FDA approved one CGT BLA in Q3 2025. Precigen, Inc. received approval of Papzimeos (zopapogene imadenovec-drba), a non-replicating adenoviral immunotherapy for recurrent respiratory papillomatosis (RRP), on August 14, 2025. RRP is a chronic disease caused by persistent human papillomavirus (HPV) 6 or 11 infection, leading to the growth of benign tumors in the respiratory tract.
The review dates of two BLAs were delayed three months due to major amendments. The revised due date for Regenexbio’s RGX-121 (clemidsogene lanparvovec) will now be February 8, 2026 after submission of longer-term clinical data for all patients in the pivotal study in response to an FDA information request. Axogen Corporation now expects an FDA decision on Avance Nerve Graft by December 5, 2025 after submission of substantial new manufacturing and facility data requested by the FDA.
Complete response letters (CRL) were issued to three BLAs in close succession. On July 11, 2025, Capricor announced receipt of a CRL from FDA on their BLA application for deramiocel for treatment of Duchenne muscular dystrophy (DMD) cardiomyopathy. The company’s press release reported that the CRL stated that additional clinical data are need, citing “the BLA does not meet the statutory requirement for substantial evidence of effectiveness” and that certain CMC items were outstanding. Also on July 11, Ultragenyx announced receipt of a CRL from FDA on their BLA application for UX111 for treatment of Sanfilippo syndrome type A (MPS IIIA). The company’s press release stated that the CRL requested “additional information and improvements related to specific aspects of CMC and observations from the recently completed manufacturing facility inspections.” On July 22, 2025, Replimune Group, Inc., announced receipt of a CRL for RP1 (vusolimogene oderparepvec) in combination with nivolumab for the treatment of advanced melanoma. The company’s press release stated the CRL indicated that the IGNYTE trial is not considered to be an adequate and well-controlled clinical investigation that provides substantial evidence of effectiveness, the trial cannot be adequately interpreted due to the heterogeneity of the patient population, and that there are items related to the confirmatory trial study design which need to be addressed, including contribution of components.
For the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who:
Patients aged 12 years and older with:
Hemophilia B (congenital Factor IX deficiency) adults with Hemophilia B (congenital Factor IX deficiency) who:
**This indication is approved under accelerated approval based on overall response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
List does not include HPC, Cord Blood products for use in unrelated donor hematopoietic progenitor cell transplantation procedures.
List also does not include previously approved products that are no longer being marketed.
The following information on BLAs currently under US FDA review was obtained from company press releases.
No upcoming meetings of the Cellular, Tissue, and Gene Therapies Advisory committee for CGT topics have been announced.
Upcoming:
OTP Town Hall: Gene Therapy Manufacturing CMC and Facility Readiness for BLAs and Post-licensure Changes- October 22, 2025
Individualized Therapies On The Rise - November 20, 2025
Recent past:
FDA Public Meeting: Onshoring Manufacturing of Drugs and Biological Products – September 30, 2025
Lessons Learned From the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program - September 10, 2025
On the RISE: Controls in Rare Disease Clinical Trials for Small and Diminishing Populations – September 3, 2025
FDA Roundtable on Cell and Gene Therapy June 5, 2025
OTP Town Hall: Best Practices for Regulatory Interactions with OTP – December 12, 2024
Meeting 2: Patient and Care Partner Perspectives on Early Enrollment into Gene Therapy Clinical Trials for Rare Diseases –
December 4, 2024
Workshop on Integration Site Analysis During Long Term Follow-Up for Gene Therapies with Integrating Viral Vectors –
November 19, 2024
Finding Your Support Team While Participating in a Clinical Trial –
October 30, 2024
Advancing Rare Disease Therapies Through an FDA Rare Disease Innovation Hub
October 16, 2024-recording not yet available
OTP Town Hall: Cell Therapy CMC Readiness for Late-Stage INDs
September 5, 2024- recording available
FDA Public Workshop on Best Practices for Meeting Management
July 22, 2024- recording and event materials available
The following links to past Meeting Materials include access to the recorded video link, transcript, or slides.
OTP Town Hall: CMC Readiness for Gene Therapy BLAs
June 4, 2024
Nonclinical Assessment of Cell and Gene Therapy Products
August 30, 2023
Decentralized Clinical Trials
June 20, 2023
Regulatory Education for Industry (REdI) Annual Conference 2023
June 5-9, 2023
OTP Town Hall: Cell Therapy Chemistry, Manufacturing, and Controls – June 2023
June 8, 2023
OTP Town Hall: Gene Therapy Chemistry, Manufacturing, and Controls – April 2023
April 25, 2023
Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products
April 27, 2023
Clinical Trials: The Patient Experience
April 13, 2023
FDA CBER Office of Therapeutic Products (OTP) Advanced Manufacturing and Analytical Technologies (AMAT) for Regenerative Medicine Therapies (RMT) Workshop
March 14, 2023
OTAT Town Hall: Clinical Development of Gene Therapy Products for Rare Diseases
February 7, 2023
Assessing Genetic Heterogeneity in the Context of Genome Editing Off-Targets in Gene Therapy Products
December 16, 2022
OTAT Town Hall: Cell Therapy Chemistry, Manufacturing, and Controls
December 7, 2022
FDA/PQRI Workshop on the Regulatory Framework for Distributed and Point of Care Pharmaceutical Manufacturing: An Opportunity for DM/POC Stakeholder Engagement
November 14 - 16, 2022
FDA CBER OTAT Patient-Focused Drug Development Listening Meeting — Patient Perspectives on Gene Therapy Products
November 15, 2022
Natural History Studies to Support Regenerative Medicine: A How-To Webinar
October 27, 2022
OTAT Town Hall: Gene Therapy Chemistry, Manufacturing, and Controls
September 29, 2022
2022 Center for Biologics Evaluation and Research (CBER) Science Symposium
September 20-22, 2022
Regulatory Education for Industry (REdI) Annual Conference 2022
June 6 - 10, 2022
Annual Patient Engagement & Regenerative Medicine Meeting 2022: An FDA CBER Workshop for Patient Advocates
May 24, 2022
RegenMedEd Webinar: The Critical Role of Patients in Advancing Gene Therapy Treatments for Rare Diseases
March 9, 2022
E20 Adaptive Designs for Clinical Trials
Safety Labeling Changes—Implementation of Section 505(o)(4) of the FD&C Act
Alternative Tools: Assessing Drug Manufacturing Facilities Identified in Pending Applications
E6(R3) Good Clinical Practice (GCP)
Q1 Stability Testing of Drug Substances and Drug Products
Conducting Remote Regulatory Assessments Questions and Answers
Accelerated Approval – Expedited Program for Serious Conditions
Communications From Firms to Health Care Providers Regarding Scientific Information on Unapproved Uses of Approved/Cleared Medical Products Questions and Answers: Final 01/06/2025
Recommendations for Determining Eligibility of Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); Draft Guidance for Industry
view this recorded webinar
Recommendations to Reduce the Risk of Transmission of Hepatitis B Virus (HBV) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); Draft Guidance for Industry
view this recorded webinar
Recommendations to Reduce the Risk of Transmission of Hepatitis C Virus (HCV) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); Draft Guidance for Industry
view this recorded webinar
Recommendations to Reduce the Risk of Transmission of Human Immunodeficiency Virus (HIV) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); Draft Guidance for Industry
view this recorded webinar
Advanced Manufacturing Technologies Designation Program: Final 12/31/24
E11A Pediatric Extrapolation: Final 12/30/24
Purpose and Content of Use-Related Risk Analyses for Drugs, Biological Products, and Combination Products July 2024
Platform Technology Designation Program for Drug Development May 2024
Read Kim Benton's article regarding this guidance here.
Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products; Draft Guidance for Industry
Read Amanda Mack and Alicja Fiedorowicz's publication regarding this guidance here.
Considerations for the Use of Human-and Animal-Derived Materials in the Manufacture of Cell and Gene Therapy and Tissue-Engineered Medical Products; Draft Guidance for Industry April 2024
Read Blake Bergam and Sara Mills' article on this subject here.
Providing Regulatory Submissions in Electronic Format: IND Safety Reports Guidance for Industry April 2024
Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products March 2024
Early Alzheimer’s Disease: Developing Drugs for Treatment March 2024
Q14 Analytical Procedure Development Q2 March 2024
(R2) Validation of Analytical Procedures March 2024
Advanced Manufacturing Technologies Designation Program February 2024
Human Gene Therapy Products Incorporating Human Genome Editing January 2024
Potency Assurance for Cellular and Gene Therapy Products; Draft Guidance for Industry December 2023
Master Protocols for Drug and Biological Product Development December 2023
Rare Diseases: Considerations for the Development of Drugs and Biological Products December 2023
Data Standards for Drug and Biological Product Submissions Containing Real-World Data December 2023
Translation of Good Laboratory Practice Study Reports: Questions and Answer November 2023
Submitting Clinical Trial Datasets and Documentation for Clinical Outcome Assessments Using Item Response Theory November 2023
Submitting Patient-Reported Outcome Data in Cancer Clinical Trials November 2023
Guidance for Industry: Voluntary Consensus Standards Recognitions Program for Regenerative Medicine Therapies October 2023
Guidance for Industry: Voluntary Consensus Standards Recognitions Program for Regenerative Medicine TherapiesData Standards for Drug and Biological Product Submissions Containing Real-World Data October 2023
Remote Interactive Evaluations of Drug Manufacturing and Bioresearch Monitoring Facilities October 2023
Expedited Programs for Regenerative Medicine Therapies for Serious Conditions; Draft Guidance for Industry Submit comments by November 24, 2025
Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations; Draft Guidance for Industry Submit comments by November 24, 2025
Postapproval Methods to Capture Safety and Efficacy Data for Cell and Gene Therapy Products; Draft Guidance for Industry Submit comments by December 24, 2025
Recommendations to Reduce the Risk of Transmission of Mycobacterium tuberculosis (Mtb) by Human Cells, Tissues, and Cellular and Tissue-Based Products (HCT/Ps); Guidance for Industry Final 01/06/2025.
Issued for immediate implementation but comments may be submitted.
Submit comments.
Considerations for Complying with 21 CFR 211.110
Draft 01/06/2025. Submit comments by 04/07/25
M15 General Principles for Model-Informed Drug Development:
Draft 12/30/24. Submit comments by 02/28/25
E6(R3) Good Clinical Practice: Annex 2:
Draft 12/30/24. Submit comments by 02/28/25
Protocol Deviations for Clinical Investigations of Drugs, Biological Products, and Devices:
Draft 12/30/24. Submit comments by 02/28/25
Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products; Draft Guidance for Industry:
Draft 11/19/24. Submit comments by 02/18/24
Electronic Systems, Electronic Records, and Electronic Signatures in Clinical Investigations: Questions and Answers:
Final 10/1/24. Allowing late comments.
Our first Regulatory Roundup LIVE was our Unbridled Excellence #4 and featured our unparalleled team of former FDA regulators discussing the latest and greatest regulatory news...that's DHC Master Principal Kimberly Benton and Master Practice Expert Don Fink, as well as Senior Principal Heath Coats, former Dark Horse Kevin Whittlese, and Senior Practice Expert Tal Salz. Go to the main Unbridled Excellence page to watch/download episode #4 on demand.