Around the Helix: Cell and Gene Therapy Company Updates – February 26, 2025

The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive®’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.

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1. Pfizer Jettisons FDA-Approved Hemophilia B Gene Therapy Beqvez

Pfizer has made the decision to discontinue development and commercialization of fidanacogene elaparvovec (Beqvez), an FDA-approved adeno-associated virus (AAV) vector-based gene therapy for the treatment of hemophilia B.

2. Sensorion Makes Progress in Trial for OTOF Hearing Loss Gene Therapy

Sensorion’s phase 1/2 AUDIOGENE clinical trial (NCT06370351), which is evaluating investigational AAV vector-based gene therapy SENS-501 (also known as OTOF-GT) for the treatment of otoferlin (OTOF) gene-mediated hearing loss, has received clearance from its data monitoring committee (DMC) to move onto dosing its second dose cohort.

3. FDA Accepts BLA for Precigen’s Recurrent Respiratory Papillomatosis Gene Therapy PRGN-2012 With Priority Review

The FDA has accepted the biologics license application (BLA) that Precigen submitted for PRGN-2012, a gene therapy product intended to treat recurrent respiratory papillomatosis with priority review. The Prescription Drug User Fee Act (PDUFA) action date for the BLA has been set at August 27, 2025, and no advisory committee meeting is currently planned for the BLA.

4. BioRestorative Therapies’ MSC Therapy BRTX-100 Fast Tracked for Chronic Lumbar Disc Disease

BioRestorative Therapies’ BRTX-100, an autologous mesenchymal stem cell (MSC) therapy, has received FDA fast track designation for the treatment of chronic lumbar disc disease.

5. bluebird bio to be Bought Out by Carlyle and SK Capital

blue bird bio has entered an agreement to be acquired by Carlyle and SK Capital, which will provide the company with capital to continue to commercially provide its gene therapy products, which include treatments for sickle cell disease and transfusion dependent thalassemia. Notably, uptake of nononcology gene therapy products in the field of hematology has generally been slow. "For more than a decade, bluebird has been at the forefront of gene therapy, delivering groundbreaking treatments to patients facing life-threatening genetic diseases,” Andrew Obenshain, MBA, the CEO of bluebird, said in a statement. “However, as our financial challenges mounted, it became clear that securing the right strategic partner was critical to maximizing value for our stockholders and ensuring the long-term future of our therapies."

6. Anixa Biosciences Wraps Up Dosing of Third Cohort in Ovarian Cancer CER-T Trial

Anixa Biosciences has dosed the final patient in the third cohort of its phase 1 clinical trial (NCT05316129) evaluating its novel follicle stimulating hormone receptor (FSHR)-targeting chimeric endocrine receptor (CER) T-cell therapy for the treatment of recurrent ovarian cancer. Patients in the third cohort were treated at a dose of 1 million CER T-cells, which is 10 times the dose received by the 3 patients who were treated in the study’s initial cohort (1x10⁵ CER T-cells).

7. Boehringer Ingelheim Launches Trial for Inhalable Cystic Fibrosis Gene Therapy

In conjunction with its partners UK Respiratory Gene Therapy Consortium and OXB, Boehringer Ingelheim has launched the phase 1/2 LENTICLAIR 1 clinical trial (NCT06515002), which is evaluating BI 3720931, an investigational inhaled lentiviral vector-based gene therapy product intended to treat cystic fibrosis. BI 3720931 is intended to provide the epithelial cells of the airway with functional copies of the CFTR gene.

8. Bionic Sight's Retinitis Pigmentosa Gene Therapy BS01 Snags FDA RMAT Designation

Bionic Sight's BS01, an investigational gene therapy product intended to treat retinitis pigmentosa in patients with advanced stage vision loss, has been granted regenerative medicine advanced therapy designation by the FDA. BS01 is currently being evaluated in a phase 1/2 clinical trial (NCT04278131), and the company is in the process of readying for phase 3 development.