Cell and Gene Therapy Today - October 16, 2025

This week’s Cell and Gene therapy news covers the latest developments, updates, and progress from leading biopharma companies.

In Today’s Newsletter

🧬 Rocket Pharmaceuticals BLA Resubmission for Kresladi Accepted by FDA [1]

https://www.pharmiweb.com/press-release/2025-10-14/rocket-pharmaceuticals-announces-fda-acceptance-of-bla-resubmission-of-kresladi-for-the-treatment-o
Context: Rocket Pharmaceuticals announced FDA acceptance of the resubmission for Kresladi™, a gene therapy for severe Leukocyte Adhesion Deficiency-I (LAD-I).
Key point: The therapy has shown promising results with a 100% survival rate at 12 months post-infusion in clinical trials.
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 Pilot Program [2]

https://www.ophthalmologytimes.com/view/abeona-therapeutics-abo-503-chosen-for-fda-s-rare-disease-endpoint-advancement-pilot-program
Context: Abeona Therapeutics’ ABO-503 gene therapy for X-linked retinoschisis (XLRS) has been selected for the 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.
Implication: This designation will expedite ABO-503’s development, bringing hope for patients with XLRS, a rare retinal disease.

🧬 Regeneron’s DB-OTO Shows Hearing Improvement in Otoferlin-Related Hearing Loss [3]

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

🧬 Krystal Biotech Receives FDA Platform Technology Designation for KB801 [4]

https://www.manilatimes.net/2025/10/14/tmt-newswire/globenewswire/krystal-biotech-receives-fda-platform-technology-designation-for-hsv-1-viral-vector-used-in-kb801-for-the-treatment-of-neurotrophic-keratitis/2200288
Context: Krystal Biotech’s HSV-1 viral vector used in KB801 for neurotrophic keratitis has received FDA platform technology designation.
Key point: This designation will streamline the regulatory process for KB801, supporting faster development for the treatment of neurotrophic keratitis.
Implication: It could accelerate the delivery of a much-needed therapy for patients suffering from this severe eye condition.

🧬 Genethon Confirms Long-Term Efficacy of Micro-Dystrophin Gene Therapy for DMD [5]

https://www.businesswire.com/news/home/20251009554793/en/Genethon-Confirms-2-Year-Efficacy-in-Duchenne-Muscular-Dystrophy-Patients-Treated-With-Its-Low-Dose-Micro-Dystrophin-Gene-Therapy-GNT0004-at-the-ESGCT-32nd-Annual-Congress
Context: Genethon presented data confirming two years of sustained efficacy from its low-dose micro-dystrophin gene therapy in Duchenne muscular dystrophy (DMD) patients.
Key point: The therapy showed a 75% reduction in muscle damage markers and significant motor function improvement.
Implication: These results could pave the way for approval and offer hope for children with DMD.

💉 Janssen/Legend Biotech Add Warning to Carvykti Label for Fatal Intestinal Disorder [6]

https://www.fiercepharma.com/pharma/fda-adds-warning-label-jjs-carvykti-potentially-fatal-intestinal-disorder
Context: The FDA has added a boxed warning for immune effector cell-associated enterocolitis (IEC-EC) to the label of Carvykti.
Key point: The disorder, which can be fatal, has been observed in some patients after treatment with Carvykti.
Implication: The warning may influence the prescribing and use of Carvykti, especially in patients with underlying risk factors.

🧬 Bristol Myers Squibb Acquires Orbital Therapeutics to Enhance RNA Platform [7]

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

🧬 Novo Nordisk Shuts Down Cell Therapy Unit, Ending Type 1 Diabetes Cure Research [8]

https://www.fiercebiotech.com/biotech/novo-shutters-cell-therapy-unit-ending-work-potential-type-1-diabetes-cure
Context: Novo Nordisk has ceased its cell therapy efforts, including a promising Type 1 diabetes cure program.
Key point: This decision is part of a major reorganization to save costs, affecting approximately 250 employees.
Implication: This move marks a significant setback in the pursuit of cell-based therapies for diabetes.

🧬 CRISPR Therapeutics Presents Preclinical Data for CTX460 in AATD [9]

https://www.globenewswire.com/news-release/2025/10/10/3164819/0/en/CRISPR-Therapeutics-Presents-New-Preclinical-Data-for-CTX460-Demonstrating-In-Vivo-Gene-Correction-of-Alpha-1-Antitrypsin-Deficiency-AATD-Utilizing-Novel-SyNTase-Editing-Platform.html
Context: CRISPR Therapeutics presented new data for CTX460, a gene therapy for Alpha-1 Antitrypsin Deficiency (AATD), showing high efficiency and durability in preclinical models.
Key point: The therapy achieved over 90% mRNA correction and a 5-fold increase in total AAT levels.
Implication: CTX460 could become a leading therapy for AATD, a genetic disorder causing lung and liver damage.

🧬 Shape Therapeutics Shares Data for Non-Invasive Parkinson’s Disease Treatment [10]

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
Context: Shape Therapeutics shared breakthrough preclinical data for a non-invasive gene therapy targeting Parkinson’s Disease using a novel AAV5 capsid.
Key point: The therapy showed promising results in targeting the substantia nigra, a key area for Parkinson’s disease.
Implication: This could offer a new, less invasive treatment option for Parkinson’s patients.

🧬 Encoded Therapeutics Demonstrates Potential for Non-Opioid Chronic Pain Treatment [11]

https://www.biospace.com/press-releases/new-preclinical-data-from-encoded-therapeutics-demonstrate-therapeutic-potential-of-its-one-time-non-opioid-gene-therapy-candidate-for-chronic-pain
Context: Encoded Therapeutics presented preclinical data showing that its gene therapy candidate can achieve durable pain relief in chronic pain models.
Key point: The therapy targets SCN9A, a key gene involved in pain signaling, and demonstrated robust effects in non-human primates.
Implication: This could lead to a non-opioid treatment for chronic pain, offering a safe and effective alternative to current therapies.

🧬 AskBio Reports Safety of AB-1003 for Limb-Girdle Muscular Dystrophy [12]

https://www.clinicaltrialsarena.com/news/askbio-ab1003-safety-muscular-dystrophy/?cf-view
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.
Key point: The therapy demonstrated a favorable safety profile over 52 weeks of monitoring.
Implication: This could pave the way for further development of gene therapy for LGMD, a debilitating muscle disorder.

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.
Krystal Biotech’s platform technology designation could fast-track the development of its HSV-1-based gene therapies.
Genethon’s micro-dystrophin gene therapy offers long-term improvements in Duchenne muscular dystrophy, demonstrating its potential for broad patient benefit.
Janssen’s Carvykti faces new safety warnings, which may influence prescribing practices for multiple myeloma.
Bristol Myers Squibb’s acquisition of Orbital Therapeutics strengthens its RNA-based gene therapy capabilities, especially in autoimmune diseases.
Novo Nordisk’s cell therapy exit highlights the challenges in developing Type 1 diabetes cures, marking a setback for cell therapy in diabetes treatment.

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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 Krystal Biotech’s HSV-1 platform technology designation impact KB801?

The designation supports the development of KB801 for neurotrophic keratitis, accelerating regulatory processes and reducing time to market. [4]

What were the long-term results from Genethon’s DMD gene therapy trial?

Genethon’s therapy showed sustained improvements in motor function and a significant reduction in muscle damage markers over two years. [5]

Why did the FDA add a warning to Carvykti’s label?

The warning highlights the risk of a potentially fatal intestinal disorder (IEC-EC), which has occurred in some Carvykti patients. [6]

Entities / Keywords

Rocket Pharmaceuticals; Kresladi; LAD-I; Abeona Therapeutics; ABO-503; RDEA Program; Regeneron; DB-OTO; Otoferlin-Related Hearing Loss; Krystal Biotech; HSV-1; KB801; Neurotrophic Keratitis; Genethon; Micro-Dystrophin; Duchenne Muscular Dystrophy; Janssen; Carvykti; Intestinal Disorder; Bristol Myers Squibb; Orbital Therapeutics; RNA-Based Gene Therapy; Novo Nordisk; Cell Therapy; Type 1 Diabetes; CRISPR Therapeutics; CTX460; AATD; Shape Therapeutics; Parkinson’s Disease; AAV Gene Therapy; Encoded Therapeutics; Chronic Pain; AskBio; AB-1003; Limb-Girdle Muscular Dystrophy.