Cell and Gene Therapy Video Recap – October 16, 2025

Here’s your biweekly look at the latest breakthroughs in Cell and Gene Therapy. From cutting-edge research to pivotal approvals, we highlight the stories defining this fast-evolving space.

Watch Our Video Summary Capturing Top Cell and Gene News from the Last Two Weeks

Top Stories Covered in This Video

🧬 Rocket Pharmaceuticals BLA Resubmission for Kresladi Accepted by FDA [1] [US • 14 Oct 2025]

https://www.pharmiweb.com/press-release/2025-10-14/rocket-pharmaceuticals-announces-fda-acceptance-of-bla-resubmission-of-kresladi-for-the-treatment-o
Key point: The therapy has shown a 100% survival rate at 12 months post-infusion in clinical trials.
Context: Rocket Pharmaceuticals announced the FDA’s acceptance of its resubmission for Kresladi™, a gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I).
Implication: The approval of Kresladi™ would offer a life-saving treatment for children with LAD-I, with a PDUFA target date of March 28, 2026.

🧬 Abeona Therapeutics’ ABO-503 Chosen for FDA’s Rare Disease Endpoint Advancement Pilot Program [2] [US • 14 Oct 2025]

https://www.ophthalmologytimes.com/view/abeona-therapeutics-abo-503-chosen-for-fda-s-rare-disease-endpoint-advancement-pilot-program
Key point: The program will facilitate enhanced collaboration with the FDA to develop novel efficacy endpoints for ABO-503.
Context: Abeona Therapeutics’ ABO-503 gene therapy for X-linked retinoschisis (XLRS) has been selected for the FDA’s Rare Disease Endpoint Advancement (RDEA) Pilot Program.
Implication: This designation will expedite ABO-503’s development, offering hope to patients with XLRS, a rare retinal disease.

🧬 Regeneron’s DB-OTO Shows Hearing Improvement in Otoferlin-Related Hearing Loss [3] [US • 14 Oct 2025]

https://www.cgtlive.com/view/regeneron-gene-therapy-db-oto-improves-speech-perception-children-otoferlin-related-hearing-loss
Key point: 11 of 12 patients in the Phase 1/2 CHORD trial showed clinically meaningful hearing improvements.
Context: Regeneron’s DB-OTO gene therapy improved hearing and speech perception in children with otoferlin-related hearing loss.
Implication: This advancement could set a new standard in genetic treatments for hearing loss, with a regulatory application expected by late 2025.

🧬 Bristol Myers Squibb Acquires Orbital Therapeutics to Enhance RNA Platform [4] [US • 09 Oct 2025]

https://www.businesswire.com/news/home/20251009968216/en/Bristol-Myers-Squibb-Strengthens-and-Diversifies-Cell-Therapy-Portfolio-with-Acquisition-of-Orbital-Therapeutics
Key point: The acquisition includes OTX-201, a next-generation CAR-T therapy for autoimmune diseases, using Orbital’s proprietary RNA platform.
Context: Bristol Myers Squibb has acquired Orbital Therapeutics to advance RNA-based CAR-T therapies.
Implication: This deal strengthens BMS’s leadership in cell therapy and expands access to innovative treatments.

🧬 AskBio Reports Safety Data for AB-1003 in Limb-Girdle Muscular Dystrophy [5] [US • 14 Oct 2025]

https://www.clinicaltrialsarena.com/news/askbio-ab1003-safety-muscular-dystrophy/?cf-view
Key point: The therapy demonstrated a favorable safety profile over 52 weeks of monitoring.
Context: AskBio presented safety data from its Phase I/II study of AB-1003 for limb-girdle muscular dystrophy (LGMD), showing no dose-limiting toxicities or serious adverse events.
Implication: This could pave the way for further development of gene therapy for LGMD, a debilitating muscle disorder.

🧬 Shape Therapeutics Shares Data for Non-Invasive Parkinson’s Disease Treatment [6] [US • 10 Oct 2025]

https://www.globenewswire.com/news-release/2025/10/10/3164749/0/en/Shape-Therapeutics-Shares-Breakthrough-Preclinical-Data-for-Non-Invasive-Treatment-of-Parkinson-s-Disease-Using-a-Brain-Targeted-AAV-Gene-Therapy-at-the-European-Society-of-Gene-Ce.html
Key point: The therapy showed promising results in targeting the substantia nigra, a key area affected by Parkinson’s disease.
Context: Shape Therapeutics shared breakthrough preclinical data for a non-invasive gene therapy targeting Parkinson’s Disease using a novel AAV5 capsid.
Implication: This could offer a new, less invasive treatment option for Parkinson’s patients.

Why it matters

Rocket Pharmaceuticals’ resubmission of Kresladi™ brings hope for children with severe LAD-I, a life-threatening immune disorder.
Abeona Therapeutics’ participation in the RDEA program could accelerate the development of gene therapies for rare retinal diseases.
Regeneron’s DB-OTO marks a breakthrough in genetic treatments for hearing loss, potentially eliminating the need for cochlear implants.
Bristol Myers Squibb’s acquisition of Orbital Therapeutics strengthens its RNA-based gene therapy capabilities, especially in autoimmune diseases.
AskBio’s AB-1003 shows promise as a treatment for LGMD, with a favorable safety profile.
Shape Therapeutics’ non-invasive Parkinson’s disease treatment could offer a transformative option for patients.

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FAQ

What are the key findings from Rocket Pharmaceuticals’ Kresladi™ BLA resubmission?

The FDA accepted the resubmission of Kresladi™ for LAD-I, with promising survival data and no serious adverse events in clinical trials. [1]

How will the FDA’s Rare Disease Endpoint Advancement Program benefit Abeona’s ABO-503?

The program will expedite the development of ABO-503 for XLRS by facilitating collaboration with the FDA on novel efficacy endpoints. [2]

What was the outcome of Regeneron’s DB-OTO trial?

DB-OTO showed significant hearing improvements in children with otoferlin-related hearing loss, with 11 of 12 patients demonstrating meaningful progress. [3]

How did Bristol Myers Squibb’s acquisition of Orbital Therapeutics impact its RNA-based therapies?

The acquisition strengthens BMS’s RNA-based gene therapy capabilities and expands access to next-generation CAR-T therapies. [4]

What are the safety outcomes of AskBio’s AB-1003 in LGMD?

AB-1003 showed a favorable safety profile with no dose-limiting toxicities or serious adverse events over 52 weeks. [5]

What does Shape Therapeutics’ breakthrough preclinical data mean for Parkinson’s disease treatment?

Shape’s novel AAV5 capsid demonstrated promising results in targeting the substantia nigra, a critical area for Parkinson’s disease, offering a potential new non-invasive treatment. [6]

Entities / Keywords

Rocket Pharmaceuticals; Kresladi; LAD-I; Abeona Therapeutics; ABO-503; RDEA Program; Regeneron; DB-OTO; Otoferlin-Related Hearing Loss; Bristol Myers Squibb; Orbital Therapeutics; RNA-Based Gene Therapy; CAR-T; Autoimmune Diseases; AskBio; AB-1003; Limb-Girdle Muscular Dystrophy; Shape Therapeutics; Parkinson’s Disease; AAV Gene Therapy