FDA Activity Recap: June 2025 Features New Platform Designation, IND Clearance, and More

Last month, June 2025, the CGTLive^® team was diligently tracking the FDA's activities related to the development of cell and gene therapies for the treatment of rare, complex, and otherwise challenging diseases and disorders.

The agency has continued to ramp up its activities around these therapies as more of them progress through the pipeline in tandem. Last month proved no different, with the FDA granting its new platform designation to Sarepta Therapeutics for bidridistrogene xeboparvovec (also known as SRP-9003)'s rAAVrh74 viral vector and clearing an investigational new drug (IND) application for Nuevocor's LMNA-related dilated cardiomyopathy (LMNA DCM) gene therapy NVC-001. Our team has highlighted these, and several other important actions, below.

Click the read more buttons for more details and information about each update.

Sarepta Garners Platform Technology Designation for Limb-Girdle Muscular Dystrophy Gene Therapy Vector

June 10, 2025 — Sarepta Therapeutics has received platform technology designation from the FDA for bidridistrogene xeboparvovec (also known as SRP-9003)'s rAAVrh74 viral vector.

SRP-9003 is an investigational adeno-associated virus (AAV) vector-based gene therapy product in development for limb-girdle muscular dystrophy Type 2E (LGMD2E/R4, also known as beta sarcoglycanopathy). The platform technology designation is a new designation established by the FDA that is intended to allow companies developing a product that uses a well-understood technology that has been previously used in other programs to utilize data regarding that technology from the other programs in support of investigational new drug applications and biologic license applications. Notably, rAAVrh74 is also used in Sarepta’s delandistrogene moxeparvovec-rokl (marketed as Elevidys), an FDA-approved gene therapy intended to treat Duchenne muscular dystrophy (DMD).

“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,” Louise Rodino-Klapac, PhD, the chief scientific officer and head of research & development at Sarepta, said in a statement. “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.”

Nuevocor Snags IND Clearance for Gene Therapy Trial in LMNA DCM

June 11, 2025 — Singapore-based company Nuevocor has received clearance from the FDA for an IND application for NVC-001, an AAV vector-based gene therapy intended to treat LMNA DCM.

In light of the IND clearance, the company intends to go forward with plans for a first-in-human phase 1/2 ascending-dose clinical trial. Nuevocor expects to launch the 52-week trial, which will be open to adult patients, early next year. Participants in the trial will receive a 1-time dose of the gene therapy product via intravenous infusion.

LMNA DCM, which is found in approximately 100,000 people across the United States and Europe, is characterized by nuclear envelope disruption caused by mutations in LMNA, a gene necessary for nuclear envelope integrity. The condition results in heart muscle weakening, arrhythmias, and eventually end-stage heart failure.

FDA Indicates AdComm Not Currently Required for Capricor’s BLA for DMD Cardiomyopathy Cell Therapy Deramiocel

June 24, 2025 — The FDA has indicated an advisory committee (AdComm) meeting regarding the biologics license application (BLA) for Capricor Therapeutics’ Deramiocel (also known as CAP-1002), an investigational allogeneic cardiosphere-derived cell therapy under review for the treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy, is not currently required.

The Prescription Drug User Fee Act (PDUFA) action date for the BLA continues to be set for August 31, 2025, and an in-person late-cycle review meeting for the company and the FDA is set for the middle of July. The FDA had previously told Capricor in May 2025 that an AdComm would be required for the BLA, but at the time no date had been announced for the AdComm and the agency had not identified any major deficiences in the BLA package.

“We remain confident in the strength of our submission and continue to advance toward potential approval, with our next major step being the upcoming late-cycle review meeting,” Linda Marbán, PhD, thechief executive officer of Capricor, said in a statement. “To date, all regulatory milestones have proceeded as expected, including a successful prelicense inspection and a mid-cycle review with no major issues. Our application remains under Priority Review, and we believe we are well positioned as we move toward our PDUFA date. We continue to work closely with the FDA and remain encouraged by the progress of our review.”

FDA Announces Investigation of Deaths Following Treatment With Sarepta’s DMD Gene Therapy Elevidys

June 25, 2025 — The FDA has announced that it is investigating the 2 deaths that Sarepta Therapeutics has reported in patients who received treatment with delandistrogene moxeparvovec-rokl (Elevidys), the company’s marketed AAV vector-based gene therapy for DMD.

Notably, both deaths occurred in patients who were nonambulatory at the time of treatment and both patients had been hospitalized within 2 months of treatment in relation to raised levels of transaminases. Both patients’ deaths were attributed to acute liver failure (ALF). The FDA stated that it is assessing the risk of ALF that results in hospitalization or death after treatment with the gene therapy and whether additional regulatory measures will be necessary.

“The United States prescribing information includes information on the risk of acute serious liver injury following treatment with Elevidys under Warnings and Precautions, Adverse Reactions and Patient Counseling Information, but does not include warnings regarding liver failure or death,” the agency stated in a press release.

enGene Snags FDA RMAT Designation for Bladder Cancer Gene Therapy Detalimogene

June 25, 2025 — The FDA has granted regenerative medicine advanced therapy (RMAT) designation to enGene's detalimogene voraplasmid (also known as detalimogene, and previously referred to as EG-70), an investigational nonviral immuno-oncology gene therapy, for the treatment of high-risk bacillus Calmette-Guérin (BCG)-unresponsive nonmuscle-invasive bladder cancer (NMIBC) with carcinoma in situ.

“Detalimogene has the potential to deliver what NMIBC patients and their physicians have long needed—a nonviral option with optimized delivery to the bladder, safety in handling, and ease of administration,” Ron Cooper, BSc, the chief executive officer of enGene, told CGTLive in response to a request for comment. “We’re pleased to receive this designation for detalimogene and are encouraged that we’re one step closer to potentially providing NMIBC patients a new option that may help transform the treatment landscape.”

Detalimogene is expected to be able to be utilized in urology clinics in a streamlined manner and is intended to provide an additional option for patients who might have few options besides radical cystectomy. The gene therapy is currently being evaluated in the phase 1/2 LEGEND clinical trial (NCT04752722). According to enGene, data from this study informed the FDA’s decision to grant RMAT designation to detalimogene.