This biweekly Cell and Gene Therapy video recap highlights significant regulatory actions, clinical milestones, and advancements in gene and cell therapy. Key developments include manufacturing updates, clinical trial results, and accelerated regulatory pathways, all shaping the future of medical treatments.
🎯 Watch Our Video Summary Capturing Cell and Gene Therapy News from the Last Two Weeks
🗓️ Explore details and sources
- Week 8–14 January 2026
- Weeks 15–21 January 2025
📚 See the full Cell and Gene Therapy archive on our research hub page.
Top Stories Covered in This Video
Chapters
0:00 Introduction
0:09 FDA Carves Out Manufacturing Exemptions to Accelerate CGT Development 0:35 uniQure Schedules Type A Meeting with FDA for AMT-130 Huntington’s Gene Therapy
1:02 REGENXBIO Announces Key 2026 Milestones for Duchenne Gene Therapy Program
1:27 Nanoscope Therapeutics Secures Japan’s MHLW Sakigake and Orphan Drug Designations for Retinal Gene Therapy
1:59 AstraZeneca Signs $630M Deal to Secure Full Global Rights to Armored CAR-T Therapy
2:25 AGC Biologics Celebrates US and EU Approval of Waskyra™ for Wiskott-Aldrich Syndrome
2:49 Ocugen Reports Positive Phase 2 Data for OCU410 Gene Therapy in Geographic Atrophy
3:18 Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA Following FDA Review of HOPE-3 Data
3:46 How to reach us
Transcript
Welcome to the latest edition of Gene and Cell Therapy Updates, covering breakthroughs in the past two weeks. Brought to you by LucidQuest.
The U.S. FDA has introduced more flexible regulations to accelerate the development of cell and gene therapies. Relaxations include leniency in manufacturing specifications for Phase 2 and 3 trials, which will help facilitate faster development. The goal of these changes is to streamline the approval process while ensuring safety and efficacy for CGTs.
uniQure has scheduled a Type A meeting with the FDA to discuss AMT-130, their gene therapy for Huntington’s disease. The meeting aims to resolve questions related to the Biologics License Application data package. This meeting is crucial for moving the therapy closer to accelerated approval, offering potential hope for patients with Huntington’s disease.
REGENXBIO shared promising data from the Phase 1 2 trial of RGX-202 for Duchenne muscular dystrophy. Patients showed significant improvements in motor function compared to expected disease progression, strengthening the case for accelerated approval and potential commercialization of RGX-202 in the coming years.
Nanoscope Therapeutics has secured fast-track designations for its retinal gene therapy, MCO-010, from Japan’s Ministry of Health, Labour, and Welfare. MCO-010 is the first retinal gene therapy to receive both Sakigake and orphan drug designations in Japan. These designations support expedited development and market access, positioning MCO-010 for a strong global presence.
AstraZeneca has signed a $630 million deal to acquire the remaining global rights to C-CAR031, an armored CAR-T therapy targeting GPC3 in hepatocellular carcinoma, a type of liver cancer. This acquisition strengthens AstraZeneca’s oncology portfolio, particularly in challenging cancers like liver cancer.
AGC Biologics celebrated the US and EU approval of Waskyra™ for the treatment of Wiskott-Aldrich syndrome. The company played a key role in the development of this gene therapy by providing critical manufacturing services. This approval marks a significant milestone for gene therapy in treating rare immune disorders.
Ocugen has announced positive preliminary Phase 2 data for OCU410, a gene therapy targeting geographic atrophy. OCU410 demonstrated a 46% reduction in lesion growth compared to the control group, with no serious adverse events reported. This data strongly supports continued development and regulatory filings for OCU410.
Capricor Therapeutics has provided an update on its Biologics License Application for Deramiocel following the FDA’s review of HOPE-3 trial data. The FDA has requested additional clinical data to address concerns raised in the Complete Response Letter. Capricor is working closely with the FDA to address these issues and continue the review process for this Duchenne muscular dystrophy treatment.
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Why It Matters
- Regulatory evolution is accelerating as the FDA and other regulators streamline their processes for gene therapies, ensuring more rapid access to transformative treatments.
- Breakthrough therapies for rare diseases such as Duchenne muscular dystrophy, inherited retinal diseases, and Huntington’s disease continue to gain attention, with novel gene therapies pushing the boundaries of treatment.
- Strategic collaborations are becoming increasingly vital, such as those between AstraZeneca and AbelZeta, and AGC Biologics’ ongoing partnerships, which facilitate faster development and commercialization of gene therapies.
🗓️ Explore details and sources
- Week 8–14 January 2026
- Weeks 15–21 January 2025
📚 See the full Cell and Gene Therapy archive on our research hub page.
FAQ
What does the FDA’s flexibility on CGT manufacturing mean for developers?
It eases the regulatory burden for gene therapy developers, allowing them to accelerate clinical trials without compromising safety or efficacy.
How significant is uniQure’s Type A meeting with the FDA?
It’s a key step toward obtaining regulatory clarity and moving closer to potential approval for AMT-130, a pioneering Huntington’s disease therapy.
What’s the importance of AstraZeneca securing the full rights to C-CAR031?
This acquisition strengthens AstraZeneca’s oncology portfolio, enabling them to lead in CAR-T therapies for liver cancer and other solid tumors.
How does Capricor’s update on Deramiocel affect the Duchenne community?
Capricor is working to address FDA feedback and remain on track for approval, offering hope for patients with this devastating genetic disorder.
Entities / Keywords
FDA; CGT manufacturing; AMT-130; Huntington’s disease; REGENXBIO; Duchenne muscular dystrophy; Nanoscope Therapeutics; Sakigake designation; AstraZeneca; C-CAR031; CAR-T therapy; Waskyra; Wiskott-Aldrich syndrome; OCU410; geographic atrophy; Capricor Therapeutics; Deramiocel; FDA BLA.
References
[1] https://www.biospace.com/fda/fda-carves-out-manufacturing-exemptions-for-cgts-to-accelerate-development
[2] https://www.uniqure.com/investors-media/press-releases
[3] https://www.prnewswire.com/news-releases/regenxbio-highlights-key-2026-catalysts-and-announces-positive-long-term-functional-outcomes-in-lead-duchenne-gene-therapy-program-302657988.html
[4] https://www.prnewswire.com/news-releases/nanoscope-secures-japan-mhlw-sakigake-and-orphan-drug-designations-across-inherited-retinal-diseases–a-first-for-a-retinal-gene-therapy-302664876.html
[5] https://www.fiercebiotech.com/biotech/astrazeneca-pens-630m-pact-secure-remaining-rights-armored-car-t
[6] https://www.agcbio.com/news/agc-biologics-celebrates-u.s.-and-eu-approval-of-fondazione-telethons-waskyra-treatment-for-wiskott-aldrich-syndrome
[7] https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-positive-preliminary-phase-2-data-ocu410/
[8] https://www.globenewswire.com/news-release/2026/01/20/3221849/0/en/Capricor-Therapeutics-Provides-Regulatory-Update-on-Deramiocel-BLA-Following-FDA-Review-of-HOPE-3-Topline-Data.html