Gene & Cell Therapy Update: JCR CNS/Muscle AAV, Lexeo FA Data, RareTx Launch, Kernal mRNA CAR-T and more
From JCR’s CNS/muscle-targeted AAV data, Rare Therapeutics’ ultra-orphan gene therapy launch, and Lexeo’s FA cardiomyopathy interim results—this episode’s signals are robust. We span gene therapy, cell therapy, regulatory pathways, and global access, with funding updates and clinical advances across areas, underscoring pipeline expansion and modality innovation.
In Today’s Newsletter
🧬 JCR’s JUST-AAV shows CNS/muscle targeting with liver de-targeting [1] [07 Oct 2025]
Key point: Non-clinical data at ESGCT suggest higher muscle and brain transduction versus AAV9, with markedly lower liver tropism (mice, monkeys).
Context: Platform uses peptide binders and J-Brain Cargo (reported 28–52× muscle/heart transduction and ~160× liver reduction vs AAV9 in mice, study conditions).
Implication: Signals pipeline investment and modality expansion.
🧡 Rare Therapeutics launches for ultra-orphan gene therapies (GEMMABio) [2] [07 Oct 2025]
Key point: New clinical-stage affiliate, RareTx, targets CNS and heart/skeletal-muscle monogenic diseases, including GM1, Krabbe and MLD.
Context: Strategy includes novel financing and public-private hubs in Brazil and Abu Dhabi for global access.
Implication: Signals pipeline investment and modality expansion.
❤️ Lexeo’s LX2006 AA path talks advance, interim data positive in FA cardiomyopathy [3] [07 Oct 2025]
Key point: FDA open to pooled Phase I/II plus pivotal data for accelerated approval; earlier LVMI timepoint may be acceptable.
Context: Interim shows mean LVMI reductions of 18% at 6 months and 23% at 12 months in abnormal baseline subgroup; mFARS improvements reported; safety generally favorable.
Implication: May influence prescriber choice and payer reviews pending full data.
💉 Canada moves toward public coverage for Hemgenix (CSL Behring) [4] [06 Oct 2025]
https://finance.yahoo.com/news/csl-behring-signs-letter-intent-120000800.html
Key point: LOI with pCPA enables provinces and territories outside Quebec to list Hemgenix for hemophilia B prophylaxis.
Context: CDA earlier recommended reimbursement based on HOPE-B; most treated patients discontinued routine prophylaxis per cited results.
Implication: Introduces competition that may affect pricing and formulary access.
🧪 Takeda exits cell therapy amid strategic pivot [5] [02 Oct 2025]
Key point: Takeda to divest cell therapy platform and preclinical assets, with 137 layoffs; focus shifts to small molecules, biologics and ADCs.
Context: Prior deals included GammaDelta, Adaptate and Immusoft; impairment charges noted by source.
Implication: Signals pipeline investment and modality expansion.
🧫 ARPA-H backs Kernal’s in vivo mRNA CAR-T with up to $48M [6] [07 Oct 2025]
https://www.fiercebiotech.com/biotech/us-government-bankrolls-48m-kernals-mrna-car-t
Key point: Funding under EMBODY program supports KR-402 and platform expansion for hematologic cancers and autoimmune diseases.
Context: Approach combines selective mRNA and targeted LNPs; collaborators include Stanford, Dana-Farber and Jackson Laboratory.
Implication: Signals pipeline investment and modality expansion.
🌎 CTMC–Einstein Hospital Israelita launch Global Cell Therapy Network Alliance [7] [02 Oct 2025]
Key point: Alliance aims to expand access to cell therapies across LATAM via training, SOPs and proprietary reagents.
Context: Einstein becomes first international member; model emphasizes patient-adjacent manufacturing.
Implication: May expand screening, initiation, and follow-up at scale.
🚀 Rocket withdraws BLA for Fanconi anemia gene therapy RP-L102 [8] [03 Oct 2025]
https://www.fiercebiotech.com/biotech/rocket-aborts-mission-get-gene-therapy-approved-fanconi-anemia
Key point: Voluntary BLA withdrawal due to portfolio reprioritization; may seek partnerships to advance later.
Context: Follows EU withdrawal and restructuring to focus on cardiovascular programs.
Implication: Signals pipeline investment and modality expansion.
🧠 Kernal Bio joins Lilly Gateway Labs in Boston [9] [03 Oct 2025]
Key point: Relocation to LGL for access to Lilly expertise and shared infrastructure while retaining independence.
Context: Company highlights kernaLM for mRNA design and NVIDIA Inception participation.
Implication: Signals pipeline investment and modality expansion.
👧 Taysha’s TSHA-102 gets FDA Breakthrough Therapy for Rett syndrome [10] [02 Oct 2025]
https://finance.yahoo.com/news/taysha-gene-therapies-announces-fda-120000932.html
Key point: BTD based on Part A data from 12 patients and alignment on pivotal REVEAL protocol including a 6-month interim analysis.
Context: Single-arm, open-label pivotal will assess milestone gain/regain after high-dose IT AAV9 in 15 females aged 6–<22.
Implication: May influence prescriber choice and payer reviews pending full data.
🧯 Avobis Bio’s AVB-114 receives RMAT for Crohn’s perianal fistulas [11] [03 Oct 2025]
Key point: RMAT supports intensive FDA guidance toward Phase III planning for AVB-114.
Context: Phase I STOMP-I reported 76% drainage cessation at 12 months; Phase II primary analysis completed with data presentation planned.
Implication: May influence prescriber choice and payer reviews pending full data.
⚡ Neurona expands NRTX-1001 into MTLE without MTS, first subject dosed [12] [07 Oct 2025]
Key point: New non-MTS cohort added to Phase 1/2; no significant surgical complications reported to date.
Context: Program spans unilateral and bilateral MTLE, with RMAT interactions and planned Phase 3 EPIC study.
Implication: May influence prescriber choice and payer reviews pending full data.
Why it matters
- Accelerated pathways and designations continue to shape timelines for cell and gene therapies.
- Access strategies, from Canada’s Hemgenix LOI to LATAM alliances, are evolving alongside science.
- Portfolio reshaping persists, with exits and funding reallocations determining which modalities advance.
- Platform advances, including capsid engineering and in vivo mRNA CAR-T, aim to improve targeting, safety and scalability.
📢 Stay Ahead in Cell & Gene research!
✅ Contact LucidQuest at info@lqventures.com for expert guidance on innovations shaping the future of cell and gene treatments.
FAQ
What is Lexeo’s regulatory plan for LX2006 in FA cardiomyopathy?
Lexeo reports FDA openness to pooling ongoing Phase I/II data with pivotal results for accelerated approval, with potential earlier LVMI assessment, subject to comparability and nonclinical steps. [3]
Which diseases will Rare Therapeutics initially target?
RareTx, launched by GEMMABio, is focused on ultra-orphan CNS lysosomal storage disorders including GM1 gangliosidosis, Krabbe disease and metachromatic leukodystrophy. [2]
What does Canada’s LOI for Hemgenix change for patients?
The LOI with pCPA allows provinces and territories outside Quebec to proceed with public formulary listing, advancing access to the one-time AAV5 therapy for hemophilia B prophylaxis. [4]
Why did Rocket withdraw the Fanconi anemia BLA?
Rocket cites strategic reprioritization and resource focus on programs with clearer regulatory and commercial pathways; it may pursue partnerships to potentially re-engage later. [8]
What is Kernal Bio building with ARPA-H support?
Under EMBODY, Kernal will progress in vivo mRNA-encoded CAR-T (KR-402) and expand its mRNA 2.0 platform for hematologic and autoimmune indications, with academic collaborators. [6]
How is Neurona broadening NRTX-1001’s MTLE reach?
Neurona added a non-MTS unilateral MTLE cohort to its Phase 1/2 program and plans a Phase 3 EPIC study to support a future BLA in drug-resistant MTLE. [12]
Entities / Keywords
JCR Pharmaceuticals; JUST-AAV; J-Brain Cargo; GEMMABio; Rare Therapeutics (RareTx); GM1; Krabbe; MLD; Lexeo Therapeutics; LX2006; Friedreich ataxia cardiomyopathy; LVMI; mFARS; CSL Behring; Hemgenix; pCPA; Takeda; ADCs; Kernal Bio; ARPA-H; EMBODY; KR-402; Lilly Gateway Labs; CTMC; Einstein Hospital Israelita; CAR-T; CAR-NK; Rocket Pharmaceuticals; RP-L102 (Fanca-cel); Fanconi anemia; Taysha; TSHA-102; Rett syndrome; Avobis Bio; AVB-114; Crohn’s perianal fistulas; Neurona Therapeutics; NRTX-1001; MTLE; MTS.
References
- https://www.businesswire.com/news/home/20251007929013/en/JCR-Pharmaceuticals-Enhances-Delivery-of-AAV-Gene-Therapy-to-Central-Nervous-System-and-Muscle-with-Novel-Capsid-Engineering-Platform-Technology
- https://www.prnewswire.com/news-releases/gemmabio-launches-rare-therapeutics-inc-to-develop-and-deliver-gene-therapies-for-ultraorphan-diseases-302576274.html
- https://www.globenewswire.com/news-release/2025/10/07/3162307/0/en/Lexeo-Therapeutics-Announces-Progress-in-FDA-Discussions-for-Accelerated-Approval-Pathway-and-Positive-Interim-Clinical-Data-for-LX2006-in-Friedreich-Ataxia-Cardiomyopathy.html
- https://finance.yahoo.com/news/csl-behring-signs-letter-intent-120000800.html
- https://www.biospace.com/business/takeda-exits-cell-therapy-amid-strategic-pivot-dealing-blow-to-once-promising-modality
- https://www.fiercebiotech.com/biotech/us-government-bankrolls-48m-kernals-mrna-car-t
- https://www.businesswire.com/news/home/20251002418318/en/CTMC-and-Einstein-Hospital-Israelita-in-Brazil-Partner-to-Launch-the-Global-Cell-Therapy-Network-Alliance
- https://www.fiercebiotech.com/biotech/rocket-aborts-mission-get-gene-therapy-approved-fanconi-anemia
- https://www.businesswire.com/news/home/20251003567884/en/Kernal-Bio-Joins-Lilly-Gateway-Labs-to-Accelerate-In-Vivo-Cell-Therapies
- https://finance.yahoo.com/news/taysha-gene-therapies-announces-fda-120000932.html
- https://www.prnewswire.com/news-releases/fda-grants-rmat-designation-to-avobis-bios-implantable-cell-therapy-avb-114-for-crohns-perianal-fistulas-302574412.html
- https://www.globenewswire.com/de/news-release/2025/10/07/3162937/0/en/Neurona-Expands-Investigation-of-NRTX-1001-Cell-Therapy-into-Mesial-Temporal-Lobe-Epilepsy-MTLE-without-Mesial-Temporal-Sclerosis-MTS.html