Cell and Gene Therapy Weekly News - October 2nd 2025

Gene & Cell Therapy Update: eXmoor × Siam Bioscience Partnership, FDA Fast Tracks and Draft Guidelines and More

From global partnerships and technological breakthroughs, this week’s digest highlights the innovations redefining gene and cell therapy. Whether your focus is rare disease, CNS, or manufacturing efficiency, these updates bring you the latest advances and market drivers.

In Today’s Newsletter

🏭 eXmoor & Siam Bioscience to build CGT hub in SE Asia [1] [TH • 25 Sep 2025]

https://www.nationthailand.com/pr-news/pr-news/40055903
Context: Aims to support local CGT programs and introduce international CGTs/tech into ASEAN.
Key point: Strategic partnership to offer consultancy, process/analytical dev, and GMP clinical/commercial manufacturing near Bangkok.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

🧬 YouthBio gets INTERACT nod to prep first-in-human for YB002 (Alzheimer’s) [2] [US • 25 Sep 2025]

https://www.prnewswire.com/news-releases/youthbio-therapeutics-announces-positive-fda-interact-feedback-for-yb002-establishing-clear-path-to-clinic-for-first-in-class-alzheimers-gene-therapy-302563494.html
Context: Transient Yamanaka factor expression in brain; next steps include CMC and pilot tox toward Pre-IND.
Key point: FDA agreed existing preclinical data support bioactivity and proposed FIH plan for partial reprogramming gene therapy.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

🏛️ FDA drafts outline streamlined CGT development & approvals [3] [US • 25 Sep 2025]

https://www.biospace.com/fda/fda-details-thinking-for-streamlining-development-of-cell-gene-regenerative-therapies
Context: Three CBER draft guidances; emphasizes quality controls early to ensure strength/identity/purity.
Key point: Recommends innovative designs (single-arm baseline-controlled, historical/RWD controls, adaptive), expedited paths, and post-approval RWE.
Implication: Observational/RWE: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🧰 IDT launches Alt-R HDR Enhancer Protein for CRISPR [4] [US • 25 Sep 2025]

https://www.businesswire.com/news/home/20250925291776/en/Integrated-DNA-Technologies-Launches-New-Enhancer-Protein-to-Accelerate-CRISPR-Based-Therapies-from-Translational-Research-to-Clinical-Breakthrough
Context: Manufactured by Aldevron; compatible with Cas9/Cas12a and standard delivery methods.
Key point: Company reports up to ~2× HDR gains in iPSCs/HSPCs with maintained specificity/viability (per internal data); RUO now, cGMP to follow.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

💪 Capricor’s Deramiocel (DMD) BLA path clarified after Type A meeting [5] [US • 25 Sep 2025]

https://www.globenewswire.com/news-release/2025/09/25/3156168/0/en/Capricor-Therapeutics-Provides-Regulatory-Update-on-Deramiocel-Program-for-Duchenne-Muscular-Dystrophy-Following-Type-A-Meeting.html
Context: CRL issued July 2025; HOPE-3 completed; topline planned mid-Q4 2025.
Key point: FDA aligned that HOPE-3 can serve as the “additional study” in current BLA; PUL v2.0 primary, LVEF key secondary.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

🧫 Verismo × Miltenyi: LVV supply secured for STAR-101 (SynKIR-110) [6] [US • 26 Sep 2025]

https://www.biospace.com/press-releases/verismo-therapeutics-announces-partnership-with-miltenyi-biotec-to-support-advancing-clinical-programs
Context: Collaboration aimed at Phase 2/commercial readiness and supply continuity for KIR-CAR platform.
Key point: First clinical cell product manufactured using Miltenyi Bioindustry lentiviral vector for Phase 1 mesothelin program.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

⚖️ Factor Bioscience sues Cellectis & AstraZeneca over mRNA TALENs IP [7] [US • 26 Sep 2025]

https://themalaysianreserve.com/2025/09/26/factor-bioscience-files-complaint-against-cellectis-and-astrazeneca-alleging-infringement-of-foundational-gene-editing-patents/amp/
Context: Factor cites impact on allogeneic cancer cell therapy programs and AZ partnership activities.
Key point: Complaint alleges infringement of US Patents 10,662,410; 10,829,738; 10,982,229 tied to mRNA TALENs.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

🧱 Aera nominates AERA-109: targeted in vivo CAR-T for autoimmune disease [8] [US • 25 Sep 2025]

https://www.biospace.com/press-releases/aera-therapeutics-presents-preclinical-data-and-announces-nomination-of-first-development-candidate-aera-109-a-targeted-in-vivo-car-t-therapy-for-b-cell-mediated-autoimmune-diseases
Context: tLNP delivery; non-viral in vivo CAR-T generation with design avoiding lymphodepletion and viral vectors.
Key point: Preclinical data in humanized mice/NHPs show deep B-cell depletion; plans clinical entry mid-2026 (endpoint not specified).
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

👁️ Opus Genetics posts pediatric LCA5 data; FDA talks next [9] [US • 30 Sep 2025]

https://www.fiercebiotech.com/biotech/opus-sets-sights-fda-talks-after-posting-pediatric-gene-therapy-data
Context: AAV8 augmentation using Luxturna-like promoter; adult data previously suggested durability to 18 months.
Key point: Early pediatric cohort showed average ~0.3 logMAR improvement; meeting with FDA planned in Q4 (per report).
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

🚀 Umoja’s in vivo CAR-T (UB-VV111) gets FDA Fast Track [10] [US • 30 Sep 2025]

https://www.manilatimes.net/2025/09/30/tmt-newswire/globenewswire/umoja-biopharma-announces-that-ub-vv111-receives-fda-fast-track-designation-for-relapsedrefractory-b-cell-malignancies/2192356/amp
Context: Off-the-shelf in vivo CD19 CAR-T aims to bypass ex vivo manufacturing constraints.
Key point: Fast Track for R/R LBCL and CLL after ≥2 prior lines; Phase 1 ongoing; AbbVie holds option rights.
Implication: Clinical topline/efficacy: May influence prescriber choice and payer reviews pending full data.

🧪 CRISPR origin: “TranC” intermediates & RNA-splitting mechanism (Cell) [11] [CN • 29 Sep 2025]

https://phys.org/news/2025-09-scientists-reveal-functional-rna-mechanism.html
Context: CAS/Tsinghua teams used phylogeny, cryo-EM, and engineering to show RNA-level innovation enables CRISPR-like activity.
Key point: Work identifies TnpB→Cas12 evolutionary intermediates (TranCs) where reRNA split into tracrRNA+crRNA.
Implication: Observational/RWE: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🧯 Biogen exits AAV gene therapy; small staff shuffle [12] [US • 29 Sep 2025]

https://www.biospace.com/business/biogen-shuffles-20-staff-after-ending-aav-work
Context: Portfolio refocus toward “pioneer modalities” after multi-year restructuring and share underperformance.
Key point: Company discontinued AAV efforts; ~20 roles affected, many reassigned; part of broader cost cuts.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.

Why it matters

Regulatory tailwinds (INTERACT, Fast Track, FDA drafts) continue to clear pathways for first-in-class CGTs.
Manufacturing/supply chain moves (Bangkok hub; Miltenyi vectors) aim to regionalize access and de-risk scale-up.
In vivo CAR-T momentum (Umoja, Aera) could compress cost and logistics vs ex vivo approaches.

📢 Stay Ahead in Cell and Gene Therapy!

✅ Contact LucidQuest at 📧 info@lqventures.com for strategic guidance on gene therapy innovations and clinical researchbreakthroughs.

FAQ

What did FDA agree to in Capricor’s Type A meeting? [5]

Answer: That HOPE-3 can serve as the “additional study” within the current DMD BLA; PUL v2.0 remains the primary endpoint with LVEF as a key secondary. Topline HOPE-3 data are planned for mid-Q4 2025 per the company.

What does Fast Track for UB-VV111 practically provide? [10]

Answer: More frequent FDA interactions and potential rolling review for Umoja’s in vivo CD19 CAR-T in R/R LBCL and CLL. A Phase 1 trial is ongoing (endpoint specifics not detailed here).

What exactly is YouthBio’s YB002 approach? [2]

Answer: A brain-targeted, partial cellular reprogramming gene therapy with transient expression of Yamanaka factors. INTERACT feedback supports the preclinical bioactivity package and proposed FIH plan.

How might IDT’s HDR Enhancer affect editing workflows? [4]

Answer: Company reports up to ~2× HDR in difficult cells (iPSCs/HSPCs) without increased off-targets/translocations (per internal data), potentially improving knock-in efficiency. RUO now; cGMP planned.

What’s new about the CRISPR origin work? [11]

Answer: Identification of TranC clades bridging TnpB to Cas12, showing that splitting a transposon reRNA into tracrRNA+crRNA underlies type V emergence and can be engineered to confer CRISPR-like function.

Entities / Keywords

eXmoor Pharma; Siam Bioscience (CGT GMP hub; ASEAN); YouthBio (YB002; partial reprogramming; Alzheimer’s); FDA CGT draft guidances (CBER; RWD; innovative designs); Integrated DNA Technologies / Aldevron (Alt-R HDR Enhancer Protein; HDR; iPSC/HSPC); Capricor (Deramiocel; DMD; HOPE-3; PUL v2.0; LVEF; CRL); Verismo (SynKIR-110; STAR-101; mesothelin; Miltenyi Bioindustry; LVV); Factor Bioscience (mRNA TALENs; patents 10,662,410 / 10,829,738 / 10,982,229; Cellectis; AstraZeneca); Aera Therapeutics (AERA-109; tLNP; in vivo CAR-T; autoimmune); Opus Genetics (LCA5; AAV8; pediatric logMAR); Umoja (UB-VV111; Fast Track; CD19; in vivo CAR-T); CAS/Tsinghua (TranC; TnpB→Cas12; RNA splitting); Biogen (AAV exit; restructuring).

References