Gene therapies been re-branded 'advanced therapies'—the feeding frenzy continues

Even FDAs top vaccine guy, Peter Marks, is in on it!

[Note: this is a recycle from Jun 21, 2024]

Keep getting this advanced therapy conference in my mailbox

Every few weeks, I get an email from Terrapinn, asking me to buy a ticket for their 2024 conference on advanced therapies. The speaker list is below:

Advanced Therapies 2024, 19-20th March, ExCeL London

[Note: the link now refers to the 2025 conference and Peter Marks below is not featured. However, I’m sure the conference producers will confirm his attendance from 2020 - 2024]

Scroll down to speakers with the name beginning with ‘M’, and you will see Peter Marks M.D., PhD., Director - Center for Biologics Evaluation and Research (CBER) .

Marks is the person with delegated authority to evaluate and research applications to market new biologics drugs, eg vaccines.

Could this just be a teeny weeny conflict of interest?

Not at all, it’s a massive one!!!

It is also unprecedented for someone in his position to do anything other than fly-in for a Keynote Address, then fly out again. Hob nobbing with the regulated was always a no-no.

What about the other speakers?

Now check out the other speakers. Take it from me, none of them have a clue about the complexities of manufacturing and distributing complex biologic products.

Then check out many of the companies—no more than a handful of employees with hopes of capitalizing on the latest ‘market opportunity’.

I will leave you with the message I shared yesterday in relation to Lonza’s CEO being fired. Investors in gene therapies will lose their shirts if they keep placing their chips on mRNA injections that have been positioned as the next great white hope.

They. Are. Not.

What is the takeaway question?

Why is Peter Marks, who was/is responsible for approving all the SARS-CoV-2 injections at US FDA, hob nobbing with those he should be regulating?

Why, also, is he still approving genetically engineered drugs for sale in the US?

See: FDA's Peter Marks says gene editing breakthroughs have met regulatory reality

“The FDA's Peter Marks, M.D., Ph.D., thinks gene editing—specifically base editing—could be a "game changer," but progress has been slow, likely due to discouragement with the regulatory process. 

Marks, head of the FDA’s Center for Biologics Evaluation and Research, acknowledged that there’s been a “backslide” in new gene therapy advancements as academic founders realized that their breakthrough lab findings had to fit into the larger pharma machine. 

"People kind of woke up and realized, 'Oh, this is pharmaceutical development. There's a slog through here,'" Marks said in an interview with Fierce Biotech. 

Peter Marks, M.D., Ph.D. (FDA)

Things started out strong, giving these scientific founders hope that "they were gonna get over the finish line quickly,” Marks said. “And for various reasons, they didn’t make it there.” 

This reality check fits within Marks larger concerns that the agency is simply not approving enough gene therapies, saying earlier this year that it would be a failure if the FDA only approved two to three a year moving forward. To try to ease the backlog, the FDA has so far revamped the Office of Therapeutic Products with more specialized offices and hired more reviewers to match. With more staff, Marks says you should expect to see the FDA “leaning into the policies here,” increasing and improving communications with drug sponsors. 

Related

The future of accelerated approval for FDA's Marks? More rare disease drugs and no 'hanging chads'

“Unless we can increase the throughput of products through our process, we’re going to be lagging behind what gene therapy can potentially bring to patients,” he said. 

The FDA just last month approved Roctavian, BioMarin's gene therapy to treat adults with bleeding disorder hemophilia A. 

“Unless we can increase the throughput of products through our process, we’re going to be lagging behind what gene therapy can potentially bring to patients." — Peter Marks, FDA 

One specific gene editing advancement that Marks is excited about is base editing. Boston Children’s Hospital describes the technology as “spell-check for genes,” a mechanism by which individual letters on a base pair in the genome can be changed to fix disease-causing mutations. It's one of the latest gene editing features to spawn from the Nobel Prize-winning CRISPR/Cas9 technology, uncovered by Jennifer Doudna, Ph.D., and Emmanuelle Charpentier, Ph.D.

Make of that what you will!!!