The FDA’s Center for Biologics Evaluation and Research is planning to release some crucial guidance soon for CRISPR and gene therapy developers on platform technologies, accelerated approvals for rare diseases, and potentially in the “not-too-distant future,” a platform technology guidance specifically for genome editing, CBER Director Peter Marks said Tuesday in an Alliance for a Stronger FDA webinar.

The guidance documents will be another positive sign that CBER’s staffing up is paying off. Marks noted Tuesday that hiring has improved in 2024 compared to recent years.

Continuing his defense of the use of accelerated approvals for rare diseases over the last several months, Marks said the new draft guidance will outline buckets of different types of development programs: some easier and some more challenging.

“What we’re doing is taking a calculated risk,” Marks said. “I’m not going to lose sleep if it takes two or three years to get the confirmatory evidence.”

CBER is also continuing a pilot project, first unveiled in September, looking to speed up its review of three treatments for rare pediatric diseases that can cause death or serious disability.

“If we can show even a 10% decrease in time, that translates into a sufficiently large number of dollars across different rare disease programs, that one could easily justify the staffing up in the number of reviewers that would need to be brought on board to make this a regular procedure,” he said.

There are “three major buckets,” Marks said, to the accelerated approval guidance.

The simplest bucket consists of instances when a gene therapy’s effect can be measured, and it correlates with an aspect of health “so that just measuring that product is reasonably likely to predict the gene therapy is going to be effective,” Marks said. That includes examples of gene therapies for hemophilia or beta thalassemia.

Another bucket “is you may not be able to measure the gene therapy product, but you can measure something else relatively easily, like an upstream marker or a downstream marker,” Marks said.

Those buckets, Marks said, are “the two low-hanging fruits.” More complicated situations arise with more complex genetic diseases.

“We want a guidance that helps people understand the place where there’s really low-hanging fruit, medium, and the challenging places. Not to say that we shouldn’t go where it’s very challenging, but just so that people understand where they’re operating,” Marks said.

Check out our AAV CDMO service to expedite your gene therapy research
About PackGene

PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.

Related News

Future Of Gamma Delta T Cell Therapies

Gamma delta T cell-based immunotherapies have emerged as a promising frontier in cancer treatment and beyond, building on the success of chimeric antigen receptor T-cell (CAR T cell) therapy and expanding on the potential of the immune system as a therapeutic...

read more

Related Services

Plasmids GMP Services

Multiple scales & grade of solutions of various kind of plasmids suitable for multiple treatments in a fast and cost effective way.

AAV GMP Services

Ranging from small-scale AAV production, to large-scale AAV cGMP manufacturing for animal studies.
aav icon

Technology Platforms

PackGene’s proprietary π-Alpha™ 293 AAV High-yield Platform increases AAV production by 3 to 8 times that of traditional platforms.