This Week’s CGT News: Sarepta Gets FDA Platform Technology Designation
Our latest news roundup reports on dysfunction observed in edited blood stem cells, a gene therapy for Alzheimer’s, and RFK Jr’s support for experimental treatments.
| 4 min read | News
FDA Platform Technology Designation for Sarepta
The FDA has granted Platform Technology Designation to Sarepta Therapeutics for its rAAVrh74 viral vector used in its investigational gene therapy SRP-9003 (bidridistrogene xeboparvovec). The therapy is being developed for the treatment of limb-girdle muscular dystrophy type 2E/R4 (LGMD2E/R4), a rare genetic condition characterized by progressive muscle weakness.
“This is one of the first programs to receive platform technology designation and an important recognition by FDA of the reproducibility and adaptability of this technology across multiple therapeutic programs,” said Louise Rodino-Klapac, chief scientific officer and head of research & development at Sarepta. “The designation underscores and reinforces the consistency of the data we have seen with this AAVrh74 in multiple clinical programs and is yet another example of Sarepta’s continued commitment to accelerating the development of potentially transformative treatments for patients with rare genetic diseases like LGMD type 2E/R4.”
The Platform Technology Designation recognizes that the same core components – namely the rAAVrh74 vector, the promoter, and the manufacturing process – can be applied across multiple products within Sarepta’s gene therapy pipeline. This designation is intended to enable efficiencies in both regulatory review and development, particularly for Chemistry, Manufacturing, and Controls and nonclinical aspects of new products utilizing the same platform. The designation does not confer automatic approval or expedited review, but allows for potential streamlining of regulatory processes across therapies using the shared technology.
RFK Jr signals support for experimental stem cell treatments
Speaking in a podcast hosted by Gary Brecka, HHS Secretary RFK Jr says that he wants “to end the war at FDA against alternative medicine” – including stem cells. He explained that it was the FDA’s job to “do the science on these kind of issues” and to inform the public, but the agency shouldn’t dictate what physicians can and cannot prescribe.
“If you want to take an experimental drug…you ought to be able to do that. You shouldn't have to go to Antigua to get stem cells, which I had to do for my throat. And they helped me enormously,” said RFK Jr during the podcast.
He added that he didn’t want to have the “Wild West,” but the FDA should “respect the intelligence of the American people” to explore the outcomes that could benefit them the most. “You ought to rely on democracy and the good sense of the American people, and the drive that we all have to take care of ourselves, and God's gift to us of a healthy body that has its own set of defenses that we need to respect,” he said.
During the podcast, RFK Jr spoke about the health crisis in America, emphasizing the nutrient-poor, chemically-laden food supply and the “corruption” in public health agencies such as the FDA and CDC.
Armored next-generation CAR Ts
Elpis Biopharmaceuticals and Singapore General Hospital have agreed a research collaboration to jointly develop next-generation allogeneic CAR technologies, including an armored CAR-γδT (gamma-delta-T) for acute myeloid leukemia and a bi-specific CAR-γδT for multiple myeloma. Elpis will provide its proprietary platforms, which are designed to enhance efficacy and overcome the immunosuppressive tumor microenvironment, while Singapore General Hospital will contribute its clinical and translational research expertise. A key focus of the collaboration will be multi-mechanism engineering strategies to improve safety, persistence, and antitumor activity in allogeneic CAR-T products.
Research
Gene therapy against Alzheimer’s disease
Researchers at the University of California San Diego have developed a gene therapy for Alzheimer’s disease that is showing promising results in mice. Delivering the treatment at the symptomatic stage of the disease helped to preserve hippocampal-dependent memory.
“Hippocampal delivery of AAV9-Synapsin-Cav-1 (SynCav1) at presymptomatic age (3 months) significantly attenuated cognitive deficits, prevented neurodegenerative-related synaptic loss, and maintained mitochondrial dynamics and function at symptomatic stage,” says the research paper.
A statement from the university also added, “Compared to healthy mice of the same age, the treated mice also had a similar pattern of gene expression, suggesting that the treatment has the potential to alter the behavior of diseased cells to restore them to a healthier state.”
The researchers have previously shown that Cav-1 has an important role in regulating synaptic function and signal transduction.
Understanding the dysfunction of edited blood stem cells
According to researchers at San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) in Milan, CRISPR-Cas9 gene editing in combination with AAV6 vectors can trigger inflammatory and senescence-like responses in blood stem cells, and compromise their long-term ability to regenerate the blood system. The team tested two strategies to overcome the problem: transient p53 inhibition and use of anti-inflammatory agents, particularly the IL-1 receptor antagonist Anakinra.
“Both approaches significantly reduced senescence markers in edited HSPCs and improved their ability to regenerate a healthy, diverse blood system in preclinical models. Importantly, Anakinra also reduced potential genotoxic events, such as large deletions and translocations, suggesting a safer profile compared to p53 inhibition alone,” says Anastasia Conti, first author of the study.
