Cell and Gene Therapy Today—March 19, 2026

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🧬 Einstein team reports longer-lasting CAR-T manufacturing method [1] [US • 15 Mar 2026]

https://www.biospace.com/press-releases/a-new-method-produces-car-t-cells-to-keep-fighting-disease-longer
Context: Albert Einstein College of Medicine-led work, published in Science Advances, tested an IL-7/IL-15/IL-21 cytokine-fusion scaffold, HCW9206, during CAR-T manufacturing [1].
Key point: The method produced CAR-T cells enriched for T memory stem cells and improved recall activity in mouse leukemia models, with stronger suppression of HIV-infected cells in humanized mouse models [1].
Implication: May influence prescriber choice and payer reviews pending full data.

🩸 Sana’s UP421 shows 14-month insulin secretion in type 1 diabetes [2] [EU • 16 Mar 2026]

https://www.biospace.com/drug-development/sanas-cell-therapy-sustains-insulin-production-through-14-months-in-a-t1d-patient
Context: In an investigator-sponsored first-in-human study at Uppsala University Hospital, one patient with type 1 diabetes received UP421 without immunosuppression [2].
Key point: Sana Biotechnology said donor-derived hypoimmune islet cells survived 14 months, continued producing insulin, and were associated with tighter glycemic control between months 12 and 14 [2].
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

🧫 Grit’s GT307 TIL therapy clears FDA IND [3] [US • 16 Mar 2026]

https://www.accessnewswire.com/newsroom/en/biotechnology/ubrigene-congratulates-strategic-partner-grit-biotechnologies-on-fda-ind-clearance-fo-1147334
Context: Grit Biotechnologies’ GT307 is a next-generation, gene-edited tumor-infiltrating lymphocyte therapy; uBriGene Biosciences supported process development, GMP manufacturing, and regulatory work [3].
Key point: FDA cleared the IND for GT307, allowing first-in-human clinical development to proceed [3].
Implication: Signals pipeline investment and modality expansion.

💪 Sarepta opens ENDEAVOR Cohort 8 for ELEVIDYS with enhanced immunosuppression [4] [US • 16 Mar 2026]

https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-announces-screening-and-enrollment-are-underway-endeavor
Context: Cohort 8 of ENDEAVOR will enroll about 25 non-ambulant Duchenne muscular dystrophy participants in the US and adds peri-infusion sirolimus before and after ELEVIDYS [4].
Key point: Sarepta Therapeutics said the cohort is designed to assess whether the regimen can mitigate acute liver injury risk, with primary endpoints of ALI incidence and ELEVIDYS-dystrophin expression at 12 weeks [4].
Implication: May influence prescriber choice and payer reviews pending full data.

🧪 Ultragenyx’s DTX301 hits first co-primary Phase 3 endpoint in OTC deficiency [5] [US • 12 Mar 2026]

https://www.fiercebiotech.com/biotech/ultragenyx-gene-therapy-lessens-ammonia-levels-ph-3-rare-disease-win
Context: The Phase 3 trial enrolled 37 patients with ornithine transcarbamylase deficiency and compares one-time AAV gene therapy DTX301 against placebo, with a second co-primary endpoint due later [5].
Key point: At 36 weeks, DTX301 lowered 24-hour plasma ammonia levels by 18% versus placebo, meeting one co-primary endpoint; one treated patient had serious acute liver inflammation, and five placebo patients had hyperammonemic crises [5].
Implication: May influence prescriber choice and payer reviews pending full data.

🫀 Solid highlights Duchenne biomarker and cardiac signals for SGT-003 [6] [US • 13 Mar 2026]

https://www.biospace.com/drug-development/solid-adds-to-differentiated-profile-with-new-data-for-duchenne-gene-therapy
Context: Interim Phase 1/2 INSPIRE DUCHENNE data included 90-day biopsies from 20 treated patients; the study does not include an untreated control arm [6].
Key point: Solid Biosciences reported robust microdystrophin expression, restoration of dystrophin-associated protein complex markers, and early cardiac and muscle biomarker improvements for SGT-003 [6].
Implication: May influence prescriber choice and payer reviews pending full data.

⚠️ CSL reports temporary global stockout of HEMGENIX [7] [17 Mar 2026]

https://newsroom.csl.com/2026-03-17-Our-Commitment-to-the-Hemophilia-B-Community-An-Update-on-HEMGENIX-R-etranacogene-dezaparvovec-drlb-Availability
Context: CSL addressed the hemophilia B community directly regarding commercial supply of HEMGENIX (etranacogene dezaparvovec-drlb) [7].
Key point: The company said a temporary global stockout will delay treatment for some eligible individuals in countries with established access, and stated the issue is not related to safety or effectiveness [7].
Implication: Introduces competition that may affect pricing and formulary access.

🧠 Apertura and Viralgen team up on TfR1 CapX manufacturing for CNS gene therapies [8] [US/EU • 17 Mar 2026]

https://aperturagtx.com/news/apertura-gene-therapy-and-viralgen-announce-strategic-collaboration-to-manufacture-tfr1-capx-a-next-generation-capsid-for-gene-therapies-designed-to-treat-central-nervous-system-diseases/
Context: Apertura Gene Therapy’s TfR1 CapX is an AAV capsid designed to bind human transferrin receptor 1, cross the blood-brain barrier, and support CNS delivery; Viralgen will provide manufacturing support [8].
Key point: The companies announced a strategic collaboration to supply plasmid and manufacturing infrastructure intended to move TfR1 CapX-based programs from research toward clinical development [8].
Implication: Signals pipeline investment and modality expansion.

🧬 ENCell advances EN001 to Phase 2a in CMT1A [9] [South Korea • 18 Mar 2026]

https://www.koreabiomed.com/news/articleViewAmp.html?idxno=30966
Context: ENCell completed a Phase 1b repeat-dose study of EN001 in Charcot-Marie-Tooth disease type 1A and plans a randomized, double-blind, placebo-controlled Phase 2a trial [9].
Key point: The company reported favorable tolerability, no dose-limiting toxicities, mild adverse drug reactions, and preliminary efficacy signals on CMTNSv2, while noting the study was small and not powered to confirm efficacy [9].
Implication: May influence prescriber choice and payer reviews pending full data.

Why It Matters

Duchenne gene therapy remains highly active, with both Sarepta and Solid pushing differentiated safety and efficacy narratives in non-ambulant and broader DMD settings [4][6].
Manufacturing is a central theme this week, from CAR-T process design and TIL production to AAV supply partnerships and a commercial stockout for an approved gene therapy [1][3][7][8].
Early clinical signals continue to shape investor and partner interest in cell therapy for diabetes and rare neuromuscular disease, but several readouts remain small, uncontrolled, or preliminary [2][9].
Platform value is increasingly tied to delivery and persistence, seen in long-lived CAR-T cells, hypoimmune islet grafts, and CNS-directed AAV capsids [1][2][8].
Regulatory progress is steady but uneven, with an IND clearance, a Phase 3 co-primary win, and safety-focused protocol adjustments all pointing to a field still balancing efficacy with manufacturability and risk management [3][4][5].

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FAQ

What changed for ELEVIDYS in Sarepta’s ENDEAVOR Cohort 8?

Sarepta added peri-infusion sirolimus for about 25 non-ambulant Duchenne participants to test whether enhanced immunosuppression can reduce acute liver injury risk while preserving dystrophin expression at 12 weeks [4].

How strong is Sana’s UP421 evidence in type 1 diabetes?

The update is based on one first-in-human patient treated at Uppsala University Hospital. Sana reported 14 months of graft survival and insulin production without immunosuppression, but the dataset is still very limited [2].

Did Ultragenyx’s DTX301 already finish Phase 3 in OTC deficiency?

Not yet. DTX301 met one co-primary endpoint, reduction in 24-hour plasma ammonia at 36 weeks, but the second co-primary endpoint on disease-management response is still pending and expected later [5].

What is notable about Solid Biosciences’ SGT-003 update?

Solid reported biomarker restoration and early cardiac-related signals in INSPIRE DUCHENNE, which helps support mechanism and differentiation. The study is uncontrolled, so interpretation should remain cautious [6].

What does CSL’s HEMGENIX stockout mean for patients and providers?

CSL said some treatments will be delayed in countries with established commercial access. The company stated the issue reflects manufacturing complexity, not a new safety or efficacy concern [7].

Why does the Apertura–Viralgen deal matter?

It is a manufacturing-readiness move around TfR1 CapX, a CNS-targeting AAV capsid. The collaboration is intended to help Apertura licensees access plasmid supply and scalable production as programs approach the clinic [8].

Entities / Keywords

Albert Einstein College of Medicine, Einstein, Harris Goldstein, HCW9206, CAR-T, T memory stem cells, HIV, leukemia [1]
Sana Biotechnology, UP421, SC451, hypoimmune islet cells, type 1 diabetes, C-peptide, Uppsala University Hospital [2]
Grit Biotechnologies, uBriGene Biosciences, GT307, TIL therapy, gene-edited TIL, CRISPR/AaCas12bMAX, IND clearance [3]
Sarepta Therapeutics, ELEVIDYS, delandistrogene moxeparvovec-rokl, ENDEAVOR, Cohort 8, Duchenne muscular dystrophy, sirolimus [4]
Ultragenyx, DTX301, ornithine transcarbamylase deficiency, OTC deficiency, AAV gene therapy, ammonia [5]
Solid Biosciences, SGT-003, INSPIRE DUCHENNE, microdystrophin, dystrophin-associated protein complex, DMD [6]
CSL, HEMGENIX, etranacogene dezaparvovec-drlb, hemophilia B, gene therapy supply [7]
Apertura Gene Therapy, Viralgen, TfR1 CapX, AAV capsid, blood-brain barrier, CNS gene therapy, hTfR1 [8]
ENCell, EN001, Charcot-Marie-Tooth disease type 1A, CMT1A, CMTNSv2, stem cell therapy [9]