From The Editor | June 29, 2023

What A Week In CGT: Approvals And Advancements


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1

C&G News

The cell and gene therapy sector has had quite a week. From FDA approvals to groundbreaking advancements, the cell and gene therapy space has had good news to share. Here’s what’s gone on in just the last week alone.

Bayer Pharmaceuticals

Bayer Pharmaceuticals’ experimental stem cell treatment for Parkinson’s disease suggests we may be one step closer to finding a successful treatment for the progressive disease. Parkinson’s affects over 10 million people worldwide. The cell therapy, bemdaneprocel, is in development at Bayer’s BlueRock Therapeutics subsidiary. Bayer said that one year into a Phase I trial with 12 volunteers, BlueRock’s therapy was shown to be well-tolerated and that transplanted cells grew as intended in patients’ brains. BlueRock has become the first company to report initial success treating Parkinson’s disease in humans using an experimental stem cell therapy. A phase 2 clinical trial on course to open enrollment next year.

Speaking of BlueRock Therapeutics, join me on Tuesday, July 11th at 11 am EST when our expert panelists, Craig Beasley, Chief Technical Officer at BlueRock Therapeutics and Narinder Singh, Chief Technical Officer at Arcellx present how to improve cost and scalability in cell therapy manufacturing via standardization and automation. Registration is free.

Sarepta Therapeutics

Back in March, I wrote an article based on my conversation with Michael G. Kelly, Ph.D., Chief Scientific Officer at CureDuchenne to learn more about the organization and its partnership with Sarepta Therapeutics. Since the publication of that article, the FDA has granted Accelerated Approval of SRP-9001, an important milestone that keeps us on a path toward identifying truly transformative treatments for those living with Duchenne muscular dystrophy (DMD). Upon the approval of SRP-9001, Debra Miller, CEO of CureDuchenne, shared with me:

We are pleased that the FDA has granted Accelerated Approval of SRP-9001, as it is an important milestone that keeps us on a path toward identifying truly transformative treatments for those living with Duchenne muscular dystrophy.  

  While the FDA has granted this approval for patients aged 4-5 years old, we recognize that many in the community will be disappointed due to the narrow age range specified. We look forward to the full data readout from the EMBARK trial and expect this confirmatory study will generate important data to further inform the best clinical use of this therapy. We are hopeful it will demonstrate efficacy and safety to support an expanded age range, and eventually expand to include non-ambulatory individuals.   

   While this is a significant milestone, our work is far from done. There are still many individuals waiting for effective treatments, and CureDuchenne will continue to support and invest in a diverse array of promising research to bring solutions to everyone living with Duchenne, including many technologies that could potentially overcome the limitations of first-generation gene therapies.”

If someone you love has DMD, this groundbreaking approval offers something you may not have had before – hope.

CellTrans’ Lantirda

CellTrans, Inc.’s Lantidra is the first FDA-approved allogeneic pancreatic islet cellular therapy made from deceased donor pancreatic cells for the treatment of type 1 diabetes (T1D). This approval is a significant milestone in cell therapy and diabetes treatment, offering a promising future for patients. Lantidra is approved for the treatment of adults with type 1 diabetes who are unable to approach target glycated hemoglobin (average blood glucose levels) because of current repeated episodes of severe hypoglycemia (low blood sugar) despite intensive diabetes management and education. Lantidra is designed to assist adults who struggle to maintain target glycated hemoglobin due to frequent severe hypoglycemia even with intensive diabetes management. The treatment is administered as a single infusion into the hepatic portal vein, with additional doses possible depending on patient response. Clinical trials with 30 participants resulted in 21 patients not needing insulin for a year or more, signifying Lantidra’s potential efficacy.

Note that there are other companies making significant strides in T1D. Sernova Corp., for example, is a clinical-stage regenerative therapies company currently focused on insulin-dependent diabetes. Last year, I invited Sernova Corp.’s CEO and President, Philip Toleikis, Ph.D., to Cell & Gene: The Podcast to discuss company's on-going clinical trial to treat T1D as well as the very real potential to progress from simply masking symptoms to having a functional cure. Toleikis also provided his take on the near-term future of regenerative medicine.