Cell therapy weekly: FDA approves epidermolysis bullosa gene therapy
This week: The US Food and Drug Administration (FDA; MD, USA) approved the first cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB), QIAGEN (Hilden, Germany) expanded its digital PCR (dPCR) portfolio to include solutions for lentivirus-based applications, and the California Institute for Regenerative Medicine (CIRM; USA) awarded US$8 million to support the development of a gene therapy for retinitis pigmentosa.
The news highlights:
- First cell-based gene therapy for RDEB approved by FDA
- New lentivirus dPCR solutions to strengthen cell and gene therapy quality control
- Strategic partnership to manufacture iPSCs for cardiovascular disease
First cell-based gene therapy for RDEB approved by FDA
The FDA has approved Abeona Therapeutics’ (OH, USA) ZEVASKYN, the first autologous cell-based gene therapy for treating wounds in patients with RDEB, a severe genetic skin condition characterized by extremely fragile skin that blisters and tears easily.
RDEB results from mutations in both copies of the COL7A1 gene, which encodes Type VII collagen. ZEVASKYN, is a sheet of autologous keratinocytes that have been genetically modified to produce Type VII collagen. The sheets are surgically applied to wound sites, enabling proper wound healing.
“Today’s approval of ZEVASKYN represents a pivotal moment in the treatment of RDEB, answering the call of people living with the clinical, economic and human impact of this devastating disease,” expressed Vish Seshadri, CEO of Abeona. “We have heard from the RDEB community that there is a persistent unmet need to reliably address RDEB wounds, especially those that are chronic and prone to infection. Through a single surgical application, ZEVASKYN can now offer people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds, as evidenced by the results from our pivotal Phase III study.”
New lentivirus dPCR solutions to strengthen cell and gene therapy quality control
QIAGEN has expanded its dPCR portfolio to include solutions for analyzing and measuring lentiviral vectors in cell and gene therapy applications, including kits for safety testing, viral quantification and streamlined quality control processes.
Justus Krause-Harder, Vice President and Head of Molecular Tools & Oncology at QIAGEN, explained: “Cell and gene therapy developers face increasing demands for precise, scalable solutions to help ensure therapy safety and efficacy. With our expanded dPCR portfolio, we are not only meeting the needs of lentivirus-based therapies but also reaffirming our long-term commitment as a partner for quality control in [cell and gene therapy] development and manufacturing.”
The new lentiviral solutions include:
- The QIAcuity RCL Quant Kit for detection of replication-competent lentivirus
- The QIAcuity CGT dPCR assays for viral vector genome titration and vector copy number determination
- The CGT Lentivirus Lysis Kit and protocol for vector genome titer quantification with fewer manual steps
US$8 million grant for retinitis pigmentosa gene therapy
The CIRM has awarded a US$8 million grant to Ray Therapeutics (CA, USA) to support the clinical development of RTx-015, an optogenetic gene therapy for retinitis pigmentosa.
“Retinitis pigmentosa remains a devastating condition with no approved treatments for the vast majority of patients,” said Paul Bresge, CEO and Co-Founder of Ray Therapeutics. “We are deeply grateful to CIRM for their belief in our science and their continued support of our programs. We are honored to partner with CIRM as we advance therapies that have the potential to transform the lives of patients.”
RTx-015 utilizes an AAV.7m8 viral vector to introduce a gene encoding a highly light-sensitive protein to retinal ganglion cells, aiming to restore visual function.