Cell and Gene Therapy Today—January 22, 2026

In Today’s Newsletter

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🏛️ Nanoscope secures Japan MHLW Sakigake and Orphan Drug designations for retinal gene therapy [1] [US • 20 Jan 2026]

https://www.prnewswire.com/news-releases/nanoscope-secures-japan-mhlw-sakigake-and-orphan-drug-designations-across-inherited-retinal-diseases–a-first-for-a-retinal-gene-therapy-302664876.html
Context: Nanoscope’s MCO-010, an optogenetic gene therapy for inherited retinal diseases, has been granted the Sakigake and Orphan Drug designations by Japan’s Ministry of Health, Labour and Welfare.
Key point: These designations allow accelerated regulatory pathways and global pre-commercialization readiness.
Implication: Signals continued momentum in the gene therapy space with expanded regulatory recognition.

🤝 Fresenius Kabi teams up with TQ Therapeutics to scale cell therapy manufacturing [2] [US • 20 Jan 2026]

https://www.businesswire.com/news/home/20260114359760/en/Fresenius-Kabi-TQ-Therapeutics-Announce-Cell-Therapy-Technology-Agreement
Context: Fresenius Kabi secures an exclusive license to TQ Therapeutics’ cell selection technology to improve manufacturing processes.
Key point: The partnership aims to advance scalable and efficient cell therapy manufacturing for broader patient access.
Implication: Highlights advancements in cell and gene therapy manufacturing.

🧪 AstraZeneca acquires full global rights to C-CAR031 CAR-T therapy [3] [19 Jan 2026]

https://www.fiercebiotech.com/biotech/astrazeneca-pens-630m-pact-secure-remaining-rights-armored-car-t
Context: AstraZeneca finalizes a $630M deal for the remaining global rights to C-CAR031, an armored CAR-T therapy for hepatocellular carcinoma (HCC).
Key point: AstraZeneca now owns full rights to C-CAR031 after initial co-licensing with AbelZeta.
Implication: Aims to expand the global reach of C-CAR031 for treating HCC.

🧬 ImmunityBio reports durable responses with CAR-NK + rituximab for Waldenström Lymphoma [4] [US • 16 Jan 2026]

https://www.businesswire.com/news/home/20260116046721/en/ImmunityBio-Announces-Durable-Complete-Response-of-15-Months-with-a-Chemotherapy-Free-CD19-CAR-NK-Cell-Therapy-in-Waldenstrom-Lymphoma
Context: ImmunityBio’s QUILT-106 trial reports long-term complete responses with CD19 CAR-NK therapy for Waldenström lymphoma patients.
Key point: CAR-NK + rituximab shows durable remissions, with one patient achieving 15 months of complete response.
Implication: Could revolutionize immunotherapy for lymphoma with long-lasting, chemotherapy-free options.

💡 AGC Biologics celebrates approvals for Waskyra™ gene therapy [5] [US • 16 Jan 2026]

https://www.agcbio.com/news/agc-biologics-celebrates-u.s.-and-eu-approval-of-fondazione-telethons-waskyra-treatment-for-wiskott-aldrich-syndrome
Context: Waskyra™, a gene therapy for Wiskott-Aldrich syndrome, receives regulatory approval in the US and EU.
Key point: AGC Biologics is integral to Waskyra’s commercialization process.
Implication: Provides a significant step forward in the treatment of rare immune disorders.

👁️ Ocugen announces positive Phase 2 data for OCU410 in geographic atrophy [6] [US • 15 Jan 2026]

https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-positive-preliminary-phase-2-data-ocu410
Context: Ocugen presents promising Phase 2 data showing a 46% reduction in lesion growth with OCU410 for geographic atrophy in dry AMD.
Key point: The therapy shows slower progression of retinal degeneration in treated patients.
Implication: Could offer a new long-term treatment option for dry AMD patients.

📝 Capricor provides update on Deramiocel BLA for Duchenne muscular dystrophy [7] [US • 20 Jan 2026]

https://www.globenewswire.com/news-release/2026/01/20/3221849/0/en/Capricor-Therapeutics-Provides-Regulatory-Update-on-Deramiocel-BLA-Following-FDA-Review-of-HOPE-3-Topline-Data.html
Context: Capricor responds to FDA requests for more data related to its Duchenne muscular dystrophy therapy, Deramiocel.
Key point: The company plans to submit additional materials to the FDA to support its Biologics License Application.
Implication: A step closer to potential approval for a novel treatment for DMD.

🧠 iRegene secures RMAT for NouvNeu001, an iPSC therapy for Parkinson’s Disease [8] [US • 19 Jan 2026]

https://www.prnewswire.com/news-releases/fda-grants-regenerative-medicine-advanced-therapy-rmat-designation-to-iregenes-nouvneu001-making-it-the-worlds-first-ipsc-therapy-with-both-ftd-and-rmat-recognitions-2261156
Context: iRegene’s NouvNeu001, a Parkinson’s disease therapy, has received both Fast Track and RMAT designations from the FDA.
Key point: The recognition accelerates the therapy’s development and review processes.
Implication: Highlights potential for groundbreaking Parkinson’s disease treatment.

💉 Lukas Bio reports positive results for CIK cell therapy in high-risk lymphoma [9] [21 Jan 2026]

https://www.koreabiomed.com/news/articleView.html?idxno=30370
Context: Lukas Bio’s CIK cell therapy shows significant improvements in survival and recurrence prevention for high-risk lymphoma patients.
Key point: CIK cell therapy improves outcomes after hematopoietic stem cell transplantation.
Implication: Potential breakthrough for high-risk lymphoma treatment.

Why It Matters

Nanoscope’s Sakigake designation highlights global regulatory progress in optogenetic therapies.
Fresenius Kabi and TQ Therapeutics’ collaboration signals expansion in cell therapy manufacturing.
AstraZeneca’s acquisition of C-CAR031 provides further evidence of CAR-T’s growing significance in oncology.
ImmunityBio’s CAR-NK data for Waldenström lymphoma could set a new standard in lymphoma treatment.
AGC Biologics plays a key role in advancing gene therapies for rare diseases, including Wiskott-Aldrich syndrome.

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FAQ

What is the significance of Nanoscope’s Sakigake designation for MCO-010?

Nanoscope’s receipt of the Sakigake designation is crucial as it accelerates the regulatory process for their optogenetic retinal gene therapy, MCO-010, in Japan, potentially bringing the treatment to patients faster.

How does Fresenius Kabi’s partnership with TQ Therapeutics impact cell therapy manufacturing?

This partnership will advance scalable manufacturing technologies, making cell therapies more accessible to patients by enhancing efficiency and reliability in production.

What does AstraZeneca’s acquisition of C-CAR031 mean for CAR-T therapies?

AstraZeneca’s full acquisition of C-CAR031 solidifies its position in the CAR-T space, ensuring global access to a promising therapy for hepatocellular carcinoma (HCC).

How does ImmunityBio’s CAR-NK therapy work for Waldenström lymphoma?

ImmunityBio’s CAR-NK therapy combined with rituximab offers a promising, chemotherapy-free treatment option that demonstrates long-term, durable remissions in patients with Waldenström lymphoma.

What does iRegene’s RMAT designation for NouvNeu001 mean for Parkinson’s disease treatment?

The RMAT designation accelerates the development and review of NouvNeu001, an iPSC-derived therapy for Parkinson’s disease, which could potentially revolutionize the treatment of this debilitating condition.

Entities / Keywords

FDA; Sakigake; Orphan Drug Designations; RMAT; CAR-T; iPSC; C-CAR031; MCO-010; Fresenius Kabi; TQ Therapeutics; ImmunityBio; Waldenström lymphoma; Ocugen; Deramiocel.