Cell and Gene Therapy Today—March 5, 2026

In Today’s Newsletter

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🧬 Orsini expands specialty pharmacy distribution for LYFGENIA, ZYNTEGLO, SKYSONA [1] [US • 26 Feb 2026]

https://www.biospace.com/press-releases/orsini-now-distributing-gene-therapies-lyfgenia-zynteglo-skysona
Context: Orsini’s Cell and Gene Therapy Center of Excellence now supports 12 therapies (per release).
Key point: Orsini was selected as a specialty pharmacy partner supporting three FDA-approved autologous HSC-based gene therapies, LYFGENIA, ZYNTEGLO, SKYSONA.
Implication: May expand screening, initiation, and follow-up at scale.

🤝 Pfizer takes global rights option on Beam base-editing liver candidate [2] [25 Feb 2026]

https://www.fiercebiotech.com/biotech/after-gene-therapy-exit-pfizer-locks-global-license-beam-gene-editing-candidate
Context: Follows a multi-year Pfizer–Beam collaboration focused on in vivo base editing (asset unnamed in the article).
Key point: Pfizer exercised an option for global rights, taking over development, manufacturing, and potential commercialization for an unnamed liver disease candidate using Beam delivery technology.
Implication: Signals pipeline investment and modality expansion.

👶 GEMMABio doses first patient in SMA1 next-generation gene therapy trial [3] [US • 26 Feb 2026]

https://www.prnewswire.com/news-releases/gemmabio-announces-first-patient-dosed-in-charisma-trial-of-gb221-investigational-nextgeneration-gene-therapy-for-spinal-muscular-atrophy-type-1-sma1-302698146.html
Context: Phase 1/2 CHARISMA (NCT07070999) evaluates safety, tolerability, efficacy in infants, includes symptomatic and presymptomatic cohorts (n not stated).
Key point: First patient dosed with GB221 for SMA type 1, and FDA granted GB221 rare pediatric disease designation.
Implication: May influence prescriber choice and payer reviews pending full data.

🧾 FDA pushes uniQure toward randomized sham-controlled path for AMT-130 [4] [US • 02 Mar 2026]

https://www.globenewswire.com/news-release/2026/03/02/3247236/0/en/uniQure-Provides-Regulatory-Update-on-AMT-130-for-Huntington-s-Disease.html
Context: FDA feedback reflects a Type A meeting (30 Jan 2026) on AMT-130 for Huntington’s disease.
Key point: FDA said Phase I/II data versus external control are not sufficient as primary evidence of effectiveness, and strongly recommended a prospective randomized, double-blind, sham-controlled study. Implication: May influence prescriber choice and payer reviews pending full data.

👁️ Ocugen completes Phase 3 enrollment for OCU400 in broad retinitis pigmentosa [5] [US • 02 Mar 2026]

https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-phase-3-limelight-enrollment-completion-ocu400
Context: liMeliGhT enrolled 140 patients (randomized 2:1), one-year trial, primary endpoint is LDNA at 12 months (per release).
Key point: Enrollment completed, topline Phase 3 data expected in 1Q 2027, company also cited EMA acceptability of the US-based trial for an MAA submission.
Implication: May influence prescriber choice and payer reviews pending full data.

🧪 FDA lifts Intellia MAGNITUDE Phase 3 clinical hold in ATTR-CM [6] [US • 02 Mar 2026]

https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-fda-lift-clinical-hold-0
Context: Holds were imposed 29 Oct 2025 after a Grade 4 liver lab event meeting protocol pausing criteria (per release).
Key point: FDA removed the clinical hold on MAGNITUDE (nexiguran ziclumeran, nex-z) with aligned mitigation measures, including enhanced liver monitoring and added exclusion criteria.
Implication: May influence prescriber choice and payer reviews pending full data.

🔬 Sanaregen SVT-001 cleared for Phase 1/2 in Familial Drusen [7] [US • 03 Mar 2026]

https://sg.finance.yahoo.com/news/sanaregen-vision-therapeutics-receives-fda-110000202.html
Context: Phase 1/2 will assess safety and effectiveness in Familial Drusen (no approved disease-modifying treatments noted).
Key point: FDA cleared Sanaregen Vision Therapeutics to initiate a Phase 1/2 clinical trial of SVT-001 (investigational cell therapy) for retinal degeneration in Familial Drusen.
Implication: May influence prescriber choice and payer reviews pending full data.

🧫 T-knife wins EU CTA to start Phase 1 for CRISPR-edited PRAME T cell therapy [8] [EU • 03 Mar 2026]

https://www.t-knife.com/news/press-releases/03-03-26/
Context: ATLAS is described as adaptive, first-in-human, open-label Phase 1 in advanced PRAME-positive solid tumors (timelines and n not stated).
Key point: T-knife announced EU authorization of its clinical trial application to initiate Phase 1 ATLAS for TK-6302, a multi-armored, CRISPR-based PRAME-targeted T cell therapy.
Implication: Signals pipeline investment and modality expansion.

🌏 ISCT and JSRM launch a 5-year clinical iPSC collaboration, inaugural event in Kobe [9] [Japan • 03 Mar 2026]

https://www.prnewswire.com/news-releases/isctjsrm-5-year-joint-strategic-collaboration-on-clinical-ipscs-launches-inaugural-joint-collaboration-in-kobe-japan—march-21-2026-302702761.html
Context: Collaboration focuses on clinical iPSCs, infrastructure, and harmonization across regulation, manufacturing, and trials (deliverables not specified).
Key point: ISCT and JSRM announced a 5-year strategic partnership on clinical iPSCs, with an inaugural joint collaboration event tied to the JSRM Annual Meeting in Kobe (21 Mar 2026).
Implication: Signals pipeline investment and modality expansion.

Why It Matters

Distribution and care-network buildout (Orsini) can be a practical bottleneck for approved CGTs reaching patients at scale. [1]
Big Pharma option exercises (Pfizer–Beam) continue to shape financing and ownership for in vivo editing programs. [2]
FDA feedback on evidentiary standards (uniQure, Intellia) highlights how trial design and safety monitoring can redefine timelines. [4], [6]
Ophthalmology remains active across modalities, with gene therapy (Ocugen) and cell therapy (Sanaregen) moving through clinical gates. [5], [7]
International coordination efforts (ISCT–JSRM) aim to standardize pathways for iPSC translation, which could reduce friction across regions. [9]

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FAQ

What gene therapies is Orsini supporting, and for what indications?

Orsini is distributing LYFGENIA (sickle cell disease), ZYNTEGLO (transfusion-dependent β-thalassemia), and SKYSONA (cerebral adrenoleukodystrophy in boys, specific criteria per label). [1]

What did Pfizer license from Beam Therapeutics?

Pfizer exercised an option for global rights to an unnamed liver disease base-editing candidate that uses Beam’s liver-targeting lipid nanoparticle delivery for base-editing reagents (asset name not provided). [2]

What is GB221, and what changed this week for GEMMABio’s SMA1 program?

GB221 is an investigational next-generation gene therapy for SMA type 1, and GEMMABio reported first patient dosed in Phase 1/2 CHARISMA plus FDA rare pediatric disease designation. [3]

Why did FDA push back on uniQure’s AMT-130 submission approach?

FDA said Phase I/II data compared to an external control were not sufficient as primary evidence of effectiveness, and recommended a prospective randomized, double-blind, sham surgery-controlled study. [4]

What’s the current status of Intellia’s MAGNITUDE trial in ATTR-CM?

FDA lifted the clinical hold, and Intellia described added risk-mitigation steps including enhanced liver monitoring and updated exclusion criteria. [6]

What is the ISCT–JSRM collaboration trying to achieve?

ISCT and JSRM announced a 5-year partnership to advance clinical iPSC therapies globally, including workstreams spanning trial design, manufacturing, safety standards, and regulatory harmonization (specific outputs not detailed). [9]

Entities / Keywords

Orsini (Orsini Healthcare), Cell and Gene Therapy Center of Excellence, specialty pharmacy distribution
LYFGENIA (lovotibeglogene autotemcel), ZYNTEGLO (betibeglogene autotemcel), SKYSONA (elivaldogene autotemcel), ex vivo gene addition, autologous HSC
Pfizer, Beam Therapeutics, in vivo base editing, lipid nanoparticle (LNP), liver disease (asset unnamed)
GEMMABio (Gemma Biotherapeutics), GB221, CHARISMA (NCT07070999), SMA type 1 (SMA1), rare pediatric disease designation
uniQure, AMT-130, Huntington’s disease, Type A meeting, sham-controlled randomized trial
Ocugen, OCU400, liMeliGhT Phase 3, retinitis pigmentosa (RP), LDNA
Intellia Therapeutics, nexiguran ziclumeran (nex-z), MAGNITUDE, ATTR-CM, clinical hold lift, liver monitoring
Sanaregen Vision Therapeutics, SVT-001, Familial Drusen, retinal degeneration, cell therapy
T-knife Therapeutics, TK-6302, PRAME, CRISPR-based T cell therapy, EU CTA, ATLAS Phase 1
ISCT, JSRM, clinical iPSCs, Kobe, regenerative medicine collaboration