Long-term follow-up data and clinical holds define the start of 2026

Lilly entered $2.4B agreement to acquire Orna Therapeutics for in vivo CAR-T platform

Eli Lilly and Company announced a definitive agreement to acquire Orna Therapeutics in a transaction valued at up to $2.4B. Orna is developing circular RNA therapeutics delivered via lipid nanoparticles to enable in vivo cell engineering. Its lead candidate, ORN-252, is a clinical trial–ready CD19-targeted in vivo CAR-T cell therapy for B cell-driven autoimmune diseases. The acquisition provides Lilly with access to Orna’s circular RNA and delivery platform for in vivo CAR-T generation. The companies stated that the approach may address scalability and logistical limitations associated with ex vivo autologous CAR-T therapies [1]PR Newswire. Lilly to acquire Orna Therapeutics to advance cell therapies. Feb 9, 2026..

Lilly entered $2.4B agreement to acquire Orna Therapeutics for in vivo CAR-T platform; Market Trends. Credit: PRNewsfoto/Eli Lilly and Company.

Orca Bio raised $250M ahead of Orca-T PDUFA date

Orca Bio announced $250M in aggregate equity financing across two recent rounds, alongside an amended credit facility providing access to up to $100M in additional liquidity. The funding is intended to support commercial readiness ahead of the April 6, 2026 Prescription Drug User Fee Amendments (PDUFA) target action date for Orca-T, an investigational allogeneic T cell immunotherapy. Capital will also support manufacturing expansion, including East Coast capacity, and continued development of Orca-T and Orca-Q in additional conditioning settings. The financing reflects continued investment in late-stage cell therapy programs approaching potential regulatory decisions [2]Business Wire. Orca Bio announces $250M in aggregate financing in preparation for potential commercialization. Jan 9, 2026..

Innovacell targeted $92M in Tokyo IPO to advance Phase 3 regenerative program

Japan-based Innovacell announced plans to raise approximately $92M through an initial public offering on the Tokyo Stock Exchange. The company is advancing ICEF15, a regenerative medicine therapy currently in a multi-regional Phase 3 trial for urge fecal incontinence. Proceeds are expected to support ongoing late-stage development in Japan and Europe, preparation for a US study, manufacturing scale-up, and commercialization infrastructure across major markets. The planned listing follows a Series D financing of approximately $48M in August 2025. Company leadership cited investor interest in late-stage biotech assets approaching commercialization as a contributing factor in timing the offering, amid limited recent biotech IPO activity in Japan [3]Japan Exchange Group. Innovacell listing outline. Feb 2026..

Dispatch Bio secured US FDA IND clearance for dual-component immunotherapy in solid tumors; Regulatory Changes and Updates. Credit: US FDA.

Intellia Therapeutics reported FDA lift of clinical hold on MAGNITUDE-2 Phase 3 trial

Intellia Therapeutics announced that the FDA lifted the clinical hold on the MAGNITUDE-2 Phase 3 trial evaluating nexiguran ziclumeran, a CRISPR-based gene editing therapy, in hereditary transthyretin amyloidosis with polyneuropathy. The hold had been initiated following a case of Grade 4 liver transaminase elevations in a related study. The company aligned with the FDA on protocol amendments, including enhanced liver monitoring and increased enrollment to approximately 60 patients. A separate clinical hold remains in place for the cardiomyopathy indication [4]GlobeNewswire. Intellia Therapeutics announces FDA lift of clinical hold on MAGNITUDE-2 Phase 3 clinical trial in ATTRv-PN. Jan 27, 2026..

Siren Biotechnology received FDA IND clearance for AAV-based immuno-gene therapy in glioma

Siren Biotechnology announced that the FDA cleared its first IND application, enabling initiation of a first-in-human clinical trial of an AAV-based immuno-gene therapy for recurrent high-grade glioma. The program previously received orphan drug and rare pediatric disease designations. The therapy is designed to deliver immune-modulating payloads directly within tumors. The clearance marks the company’s transition to clinical-stage development and supports evaluation in adult patients with recurrent disease [5]GlobeNewswire. Siren Biotechnology announces FDA clearance of first IND, advancing company to clinical stage. Jan 28, 2026..

FDA placed clinical holds on Regenxbio gene therapy trials

Regenxbio reported that the FDA placed clinical holds on two AAV gene therapy programs, RGX-111 for Hurler syndrome and RGX-121 for Hunter syndrome. The decision followed identification of a brain tumor in one patient approximately 4 years after receiving RGX-111. The FDA cited potential shared risk between the programs. No additional tumors were reported among other treated patients, and the company stated that investigations are ongoing [6]Reuters. US FDA places clinical hold on Regenxbio’s gene therapy trials. Jan 28, 2026..

Dispatch Bio secured FDA IND clearance for dual-component immunotherapy in solid tumors

Dispatch Bio announced FDA clearance of its IND application for DISP-10, a first-in-class immunotherapy comprising DV-10, a tumor-selective viral vector engineered to deliver a modified antigen and immunomodulatory factors, followed by Bristol Myers Squibb’s BCMA-directed CAR-T therapy (ide-cel). The Phase 1 study will evaluate the approach in solid tumors, beginning with gastrointestinal cancers. The strategy aims to enhance CAR-T activity in solid tumors by modifying the tumor microenvironment and introducing a synthetic antigen target [7]Business Wire. Dispatch Bio announces FDA clearance of IND application for DISP-10, a first-in-class immunotherapy treatment, in solid tumors. Jan 11, 2026..

Zemcelpro received NUB Status 1 in Germany following conditional European approval

ExCellThera and its subsidiary Cordex Biologics announced that Zemcelpro (dorocubicel), a UM171-expanded cord blood stem cell therapy, was granted NUB Status 1 in Germany. The designation enables hospitals to apply for temporary supplementary reimbursement while broader reimbursement pathways are evaluated. Zemcelpro recently received conditional marketing authorization from the European Commission for adults with hematological malignancies requiring allogeneic hematopoietic stem cell transplantation who lack suitable donors. The company is pursuing additional regulatory filings internationally [8]PR Newswire. Zemcelpro^® (dorocubicel) receives NUB Status 1 in Germany. Feb 16, 2026..

FDA granted RMAT designation to inhaled gene therapy for advanced lung tumors

The FDA granted regenerative medicine advanced therapy designation to KB707, an inhaled gene therapy under development by Krystal Biotech for locally advanced or metastatic lung tumors progressing after standard therapy. The designation was based on preliminary clinical evidence suggesting potential benefit in non-small cell lung cancer. Regenerative medicine advanced therapy status provides enhanced regulatory interaction and eligibility for accelerated approval pathways [9]Krystal Biotech. Krystal Biotech receives FDA fast track designation for inhaled gene therapy. Feb 2026..

Ultragenyx reported long-term functional benefit of UX111 in Sanfilippo syndrome

Ultragenyx presented up to 8.5-year follow-up data for UX111, an AAV9 gene therapy for Sanfilippo syndrome Type A. The data demonstrated sustained reductions in cerebrospinal fluid heparan sulfate and continued functional improvements across developmental domains compared with natural history data. The therapy was reported to be well tolerated, with a favorable safety profile maintained over long-term follow-up. The company resubmitted its Biologics License Application (BLA) to the FDA in January 2026, with a review period of up to 6 months anticipated [10]GlobeNewswire. Ultragenyx announces positive longer-term data demonstrating treatment with UX111 gene therapy results in sustained reductions in CSF-HS. Feb 3, 2026..

Sarepta reported 3-year Phase 3 EMBARK data for ELEVIDYS in Duchenne muscular dystrophy

Sarepta announced 3-year results from the EMBARK Phase 3 study evaluating ELEVIDYS, an AAV gene therapy for ambulatory patients with Duchenne muscular dystrophy. Treated patients maintained mean North Star Ambulatory Assessment scores above baseline and demonstrated 70% or greater slowing of decline in time to rise and 10-meter walk/run compared with an external control group. No new treatment-related safety signals were reported. ELEVIDYS is currently approved in the USA for ambulatory individuals aged 4 years and older [11]Business Wire. Sarepta announces positive topline three-year EMBARK results showing ELEVIDYS significantly slows disease progression. Jan 26, 2026..

Sarepta reported 3-year Phase 3 EMBARK data for ELEVIDYS in Duchenne muscular dystrophy; Clinical Trials and Research. Credit: https://unsplash.com.

ImmunityBio reported durable responses with off-the-shelf CD19 CAR-NK therapy

ImmunityBio provided updated results from the ongoing QUILT-106 study evaluating an allogeneic CD19-targeted CAR-NK therapy combined with rituximab in Waldenström Non-Hodgkins lymphoma. Among the first four treated patients, the company reported 100% disease control, including complete responses lasting up to 15 months without further therapy. The regimen was administered without chemotherapy or lymphodepletion and in an outpatient setting [12]Business Wire. ImmunityBio announces durable complete response of 15 months with a chemotherapy-free CD19 CAR-NK cell therapy in Waldenstrom lymphoma. Jan 16, 2026..

Boehringer Ingelheim discontinued inhaled lentiviral vector gene therapy in cystic fibrosis

Boehringer Ingelheim terminated development of BI 3720931, an inhaled lentiviral vector gene therapy for cystic fibrosis, following Phase 1/2 evaluation in five patients. The company stated that clinical data did not support further development, although the safety profile was consistent with expectations. The program, known as Lenticlair 1, evaluated multiple dose levels and included a placebo-controlled expansion phase [13]Fierce Biotech. Boehringer axes inhaled gene therapy after viewing cystic fibrosis data. Feb 2026..

First NHS patient received obe-cel CAR-T therapy for acute lymphoblastic leukemia

The first patient in England was treated within the National Health Service with obe-cel (Aucatzyl^®), a CD19-directed CAR-T therapy for B cell acute lymphoblastic leukemia. The therapy, developed by UCL researchers and commercialized by Autolus, received approval from the National Institute for Health and Care Excellence in November. Obe-cel was designed to reduce immune toxicity and improve persistence relative to earlier CAR-T constructs. Treatment was administered in two doses at Manchester Royal Infirmary [14]UCL News. First leukaemia patient treated with pioneering therapy. Jan 2026..

First NHS patient received obe-cel CAR-T therapy for acute lymphoblastic leukemia; Clinical Trials and Research. Credit: https://commons.wikimedia.org.

Advanced Therapies Week 2026 highlighted operational and regulatory shifts in CGT

Phacilitate’s Advanced Therapies Week 2026 convened stakeholders to discuss emerging trends in cell and gene therapy. Key themes included increasing investment in in vivo therapies, alternative financing mechanisms, and evolving regulatory transparency, including discussion of the FDA’s move toward real-time publication of Complete Response Letters. Speakers emphasized manufacturing scalability, infrastructure readiness, and diversification across regulatory regions as central to the next phase of sector growth [15]Phacilitate. Advanced Therapies Week 2026. Feb 2026..

As a commissioning editor with extensive experience in advanced therapy journal publishing, Abi’s focus is on progressing the field by facilitating and disseminating high-impact, open access content covering novel and existing cell and gene therapies. Abi works closely with academic scientists and industry professionals to publish cutting-edge original research, expert reviews, and multimedia content with a translational and interdisciplinary focus. Abi’s key aim is to explore the latest advances in cell and gene therapy R&D, clinical development, manufacturing, and commercialization. In addition to Abi’s editorial responsibilities, she maintains a strong network of experts across the biotech and pharma industries, staying up to date with emerging trends and breakthroughs in advanced therapies.

References

1. PR Newswire. Lilly to acquire Orna Therapeutics to advance cell therapies. Feb 9, 2026.

2. Business Wire. Orca Bio announces $250M in aggregate financing in preparation for potential commercialization. Jan 9, 2026.

3. Japan Exchange Group. Innovacell listing outline. Feb 2026.

4. GlobeNewswire. Intellia Therapeutics announces FDA lift of clinical hold on MAGNITUDE-2 Phase 3 clinical trial in ATTRv-PN. Jan 27, 2026.

5. GlobeNewswire. Siren Biotechnology announces FDA clearance of first IND, advancing company to clinical stage. Jan 28, 2026.

6. Reuters. US FDA places clinical hold on Regenxbio’s gene therapy trials. Jan 28, 2026.

7. Business Wire. Dispatch Bio announces FDA clearance of IND application for DISP-10, a first-in-class immunotherapy treatment, in solid tumors. Jan 11, 2026.

8. PR Newswire. Zemcelpro^® (dorocubicel) receives NUB Status 1 in Germany. Feb 16, 2026.

9. Krystal Biotech. Krystal Biotech receives FDA fast track designation for inhaled gene therapy. Feb 2026.

10. GlobeNewswire. Ultragenyx announces positive longer-term data demonstrating treatment with UX111 gene therapy results in sustained reductions in CSF-HS. Feb 3, 2026.

11. Business Wire. Sarepta announces positive topline three-year EMBARK results showing ELEVIDYS significantly slows disease progression. Jan 26, 2026.

12. Business Wire. ImmunityBio announces durable complete response of 15 months with a chemotherapy-free CD19 CAR-NK cell therapy in Waldenstrom lymphoma. Jan 16, 2026.

13. Fierce Biotech. Boehringer axes inhaled gene therapy after viewing cystic fibrosis data. Feb 2026.

14. UCL News. First leukaemia patient treated with pioneering therapy. Jan 2026.

15. Phacilitate. Advanced Therapies Week 2026. Feb 2026.