Cell and Gene Therapy Today—January 15, 2026

LucidQuest

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🏛️ FDA eases certain CGT CMC rules to accelerate development [1] [US • 12 Jan 2026]

https://www.biospace.com/fda/fda-carves-out-manufacturing-exemptions-for-cgts-to-accelerate-development
Context: Agency statements describe flexibilities for individualized or small-batch CGTs.
Key point: FDA will not require some Chapter 600 manufacturing specs beyond Phase I and will allow minor manufacturing changes with supporting data.
Implication: May expand screening, initiation, and follow-up at scale.

🧬 uniQure schedules FDA Type A meeting on AMT-130 accelerated approval path (Huntington’s) [2] [09 Jan 2026]

https://www.uniqure.com/investors-media/press-releases
Context: Company seeks alignment on BLA data package for AMT-130.
Key point: A Type A meeting has been scheduled to discuss a potential accelerated approval approach for AMT-130.
Implication: May influence prescriber choice and payer reviews pending full data.

🤝 Nona Biosciences, Link Cell Therapies team on HCAb-based CAR-T discovery [3] [09 Jan 2026]

https://www.prnewswire.com/news-releases/nona-biosciences-enters-into-collaboration-agreement-with-link-cell-therapies-to-advance-car-t-cell-therapies-302656985.html
Context: Uses HCAb Harbour Mice and NonaCarFx platforms to generate CAR-T candidates.
Key point: Collaboration aims to discover fully human HCAb CAR constructs to reduce immunogenicity and improve design flexibility (financials not stated).
Implication: Signals pipeline investment and modality expansion.

🧪 RMAT for Eureka’s ECT204 ARTEMIS CAR T in advanced HCC [4] [US • 12 Jan 2026]

https://www.businesswire.com/news/home/20260112165656/en/U.S.-FDA-Grants-RMAT-Designation-to-ECT204-an-ARTEMIS-CAR-AbTCR-T-Cell-Therapy-for-Advanced-Hepatocellular-Carcinoma
Context: Based on completed Phase I cohort and additional data from ARYA-3 Phase I/II.
Key point: RMAT enables enhanced FDA interaction, potential surrogate endpoints, and priority review eligibility.
Implication: May influence prescriber choice and payer reviews pending full data.

💓 Lexeo taps J&J’s Impella pumps for localized cardiac AAV delivery [5] [US • 08 Jan 2026]

https://www.fiercebiotech.com/medtech/lexeo-therapeutics-jj-team-test-heart-pump-technology-cardiac-gene-therapy-delivery
Context: Preclinical approach to concentrate AAV in the heart and lower systemic exposure.
Key point: Goal is to reduce dosing and off-target exposure by pump-assisted, cardiac-localized delivery.
Implication: Signals pipeline investment and modality expansion.

🧫 CIRM funds UC San Diego’s CRISPR stem cell therapy program for FA, $7.4m [6] [US • 08 Jan 2026]

https://www.news-medical.net/news/20260108/CIRM-awards-2474-million-to-advance-stem-cell-based-gene-therapy-for-Friedreichs-ataxia.aspx
Context: Pre-clinical work to complete IND-enabling safety and manufacturing steps.
Key point: Funding supports autologous HSC collection, CRISPR correction, and readiness for first-in-human application.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🧒 REGENXBIO posts 18-month RGX-202 DMD functional gains; multiple 2026 catalysts [7] [11 Jan 2026]

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
Context: Phase I/II AFFINITY DUCHENNE pivotal dose cohort (n=4); BLA planned mid-2026.
Key point: NSAA improved 7.4 points vs cTAP at 18 months and 6.6 at 12 months; confirmatory trial enrollment ongoing.
Implication: May influence prescriber choice and payer reviews pending full data.

🧯 AskBio IND accepted for AB-1009 in late-onset Pompe; Fast Track and Orphan [8] [US • 08 Jan 2026]

https://www.askbio.com/fda-acceptance-of-investigational-new-drug-ind-application-for-ab-1009-gene-therapy/
Context: AAV gene therapy enters Phase 1/2; U.S. trial initiated with first patient recruitment anticipated early 2026.
Key point: Regulatory designations could enable expedited development and review pathways.
Implication: May influence prescriber choice and payer reviews pending full data.

🧠 Solid Biosciences doses first FA participant with dual-route SGT-212 (FALCON) [9] [US • 12 Jan 2026]

https://www.globenewswire.com/news-release/2026/01/12/3216871/0/en/Solid-Biosciences-Doses-First-Participant-in-First-in-Class-Phase-1b-FALCON-Trial-Evaluating-SGT-212-Dual-Route-Gene-Therapy-for-the-Treatment-of-Friedreich-s-Ataxia.html
Context: Phase 1b uses MRI-guided intradentate infusion plus IV to target neurologic and cardiac disease.
Key point: First-in-human dosing completed; preliminary safety updates planned and initial data expected H2 2026.
Implication: May influence prescriber choice and payer reviews pending full data.

🤖 Avista launches ARTEMIS AI capsid platform and innovation unit [10] [US • 09 Jan 2026]

https://www.prnewswire.com/news-releases/avista-therapeutics-expands-ai-gene-therapy-platform-to-break-barriers-in-the-delivery-of-genetic-medicines-302656976.html
Context: Uses NHP single-cell datasets, protein language models, and molecular dynamics for capsid engineering.
Key point: Aims to improve AAV targeting, enable larger gene delivery, and tackle manufacturing constraints.
Implication: Signals pipeline investment and modality expansion.

Why It Matters

FDA flexibility could shorten CGT development timelines while maintaining quality safeguards.
RMAT and Fast Track designations point to clearer regulatory paths for serious indications.
Delivery innovation, from pumps to AI-designed capsids, targets dose reduction and safety.
FA and DMD see simultaneous advances across funding, early clinical dosing, and new data.
Partnerships and platform builds suggest sustained investment despite recent sector volatility.

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FAQ

What did FDA change regarding CGT manufacturing expectations?

FDA signaled flexibility beyond Phase I on certain Chapter 600 specs and minor manufacturing changes with supporting evidence [1]. The agency maintains a focus on safety, purity, and potency.

What is uniQure seeking for AMT-130 in Huntington’s?

uniQure scheduled a Type A FDA meeting to discuss its BLA data package and a potential accelerated approval pathway for AMT-130, with a formal update expected after meeting minutes [2].

Why is Eureka’s RMAT for ECT204 notable?

RMAT, granted after Phase I cohort and additional ARYA-3 data, provides enhanced FDA interaction and potential use of surrogate endpoints for advanced HCC, a tough solid tumor setting [4].

How strong are REGENXBIO’s new DMD data?

In the AFFINITY DUCHENNE Phase I/II pivotal-dose cohort (n=4), NSAA improved 7.4 points vs cTAP at 18 months and 6.6 at 12 months. A BLA filing is planned mid-2026, with confirmatory enrollment ongoing [7].

What is distinctive about Solid’s SGT-212 approach in FA?

Dual-route administration, MRI-guided intradentate nuclei plus IV, aims to address neurologic, cardiac, and systemic features. First participant has been dosed in Phase 1b FALCON [9].

What exactly is Avista’s ARTEMIS platform?

An AI capsid-engineering system combining NHP single-cell AAV datasets, protein language models, and molecular dynamics to design targeted, efficient AAV vectors across tissues [10].

Entities / Keywords

FDA; CBER; CGT CMC; RMAT; Accelerated approval; AAV; CAR-T; ARTEMIS (Eureka, Avista distinct); Impella heart pump; AMT-130 (uniQure); RGX-202 (REGENXBIO; DMD); AB-1009 (AskBio; LOPD); SGT-212 (Solid; FA); CIRM; UC San Diego; Link Cell Therapies; Nona Biosciences.