Data Roundup: December 2025 Features Updates from ASH and CTAD

Last month, December 2025, the CGTLive® team was diligently tracking the latest data readouts and published literature on cell and gene therapies within oncology, neurology, rare diseases, and more.

As more and more innovative therapies enter the clinical trial field, more data is accrued every month, buoying excitement in the field and sometimes making or breaking the fates of small biotech companies. Last month delivered data updates from conferences including the 67th American Society of Hematology (ASH) Annual Meeting and Exposition and the 18th Clinical Trials on Alzheimer’s Disease (CTAD) conference. Our team has highlighted these below.

Click the read more buttons for more details and information about each update.

FasTCAR Manufactured BCMAxCD19 CAR T-cell Therapy Shows Promise for Multiple Myeloma

December 7, 2025 - The next-generation dual BCMA and CD19-targeted chimeric antigen receptor T-cell (CAR-T) therapy AZD0120 that was manufactured rapidly using the FasTCAR technology elicited an objective response in nearly all patients with relapsed/refractory multiple myeloma (MM), according to preliminary findings from the phase 1b/2 DURGA-1 study (NCT05850234) presented at the 2025 ASH Annual Meeting.

After a median follow-up of 3.9 months, the overall response rate (ORR) with AZD0120 across 2 dose levels was 96%, with a median time to response of 28 days. The complete response (CR) and stringent CR (sCR) rate with AZD0120 was 78.3% and the partial response (PR) rate was 17.4%.

"A single infusion of AZD0120 resulted in early and deep responses. Response deepens over time and were seen in BCMA-naive and exposed patients," lead investigator Shambavi Richard, MD, from the Icahn School of Medicine at Mount Sinai, said during a presentation of the results. "The safety profile is well-suited for outpatient administration, with 35% of patients receiving infusion outpatient."

Autologous Stem Cell Therapy for Alzheimer Disease Advances to Phase 2 After Positive Early Findings

December 4, 2025 - At the 18th CTAD conference, held December 1-4, 2025, in San Diego, California, investigators shared promising phase 1 data supporting the safety and feasibility of RB-ADSC, an autologous Wnt-enriched stem cell therapy delivered directly to the brain for patients with Alzheimer disease (AD).

Developed by Regeneration Biomedical (RBI), the therapy leverages intracerebroventricular administration via an Ommaya reservoir to target ventricular stem cell niches. Following positive early results, the program has progressed to a phase 2 clinical trial, which began enrolling patients in November 2025.

Seven patients were included in the CTAD analysis out of 9 total in the dose-escalation phase 1 study. Participants were under 80 years old, had Mini-Mental State Examination (MMSE) scores between 11 and 20, and demonstrated evidence of AD through elevated PET and cerebrospinal fluid (CSF) biomarkers. All were categorized as FAST stage 4 or 5.

Steven W. Pipe, MD, on the End-of-Study Results From Hemgenix’s HOPE-B Trial

December 11, 2025 - CSL Behring/uniQure’s etranacogene dezaparvovec (marketed as Hemgenix), a gene therapy product for the treatment of severe or moderately severe hemophilia B with or without preexisting AAV5 neutralizing antibodies (NAbs), was approved by the FDA in November 2022. The therapy is intended to provide durable factor IX (FIX) expression, bleed protection, and freedom from prophylaxis.

The FDA’s decision to approve Hemgenix, which was the first hemophilia B gene therapy to be approved by the agency, was based partly on results from the pivotal phase 3 HOPE-B clinical trial (NCT03569891). Although, long-term follow-up of the study’s participants remained ongoing until early 2025.

Finally, at the 67th ASH Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida, end-of-study data from HOPE-B were presented by Steven W. Pipe, MD, a professor of pediatric hematology/oncology at the University of Michigan Health, who served as the trial’s principal investigator. CGTLive® sat down with Pipe at the conference to learn more about the updated findings.

Evaluating Gene Editing Therapy Reni-Cel for Severe Sickle Cell Disease

December 14, 2025 - There are currently 2 FDA-approved gene therapy treatments available for sickle cell disease (SCD)—Vertex Pharmaceuticals' and CRISPR Therapeutics’ gene editing therapy exagamglogene autotemcel (exa-cel; marketed as Casgevy) and bluebird bio’s gene addition therapy lovotibeglogene autotemcel (lovo-cel; marketed as Lyfgenia)—but development of new genomic medicine options for SCD remains ongoing with the aim of filling in gaps in need unserved by the current options. One such investigational candidate is renizgamglogene autogedtemcel (reni-cel, previously known as EDIT-301), a CRISPR-Cas12a gene editing candidate for severe SCD that was evaluated in the phase 1/2 RUBY clinical trial (NCT04853576).

At the 67th ASH Annual Meeting, CGTLive® spoke to Rabi Hanna, MD, the chairman of the Division of Pediatric Hematology & Oncology and BMT at Cleveland Clinic Children's, shortly before he presented new data from RUBY at the conference. Hanna went over the key findings he would be presenting and also spoke about areas of interest for further research in SCD gene therapy.

Deevyashali Parekh, MBBS, on Evaluating CAR-T in Patients With Multiple Myeloma and Renal Failure

December 9, 2025 - There are currently 2 CAR-T therapies approved by the FDA for use in the treatment of MM: Bristol Myers Squibb (BMS) and 2seventybio’s idecabtagene vicleucel (ide-cel; marketed as Abecma) and Janssen and Legend Biotech’s ciltacabtagene autoleucel (cilta-cel; marketed as Carvykti). When these therapies were in clinical trials, the studies excluded patients with certain conditions from participation that may actually now be eligible for treatment with the commercial products. Of note in this regard are patients with renal failure, who were not able to participate in the studies, but some of whom have now been treated in the real-world setting. Because of the lack of clinical trial data on outcomes for patients with renal failure, gathering real-world data on such patients who receive CAR-T in the real-world is of critical importance to inform treatment decisions for future patients.

Notably, Deevyashali Parekh, MBBS, an internal medicine resident at SUNY Upstate Medical University Hospital, presented a real-world study intended to help address this gap in knowledge at the 67th ASH Annual Meeting. The retrospective, multicenter real-world study utilized data from the TrinetX database network to compare outcomes for patients with and without renal failure who received CAR-T for MM. At the conference, CGTLive® interviewed Parekh learn more about the findings and get his insight on their implications for the field.