Cell and Gene Therapy Today—January 29, 2026

In Today’s Newsletter

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🧬 FDA Grants Breakthrough Therapy Designation to Wugen’s WU-CART-007 [US • 21 Jan 2026]

https://www.biospace.com/press-releases/u-s-fda-grants-to-wugens-wu-cart-007-breakthrough-therapy-designation-for-treatment-of-relapsed-or-refractory-t-cell-acute-lymphoblastic-leukemia-t-cell-lymphoblastic-lymphoma
Context: Wugen’s investigational CAR-T therapy receives FDA Breakthrough Therapy Designation for T-ALL/T-LBL.
Key Point: WU-CART-007 is currently in clinical trials and shows promise in treating relapsed or refractory T-cell malignancies.
Implication: The designation aims to expedite development and potential approval.

🌱 ImmunityBio Updates Phase 2 Glioblastoma Data [US • 23 Jan 2026]

https://www.businesswire.com/news/home/20260123454593/en/ImmunityBio-Reports-Median-Overall-Survival-Not-Yet-Reached-and-Lymphopenia-Reversed-in-Recurrent-Glioblastoma-Patients-Receiving-ANKTIVA-Plus-CAR-NK-Chemo-Free-Therapy
Context: ImmunityBio reports ongoing Phase 2 results for ANKTIVA® plus CAR-NK in recurrent glioblastoma patients.
Key Point: The combination therapy demonstrated increased immune competence and survival in a challenging patient population.
Implication: These results suggest a new potential treatment for glioblastoma.

🧬 Beam Therapeutics Focuses on In Vivo Delivery for Sickle Cell Therapy [US • 21 Jan 2026]

https://www.fiercebiotech.com/biotech/touching-base-beam-ceo-gene-editing-biotech-big-push-vivo-delivery
Context: Beam shifts its focus to in vivo delivery for its next-gen sickle cell disease therapies.
Key Point: The company aims to optimize gene editing technology for faster, more efficient in vivo delivery to hematopoietic stem cells.
Implication: This shift could significantly improve treatment accessibility and reduce patient burdens.

💉 Sarepta Reports Three-Year Data for Elevidys in Duchenne Muscular Dystrophy [US • 26 Jan 2026]

https://www.biospace.com/drug-development/sareptas-dmd-gene-therapy-staves-off-disease-three-years-after-treatment
Context: Sarepta announces three-year follow-up results for Elevidys, its Duchenne muscular dystrophy gene therapy.
Key Point: The therapy showed significant motor function improvements and slowed disease progression in patients.
Implication: This data is a positive step for long-term gene therapy in rare diseases like DMD.

🧬 Intellia Resumes CRISPR Trial After FDA Hold [US • 27 Jan 2026]

https://www.fiercebiotech.com/biotech/after-patient-death-fda-lifts-hold-one-2-late-stage-trials-testing-intellias-crispr-therapy
Context: Intellia’s CRISPR therapy is cleared to resume after the FDA lifted a clinical hold following a patient death.
Key Point: The FDA had placed a hold on the Magnitude and Magnitude-2 studies due to safety concerns.
Implication: Resumption of the study could lead to a breakthrough treatment for transthyretin amyloidosis.

👁️ Opus Genetics Launches Gene Therapy Trial for MERTK-Related Retinitis Pigmentosa [US • 27 Jan 2026]

https://www.globenewswire.com/news-release/2026/01/27/3226324/0/en/opus-genetics-launches-gene-therapy-clinical-trial-for-mertk-related-retinitis-pigmentosa.html Context: Opus Genetics begins a clinical trial for MERTK-related retinitis pigmentosa, a genetic disorder causing progressive vision loss. Key Point: This trial will assess the safety and efficacy of OPGx-MERTK, a gene therapy developed to restore vision in affected individuals. Implication: The trial offers hope for patients with a disease currently without effective treatments.

🧬 Genethon Advances Duchenne Gene Therapy Through Licensing and Clinical Trials [EU • 27 Jan 2026]

https://www.biospace.com/press-releases/genethon-advances-groundbreaking-gene-therapies-for-patients-with-rare-genetic-diseases
Context: Genethon makes strides in gene therapies for Duchenne muscular dystrophy through clinical trials and licensing deals.
Key Point: Genethon continues to push forward with pivotal trials for its Duchenne gene therapy program, supported by new licensing agreements.
Implication: This is a significant development for rare disease treatments, potentially improving outcomes for Duchenne patients.

🤖 Multiply Labs Partners with AstraZeneca to Automate Cell Therapy Manufacturing [US • 27 Jan 2026]

https://theaiinsider.tech/2026/01/27/multiply-labs-announces-new-collaboration-with-astrazeneca-to-automate-cell-therapy-manufacturing-with-robotics/
Context: Multiply Labs collaborates with AstraZeneca to automate commercial-scale cell therapy manufacturing using robotics.
Key Point: The collaboration aims to enhance efficiency and consistency in the manufacturing of cell therapies.
Implication: Automation could significantly reduce costs and time-to-market for cell-based treatments.

🏭 Catalent to Wind Down EU Cell Therapy Manufacturing Hub [EU • 26 Jan 2026]

https://www.fiercepharma.com/manufacturing/catalent-moves-wind-down-operations-eu-cell-therapy-manufacturing-hub
Context: Catalent announces plans to close its cell therapy manufacturing facility in Belgium, impacting 150 employees.
Key Point: The closure is part of a broader realignment following changing market dynamics.
Implication: This move reflects ongoing challenges in the cell therapy industry, with shifts in demand and capacity.

Why It Matters

Regulatory Evolution: The FDA and other regulators continue to streamline processes, helping accelerate the development of groundbreaking cell and gene therapies.
Advancements in Rare Diseases: New therapies targeting Duchenne muscular dystrophy, inherited retinal diseases, and Huntington’s disease are making significant strides.
Strategic Partnerships: Collaborations between companies, such as AstraZeneca and Multiply Labs, are pivotal to accelerating the manufacturing and delivery of cell therapies.

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FAQ

What does the FDA’s Breakthrough Therapy Designation mean for Wugen’s WU-CART-007?

This designation will expedite the development of Wugen’s CAR-T therapy, potentially leading to faster approval for treating T-ALL/T-LBL.

What’s the significance of ImmunityBio’s Phase 2 glioblastoma data?

The positive results suggest ANKTIVA® plus CAR-NK could become a new treatment option for glioblastoma patients, offering improved survival rates.

Why is Beam Therapeutics focusing on in vivo delivery for sickle cell therapies?

Shifting to in vivo delivery could reduce manufacturing complexities and improve the speed at which sickle cell therapies reach patients.

How will Sarepta’s long-term data for Elevidys impact Duchenne treatments?

The three-year follow-up data reinforces Elevidys’ potential as a long-term solution for Duchenne muscular dystrophy.

Entities / Keywords

FDA; Wugen; WU-CART-007; ImmunityBio; ANKTIVA®; CAR-NK; Beam Therapeutics; sickle cell disease; Sarepta; Elevidys; Intellia; CRISPR; Opus Genetics; MERTK; Duchenne muscular dystrophy; Genethon; Multiply Labs; AstraZeneca; Catalent.