cells become rigid and crescent-shaped interfering with blood oxygen flow. However, Big Pharma’s SCD HGE therapy is costly—roughly $3 million per patient—and the plan is to pass the cost on to Medicare and Medicaid.

What happens if the therapy provider goes under, bankrupt, out-of-business, is another burning issue.

Of particular importance—HGE is at odds with physical biology, which sees causation as circular not gene-centered, i.e., genotype to phenotype is NOT 1 to 1.

FDA presents the problem this way in its working industry guidelines:

“Some of the specific risks associated with GE approaches include off-target editing, unintended consequences of on-target editing, and the unknown long term effects of on- and off-target editing.”

FDA further cautions:

“It is also important to keep in mind that, although these processes can be accurate, they can also result in DNA insertions or deletions (indels) with unanticipated consequences. DNA cleavage events, which could be caused by multiplex on-target editing or a combination of on- and/or off-target effects, can also lead to chromosomal rearrangements, including translocations.”

And it adds:

“[W]ith regard to persistence of the GE components, the longer the GE component (e.g., the nuclease) is functionally active, the greater the risk of unintended genomic modifications, specifically off-target editing and chromosomal rearrangements.”

Thus, there have been significant side effects to HGE SCD treatment, including three deaths during clinical trials, blood cancer, fertility issues, conflicts with other medications such as HIV drugs—as well as somewhat expected nausea, vomiting, itches, fatigue, etc.

Suggested monitoring of patients post-treatment is 15 years.