Cell therapy weekly: court rules against FDA in unproven stem cell case

Written by RegMedNet

Also this week: FDA grant Orchard Therapeutics’s autologous therapy orphan drug designation, Phase II regenerative cartilage therapy awarded US$1.1 million, custom CAR-T service announced by AMSBIO and new cell and gene therapy plant in New Jersey

The news highlights:

FDA grant Orchard Therapeutics’s autologous therapy, OTL-102, orphan drug designation
FDA bid to close unproven stem cell clinics stopped by Los Angeles Judge
Phase II regenerative cartilage therapy awarded US$1.1 million by SBRI Healthcare
Custom CAR-T service announced by AMSBIO
Hitachi Chemical open new facility for commercial cell and gene therapy manufacturing

FDA grant Orchard Therapeutics’s autologous therapy, OTL-102, orphan drug designation

The US FDA (MD, USA) has granted orphan drug status to Orchard Therapeutics’s (London, UK) OTL-102 treatment for X-linked granulomatous disease (X-CGD). OTL-102 involves utilizing gene therapy and autologous ex vivo hematopoietic stem cells with the aim to correct the immunodeficiency seen in patients. By obtaining the orphan drug status the company can now receive financial incentives and clinical support whilst working on treating the rare condition.

“We are pleased to have received this orphan drug designation from the FDA, which recognizes the potential of OTL-102 to address a rare population of patients with X-CGD, a life-threatening disease with a critical unmet need,” commented Anne Dupraz-Poiseau (Orchard Therapeutics). “We are encouraged by the clinical data published to date and are eager to advance OTL-102 development as quickly as possible for patients with X-CGD.”

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FDA bid to close unproven stem cell clinics stopped by Los Angeles Judge 

A Los Angeles (CA, USA) judge has ruled against the US FDA (MD, USA), denying a summary judgement to immediately shut down the California Stem Cell Treatment Center and the Cell Surgical Network (both CA, USA). The FDA has attempted to tackle unproven stem cell treatments in recent years, with moderate success previously in Miami; however, the recent ruling will be a blow to their campaign. Whilst the case is still pending further litigation, the Los Angeles based operation can continue their work.

“Cell-base regenerative medicine holds significant medical opportunity,” explained Scott Gottlieb, the FDA Commissioner at the time the lawsuits were filed. “But we’ve also seen some bad actors leverage the scientific promise of this field to peddle unapproved treatments that put patients’ health at risk.”

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Phase II regenerative cartilage therapy awarded US$1.1 million by SBRI Healthcare

SBRI Healthcare (UK) has awarded US$1.1 million to Mogrify (Cambridge, UK) to help develop their Phase II regenerative cartilage therapy. Mogrify specialize in ‘big data’ cell reprogramming, gleaming essential transcription factors from next-generation sequencing with the goal of producing scalable levels of chondrocytes in vitro. They also hope to eventually enable in vivo reprogramming in conditions such as osteoarthritis.

“We select projects strategically based on both commercial and scientific considerations and are delighted with the progress of this collaboration with Dr. Wael Kafienah’s laboratory at the University of Bristol (UK),” explained Karin Schmitt (Mogrify). “The continued support for our lead musculoskeletal asset from SBRI Healthcare has not only allowed us to reach this phase but will enable us to carry the chondrocyte conversions through to the next stage.”

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Custom CAR-T service announced by AMSBIO

AMSBIO (Milton, UK) have announced their new service designed to enable the design and execution of CAR-T therapy, regardless of clinical stage. Building upon its expertise with monoclonal anitbodies, the company offer to provide aide whether targeting a specific molecule or antibody.

Utilizing a single chain variable fragment, and a lentiviral vector of the consumers choice, AMSBIO will offer to help transduce both human or mouse T-cells, as well as measure the cytotoxicity in real time with their assay, hopefully producing a more efficient pipeline for the development of exciting cell therapies.

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Hitachi Chemical open new facility for commercial cell and gene therapy manufacturing

Hitachi Chemical (Tokyo, Japan) have announced the opening of a new cell and gene therapy manufacturing facility that will provide 500 new jobs in New Jersey (USA). The US arm of the chemical giant, dubbed Hitachi Chemical Advanced Therapeutics Solutions (NJ, USA), will be producing their first ‘ground-up’ facility to meet the unique needs of the commercial cell and gene therapy market, doubling their current manufacturing ability. With new state-of-the-art quality control, microbiological laboratories, warehouses and meeting spaces, the company hope to support their clients in their commercialization of the latest therapies.

“The opening of this new facility marks an important milestone for HCATS and will offer a state-of-the-art resource for our clients as they commercialize cell and gene therapies. Access to this type of manufacturing space is needed across the industry to ensure the continued growth and momentum of these promising therapeutics,” explained Robert Preti, President and CEO of Hitachi Chemical Advanced Therapeutics Solutions. “This facility will require up to 500 more employees to reach full operational capacity over the next several years, supporting our growing roster of clients.”

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

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