Top FDA Gene and Cell Therapy News: 2025 Year-End Recap

As 2025 comes to a close, CGTLive has looked back on some of the most-read and most impactful FDA regulatory decisions and news in the cell and gene therapy space. These approvals and regulatory actions marked major progress for patients with previously limited or no treatment options, and also reflected increasing FDA flexibility in areas of rare disease, novel delivery mechanisms, and advanced biologics manufacturing.

FDA Approves Encelto, First-Ever Treatment for Macular Telangiectasia Type 2

March 6, 2025 — Neurotech Pharmaceuticals made history in 2025 with the FDA approval of Encelto (revakinagene taroretcel-lwey), the first-ever therapy approved for idiopathic macular telangiectasia type 2 (MacTel). Encelto is an allogeneic, encapsulated cell therapy implant that continuously secretes ciliary neurotrophic factor (CNTF) directly into the vitreous cavity of the eye. The approval was supported by two phase 3 trials showing a significant reduction in ellipsoid zone area loss—a marker of photoreceptor degeneration—of 56.4% (P <.0001) and 29.2% (P = .021), respectively.

The implant represents a novel platform for sustained protein delivery in retinal disease and is expected to be available in the U.S. in mid-2025. According to Neurotech, common adverse events include conjunctival hemorrhage, delayed dark adaptation, and foreign body sensation, among others. The approval signals a critical step forward for patients at risk of vision loss from MacTel, for whom no FDA-approved therapies previously existed.

>>Click here to read further

Pfizer Pulls Plug on FDA-Approved Hemophilia B Gene Therapy Beqvez

February 21, 2025 — In a surprising turn, Pfizer announced in 2025 that it would discontinue fidanacogene elaparvovec (Beqvez), its FDA-approved adeno-associated virus (AAV)-based gene therapy for adults with moderate to severe hemophilia B. Despite gaining FDA approval in April 2024 for patients without AAVRh74var-neutralizing antibodies, commercial uptake was nonexistent—no patients received the therapy post-approval. The company cited lack of community demand and clinician engagement, marking a rare example of a gene therapy withdrawn despite regulatory clearance.

Beqvez had shown promising data in the BENEGENE-2 phase 3 trial (NCT03861273), with stable factor IX expression and decreased bleeding rates. However, safety concerns—including serious gastrointestinal bleeding events likely related to steroid use—may have contributed to hesitation among clinicians. Its high price tag ($3.5 million) and challenges around steroid management and clinical infrastructure may have further limited real-world adoption.

Pfizer’s decision reflects broader challenges in commercializing hemophilia gene therapies. BioMarin’s Roctavian and UniQure’s Hemgenix have similarly faced slow market uptake despite regulatory approval. Pfizer’s exit leaves it without any gene therapy assets in development, raising questions about long-term investment in this modality within hematology.

>>Click here to read further

Tab-Cel for EBV+ PTLD Receives CRL Due to Manufacturing Inspection Issues

January 16, 2025 — Despite strong clinical data, Atara Biotherapeutics’ allogeneic EBV-specific T-cell therapy, tabelecleucel (Ebvallo), received a complete response letter (CRL) from the FDA due to inspection issues at a third-party manufacturing site. The agency did not raise concerns about the therapy’s efficacy or safety, which had been supported by a 50.7% overall response rate and a 28% complete response rate in the phase 3 ALLELE trial.

The CRL highlights ongoing regulatory scrutiny around manufacturing and quality control in cell therapy. Atara, in partnership with Pierre Fabre, intends to resubmit once the identified issues are resolved, potentially leading to U.S. approval within six months. Ebvallo is already approved in Europe and the UK, and Atara emphasized its commitment to bringing this first-in-class therapy to U.S. patients with EBV+ PTLD, a rare post-transplant complication with no approved treatment options.

In July 2025, Atara's BLA resubmission for tab-cel was accepted with priority review by the agency, with a Prescription Drug User Fee Act (PDUFA) target action date set for January 10, 2026.

>>Click here to read further

MCO-010 Gene-Agnostic Retinitis Pigmentosa Therapy Advances With Rolling BLA

September 4, 2025 — Nanoscope Therapeutics submitted the first modules of a rolling biologics license application (BLA) to the FDA for MCO-010 (sonpiretigene isteparvovec), an ambient-light activatable gene therapy for retinitis pigmentosa (RP). The therapy uses an AAV2 vector to deliver multi-characteristic opsin (MCO) to bipolar retinal cells, restoring photosensitivity through intravitreal injection—without surgery or genetic testing. If approved, it would be the first gene-agnostic gene therapy for a retinal disorder.

MCO-010 met its primary end points for best corrected visual acuity (BCVA) in the phase 2b RESTORE trial (NCT04945772), with vision gains sustained through long-term follow-up. Data from a separate phase 1/2a trial in India also showed consistent BCVA improvements. No serious ocular adverse events were observed.

The therapy has received fast-track designation and is eligible for priority review. Experts have called it a potential paradigm shift, offering hope to RP patients regardless of their genetic mutations. Notably, Nanoscope aims to complete its BLA submission in early 2026. If successful, the therapy could become a much-needed non-surgical, office-based treatment for patients with severe, irreversible photoreceptor loss.

>>Click here to read further

Deramiocel for DMD Cardiomyopathy Moves Forward With BLA and Priority Review, Followed by CRL

March 4, 2025 — Capricor Therapeutics’ allogeneic cell therapy, deramiocel (CAP-1002), was granted Priority Review by the FDA for the treatment of Duchenne muscular dystrophy (DMD)-associated cardiomyopathy. The agency accepted Capricor’s BLA and assigned a PDUFA target action date of August 31, 2025.

Derived from cardiosphere-derived cells, deramiocel represents a first-in-class therapeutic targeting a key cause of morbidity and mortality in DMD patients. The BLA was supported by data from the HOPE-2 trial (NCT03406780) and its open-label extension (NCT04428476), which demonstrated statistically significant preservation of left ventricular ejection fraction and upper limb function.

The therapy is administered quarterly and has shown a favorable safety profile. According to Capricor, deramiocel may become a lifelong supportive treatment if approved. No advisory committee meeting has yet been scheduled, and the FDA did not note any review issues at the time of acceptance.

In July, the agency responded to the application with a complete response letter, stating that the statutory requirement for “substantial evidence of effectiveness” was not met by the BLA and that more clinical data would be needed. A confirmatory phase 3 trial, HOPE-3, remains ongoing. Capricor’s efforts reflect a growing clinical recognition of cardiac decline as a critical therapeutic target in DMD. Deramiocel’s potential approval would fill a major treatment gap for cardiomyopathy in this population and could mark a new standard of care for multisystem support in DMD.

>>Click here to read further

FDA Grants Breakthrough Therapy Designation to AMT-130 for Huntington Disease

April 24, 2025 — UniQure’s AMT-130 gene therapy for Huntington disease received breakthrough therapy designation (BTD) from the FDA in 2025, marking another major step in its expedited development. The therapy, which uses an AAV vector to deliver microRNA targeting huntingtin mRNA, had previously earned regenerative medicine advanced therapy (RMAT), orphan drug, and fast track designations.

The BTD was supported by promising interim phase 1/2 data comparing treated patients to an external control cohort developed with CHDI Foundation. In the high-dose arm, AMT-130 slowed progression on the composite Unified Huntington’s Disease Rating Scale by 80% (P = .007) at 24 months. Cerebrospinal fluid neurofilament light chain levels dropped 11% from baseline (P = .02), supporting the potential for neuroprotective effect.

The FDA has agreed that AMT-130 may pursue accelerated approval based on composite UHDRS as an intermediate end point, with CSF biomarker reductions used as supportive data. No new trials are required before BLA submission. This decision sets up AMT-130 to potentially become the first disease-modifying therapy approved for Huntington disease and a foundational case study in the use of natural history controls for neurodegenerative gene therapy.

>>Click here to read further

Amvuttra Approved for ATTR-CM, Expanding RNAi Therapies in Cardiac Disease

March 20, 2025 — Alnylam Pharmaceuticals received FDA approval for vutrisiran (Amvuttra) for the treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM), expanding its label beyond its prior use in polyneuropathy. This subcutaneous RNA interference therapy demonstrated a 28% reduction in cardiovascular death and events during a 36-month blinded period in the HELIOS-B trial, with extended benefits through 42 months.

The decision marks a significant shift in the treatment paradigm for ATTR-CM, offering patients a novel mechanism of action targeting transthyretin synthesis. Amvuttra preserved functional capacity and quality of life in clinical trials and was well-tolerated, with no new safety signals reported. Alnylam leveraged a Priority Review voucher and did not require an advisory committee meeting, reflecting both urgency and confidence in the data. The approval continues the growing trend of RNA-based therapies achieving regulatory milestones in complex cardiovascular and rare systemic diseases.

>>Click here to read further

FDA Approves Zevaskyn, First Gene-Corrected Skin Therapy for RDEB

April 29, 2025 — Abeona Therapeutics’ prademagene zamikeracel (pz-cel; Zevaskyn) received FDA approval in 2025 for recessive dystrophic epidermolysis bullosa (RDEB), a devastating genetic skin disorder. The therapy is an autologous gene-corrected epidermal sheet surgically grafted onto patients’ wounds. Zevaskyn’s approval was based on the phase 3 VIITAL trial, which met its primary end points, demonstrating significant wound healing and pain reduction after a single application.

This approval follows a complex regulatory journey, including a 2024 complete response letter that was resolved via Chemistry, Manufacturing and Controls (CMC) clarification. Zevaskyn is now the second gene therapy approved for RDEB, joining Vyjuvek, and offers a complementary therapeutic option with durable, localized wound healing benefits. Abeona will launch a support program to assist patients with insurance and logistical needs. The company also received a rare pediatric disease Priority Review voucher, which it plans to sell, further underscoring the regulatory and financial significance of this milestone.

>>Click here to read further

Sanofi’s Qfitlia Wins Approval for Hemophilia A and B With or Without Inhibitors

March 31, 2025 — Sanofi’s fitusiran (Qfitlia), an siRNA-based antithrombin-lowering therapy, received FDA approval for routine prophylaxis in hemophilia A and B—regardless of inhibitor status. The ATLAS program showed the therapy reduced annualized bleeding rates by over 70% in both inhibitor and non-inhibitor populations compared to on-demand treatment.

Fitusiran is administered monthly, offering the lowest dose frequency of any approved prophylactic for hemophilia. A companion diagnostic, the Innovance Antithrombin Assay by Siemens Healthineers, was approved alongside Qfitlia to guide dosing. Common adverse events included viral infections and nasopharyngitis, with known risks of thrombotic events and gallbladder disease flagged for ongoing monitoring.

The approval adds a new treatment paradigm focused on thrombin rebalancing, with fewer infusions and broad applicability across hemophilia populations. Qfitlia joins the expanding class of RNA-based therapies reshaping treatment expectations for genetic and bleeding disorders.

>>Click here to read further