FDA Grants Platform Technology Tag to Sarepta's Gene Therapy Vector

Sarepta Therapeutics SRPT announced that the FDA has granted platform-technology designation to its viral vector rAAVrh74, used in the investigational gene therapy SRP-9003. This therapy is being evaluated in a late-stage study for treating a rare genetic disorder called limb-girdle muscular dystrophy (LGMD).

A viral vector is a modified virus used to deliver genetic material into cells for therapeutic purposes.

How Does This Benefit Sarepta?

The platform-technology designation is designed to streamline drug development by allowing companies to reuse data from a validated technology — in this case, Sarepta’s rAAVrh74 viral vector — across multiple drug programs. For Sarepta, this means that clinical and manufacturing data generated for SRP-9003 can now be used to support other therapies using the same viral vector. This significantly cuts down on repetitive studies and can potentially speed up development timelines for future therapies using the same platform.

Per Sarepta, the designation is one of the first given to a drug program since the initiative was launched by the FDA last year to support the efficient development of innovative treatments, particularly in areas of high unmet need like rare genetic diseases.

More on SRPT’s SRP-9003

Sarepta is currently evaluating the gene therapy in the phase III EMERGENE study for treating patients with LGMD type 2E/R4 (LGMD2E/R4, or beta sarcoglycanopathy). The study’s primary endpoint is the biomarker expression of the beta-sarcoglycan protein, whose absence is the sole cause of the disease. Data from this study is expected later this month.

If the data is positive, Sarepta intends to submit a regulatory filing seeking accelerated approval for SRP-9003 in the given indication before the end of the year.

SRPT Stock’s Performance

Year to date, Sarepta’s shares have plunged 68% compared with the industry’s 4% decline.

Recent Developments Around SRPT Stock

Though Sarepta remains a dominant player in the Duchenne muscular dystrophy (DMD) space, there is currently a negative sentiment around the stock. This is due to the safety concerns around Elevidys, its one-shot gene therapy for DMD.

In March, Sarepta reported the death of a young patient after treatment with Elevidys. While the cause was acute liver failure — a known risk of Elevidys — the company believes that the patient’s recent CMV infection might have contributed to the outcome. Based on this event, the EMA recently placed a clinical hold on all Elevidys-related studies. An investigation into the patient’s death is currently ongoing.

Elevidys is currently the first and only one-time gene therapy for DMD approved in the United States. Since its commercial launch in June 2023, the therapy has shown blockbuster potential. Sarepta generated around $821 million from Elevidys sales in 2024, up from $200 million in the year-ago period.

Despite this strong commercial performance, the recent adverse event has raised concerns among investors and clinicians. There is growing apprehension that physicians may become more cautious in prescribing Elevidys, potentially limiting market adoption in the United States. The clinical hold further delays the launch plans for the gene therapy in Europe.

Elevidys is a major revenue driver for Sarepta. During the first quarter of 2025, the therapy accounted for over half of the company’s topline. Due to safety concerns around Elevidys, the company revised its sales guidance for full-year 2025 to a range of $2.3–2.6 billion, a significant decline from the previous forecast of $2.9-$3.1 billion. Any additional safety concern or regulatory hurdle could pose significant risks to SRPT’s revenue growth and stock performance. These concerns are the primary reasons behind the sharp decline in Sarepta’s share price.

Sarepta developed Elevidys in partnership with pharmaceutical giant Roche RHHBY. In 2019, the company and Roche entered into a licensing agreement to develop Elevidys. Per the agreement, Roche has exclusive rights to launch and market the therapy in non-U.S. markets.

SRPT’s Zacks Rank

Sarepta currently carries a Zacks Rank #5 (Strong Sell).

Sarepta Therapeutics, Inc. Price

 

Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote

Key Picks Among Biotech Stocks

Some better-ranked stocks from the sector are Agenus AGEN and Immunocore IMCR. While AGEN sports a Zacks Rank #1 (Strong Buy) at present, IMCR carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, loss estimates for Immunocore’s 2025 have improved from $1.57 to 86 cents per share. Loss per share estimates for 2026 have narrowed from $1.80 to $1.34 during the same period. IMCR stock has gained around 28% year to date.

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Agenus’ earnings beat estimates in two of the trailing four quarters and missed the mark on the other two occasions, delivering an average negative surprise of 22.71%.

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