Dae Gon Ha

David Hoang

Eric Joseph

Gil Blum

Josh Schimmer

Mani Foroohar

Patrick Dolezal

Raju Prasad

Yaron Werber

- The U.S. Food and Drug Administration (FDA) has accepted Rocket Pharmaceuticals' resubmitted Biologics License Application (BLA) for **Kresladi**, a gene therapy for severe LAD-I, with a decision deadline set for **March 28, 2026**.  **[1281895_c69def97a4b24e7684fadbb2c23ebf53]**  This follows an earlier FDA rejection in June 2024.  **[1281895_c69def97a4b24e7684fadbb2c23ebf53]** 
- Kresladi demonstrated **100% survival at 12 months post-treatment** in clinical trials, and an analyst projects **peak sales of $294 million in 2031** for the therapy.  **[1281895_c69def97a4b24e7684fadbb2c23ebf53]** 
- Rocket Pharmaceuticals, a **clinical-stage company with no current revenue**, has a **strong balance sheet** with a current ratio of **6.39** and a debt-to-equity ratio of **0.07**.  **[1281895_c69def97a4b24e7684fadbb2c23ebf53]**  The company plans a **30% workforce reduction** to extend its operational runway into **Q2 2027** and prioritize its AAV cardiovascular pipeline and Kresladi regulatory efforts.  **[1281895_c69def97a4b24e7684fadbb2c23ebf53]**

Rocket Pharmaceuticals' Gene Therapy Application Accepted by FDA

- **Rocket Pharmaceuticals (RCKT)** announced on **October 14, 2025**, that the **FDA accepted the resubmission of its Biologics License Application (BLA) for KRESLADI™** (marnetegragene autotemcel; marne-cel).  **[1281895_0001140361-25-038030_ef20057000_ex99-1.htm:0]** **[1281895_0001140361-25-038030_ef20057000_8k.htm:1]** 
- KRESLADI™ is an investigational gene therapy for **severe Leukocyte Adhesion Deficiency-I (LAD-I)**, a rare genetic immune disorder.  **[1281895_0001140361-25-038030_ef20057000_ex99-1.htm:0]** **[1281895_0001140361-25-038030_ef20057000_8k.htm:1]** 
- The FDA has set a **PDUFA target action date of March 28, 2026**, for the BLA, and Rocket is **eligible for a Rare Pediatric Disease Priority Review Voucher (PRV)** upon approval.  **[1281895_0001140361-25-038030_ef20057000_ex99-1.htm:0]** **[1281895_0001140361-25-038030_ef20057000_8k.htm:1]** 
- The BLA is supported by positive clinical data from a Phase 1/2 study, which showed **100% overall survival at 12 months post-infusion** for all enrolled patients, with KRESLADI™ being well tolerated and meeting all primary and secondary endpoints.  **[1281895_0001140361-25-038030_ef20057000_ex99-1.htm:0]**

Rocket Pharmaceuticals Announces FDA Acceptance of KRESLADI™ BLA Resubmission

- **Rocket Pharmaceuticals** announced that the **U.S. Food and Drug Administration (FDA) has accepted the resubmission of the Biologics License Application (BLA) for KRESLADI™** (marnetegragene autotemcel; marne-cel), an investigational gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I).  **[1281895_313c4012e3b0437ca31698e581a89056_0]** 
- The **Prescription Drug User Act (PDUFA) target action date** for KRESLADI™ is set for **March 28, 2026**.  **[1281895_313c4012e3b0437ca31698e581a89056_0]** 
- The BLA is supported by positive clinical efficacy and safety data from a global Phase 1/2 study, which demonstrated **100% overall survival at 12 months post-infusion** for all enrolled patients, meeting all primary and secondary endpoints.  **[1281895_313c4012e3b0437ca31698e581a89056_0]** 
- Rocket Pharmaceuticals is **eligible for a Rare Pediatric Disease Priority Review Voucher** if KRESLADI™ is approved.  **[1281895_313c4012e3b0437ca31698e581a89056_0]**

- **Rocket Pharmaceuticals** has **voluntarily withdrawn** its biologics license application for **RP-L102 (Fanskya)**, a gene therapy for **Fanconi Anemia**.  **[1281895_f0ceb85301d1458c8f7eef716d524ea8]** 
- This decision is due to a **strategic shift** to focus on programs with clearer regulatory and commercial prospects, following a **corporate restructuring in July 2025** that included **layoffs** and a pivot toward **cardiovascular disease programs**.  **[1281895_f0ceb85301d1458c8f7eef716d524ea8]** 
- The withdrawal is **not related to safety or efficacy concerns**, as data continue to show the therapy is generally well-tolerated with a favorable risk-benefit profile.  **[1281895_f0ceb85301d1458c8f7eef716d524ea8]** 
- While internal investment in RP-L102 has ceased, Rocket Pharmaceuticals is **exploring external partnerships** to advance the therapy.  **[1281895_f0ceb85301d1458c8f7eef716d524ea8]**

Rocket Pharmaceuticals Withdraws Fanconi Anemia Gene Therapy Filing Amid Strategic Shift

- Rocket Pharmaceuticals has **shifted its focus to its AAV cardiac gene therapy portfolio**, which includes programs for Danon, PKP2, and BAG3, collectively representing over **100,000 patients** in the U.S. and Europe.  **[1281895_tx_380806_0]** **[1281895_tx_380806_6]** 
- The **clinical hold on the Danon program was lifted in less than three months** following the identification of capillary leak syndromes linked to a C3 inhibitor.  **[1281895_tx_380806_0]** **[1281895_tx_380806_1]**  The revised protocol involves a recalibrated dose closer to **4e13**, removal of the C3 inhibitor, and a lower threshold for C5 inhibition.  **[1281895_tx_380806_1]** 
- The company plans to treat the **next three Danon patients in early 2026**, with the FDA not requiring enrollment of more than 12 patients for the pivotal trial.  **[1281895_tx_380806_4]** **[1281895_tx_380806_5]** 
- For the PKP2 program, the prophylaxis regimen is similar to the revised Danon program, and the next milestone is **FDA alignment on the Phase 2 pivotal trial design**.  **[1281895_tx_380806_6]**  The BAG3 program is expected to start its Phase 1 trial next year.  **[1281895_tx_380806_6]**

ROCKET PHARMACEUTICALS (RCKT)