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RCKT's IND for RP-A701 in Dilated Cardiomyopathy Gets FDA Clearance

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Key Takeaways

  • RCKT received FDA clearance to start clinical trials for its gene therapy candidate RP-A701.
  • The phase I study will assess RP-A701's safety, biological activity and preliminary efficacy.
  • Participants will receive a single RP-A701 dose, with multiple cardiac health markers being monitored.

Rocket Pharmaceuticals (RCKT - Free Report) announced that the FDA has cleared its investigational new drug (IND) application seeking to begin clinical studies on its first-in-class gene therapy candidate, RP-A701.

The company is planning to conduct a phase I study evaluating RP-A701, an AAVrh.74-based gene therapy for the treatment of BAG3-associated dilated cardiomyopathy (BAG3-DCM), a rare and inherited heart condition, which holds significant mortality and morbidity.

BAG3-associated dilated cardiomyopathy is a genetic heart disease causing enlarged, weakened heart muscles, leading to heart failure.

The multi-center, dose-escalation phase I study will evaluate the safety, biological activity and preliminary efficacy of RP-A701 in adults with BAG3-DCM in the United States. Participants in the study will get a single RP-A701 dose. The study will track BAG3 protein expression, cardiac biomarkers, and disease progression indicators.

Besides RP-A701, RCKT is developing another AAV-based gene therapy candidate, RP-A601, which is in early-stage development for arrhythmogenic cardiomyopathy (ACM).

Year to date, shares of Rocket Pharmaceuticals have plunged 80.5% compared with the industry’s decline of 3.6%.

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RCKT's Recent Pipeline Setbacks

Setbacks related to pipeline candidates have been a major concern for Rocket Pharmaceuticals in recent times.

In May 2025, the company voluntarily paused dosing in the pivotal phase II study evaluating its gene therapy candidate, RP-A501, for treating patients with Danon disease, following a patient death. The FDA placed a clinical hold on the study. Shares of RCKT tanked on this news.

The company is working with the FDA, the Independent Data Safety Monitoring Committee, clinical investigators and scientific experts to restart the study as soon as possible, while ensuring the safety of all patients in the study.

Last June, the FDA issued a complete response letter (CRL) to the biologics license application (BLA) seeking approval for Kresladi to treat LAD-I, a rare genetic disorder.

This was the second time that the FDA had requested additional information from the company on Kresladi’s CMC. In February 2024, the FDA extended the review period of the Kresladi BLA by an additional three months.

In light of the recent pipeline setbacks, the successful development of its other gene therapy candidates remains a key focus area for the company.

RCKT's Zacks Rank & Stocks to Consider

Rocket Pharmaceuticals currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Exelixis (EXEL - Free Report) , Spero Therapeutics (SPRO - Free Report) and Puma Biotechnology (PBYI - Free Report) , each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

In the past 60 days, estimates for Exelixis’ earnings per share have increased from $2.31 to $2.64 for 2025. During the same time, earnings per share estimates for 2026 have increased from $2.85 to $3.13. Year to date, shares of EXEL have rallied 32.4%.

EXEL’s earnings beat estimates in each of the trailing four quarters, the average surprise being 48.60%.

In the past 60 days, estimates for Spero Therapeutics’ loss per share have narrowed from $2.32 to $1.43 for 2025. During the same time, loss per share estimates for 2026 have narrowed from $1.98 to $1.15. Year to date, shares of SPRO have surged 181.5%.

SPRO’s earnings beat estimates in two of the trailing four quarters, while missing the same in the remaining two occasions, the average surprise being 8.29%.

In the past 60 days, estimates for Puma Biotechnology’s earnings per share have increased from 60 cents to 65 cents for 2025. During the same time, earnings per share estimates for 2026 have risen from 46 cents to 51 cents. Year to date, shares of PBYI have gained 12.5%.

PBYI’s earnings beat estimates in each of the trailing four quarters, the average surprise being 171.43%.

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