Cell therapy weekly: New collaboration will target AAV production overhaul

Written by RegMedNet

This week in cell therapy: Graft failure therapy receives Orphan Drug Designation, Advanced Therapies Network will support London advanced therapies development and spray-on skin receives FDA approval.

The news highlights:

Cobra Biologics, GE Healthcare and Centre for Process Innovation to collaborate on AAV manufacturing overhaul
Graft failure therapy receives Orphan Drug Designation
Advanced Therapies Network will support London advanced therapies development
Spray-on skin receives FDA approval

Cobra Biologics, GE Healthcare and Centre for Process Innovation to collaborate on AAV manufacturing overhaul

Cobra Biologics, the Centre for Process Innovation (both UK) and GE Healthcare Life Sciences (IL, USA) are to collaborate to address the challenges of adeno-associated virus (AAV) vector production, making the process cheaper and more robust. AAV vectors are a crucial part in gene therapy development and administration but have proved to be a significant bottleneck in commercialization.

Daniel Smith, Chief Scientific Officer at Cobra Biologics, said: “The scalable chromatographic purification of recombinant AAV-based viral vectors for use in gene therapy applications remains an area of intense global development, essential to support the rapidly increasing market opportunity for these innovative medicines…the application and implementation of the fibre-based chromatography for the purification of AAV vectors could provide a step change in the technology available, allowing for the scalable, cost effective production of this emerging class of innovative medicines.”

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Graft failure therapy receives Orphan Drug Designation

A novel placenta-based therapeutic for the treatment of graft failure and incomplete hematopoietic recovery, developed by Pluristem Therapeutics Inc. (Haifa, Israel) has been granted Orphan Drug Designation by the FDA. PLX-R18 is currently undergoing evaluation for safety in the treatment of f insufficient hematopoietic recovery following bone marrow transplantation in an ongoing Phase 1 clinical trial in the USA and Israel.

“We believe the FDA’s decision to grant Orphan Drug Designation to PLX cell therapy for the treatment of graft failure and incomplete hematopoietic recovery reflects the potential of this regenerative therapy to improve outcomes in this underserved patient population,” said Yaky Yanay, Co-CEO and President of Pluristem. “We look forward to data from our ongoing Phase 1 clinical trial as we work to efficiently advance this novel therapy through clinical development for this and other hematopoietic indications.”

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Advanced Therapies Network will support London advanced therapies development

A new London-based network will facilitate connections, collaborations and exchange of knowledge between academia and industry, building on the work of the London Regenerative Medicine Network, run by Regenerative Medicine senior editor and industry leader Professor Chris Mason. The Advanced Therapies Network is launching on 8 November with an event at the Royal College of Physicians and will feature speakers such as Adrien Lemoine (Orchard Therapeutics) and Farzin Farzaneh (King’s College London; all London, UK).

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Spray-on skin receives FDA approval

The RECELL® System, developed by AVITA Medical (VIC, Australia), has been approved by the FDA for the treatment of second- and third-degree burns. The RECELL System utilizes autologous tissue at lower amounts than conventional grafts and is ready to apply to patients in as little as 30 minutes. The approval was based on the results of two randomized, controls clinical trials treating second- and third-degree burns respectively.

“Today’s approval of the RECELL System is a significant advancement in how we treat patients with burns,” commented James H Holmes, Wake Forest Baptist Medical Center (NC, USA). “Dramatically reducing the amount of donor skin needed to treat second- and third-degree burns has important implications for pain, scarring and costs of care, while still providing comparable healing to the current standard of care. Additionally, the potential reduction in mortality is extremely promising.”

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For more weekly cell therapy news, read previous editions of the cell therapy weekly.