Cell therapy weekly: Gastrointestinal bleeding therapy awarded Orphan Drug designation

This week: new technology for recording cellular communication may impact on cell therapies and RMAT designation for epidermolysis bullosa therapy

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Jun 27, 2019
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The news highlights:

Gastrointestinal bleeding therapy awarded Orphan Drug designation
How cells communicate; new possibilities for cell therapies
Krystal Biotech receives FDA RMAT designation for severe epidermolysis bullosa treatment

Gastrointestinal bleeding cell therapy awarded Orphan Drug designation

Mesoblast (Melbourne, Australia) has been granted FDA Orphan Drug designation for Revascor (rexlemestrocel-L), their preventative therapy for post-inflammatory mucosal bleeding. The treatment is intended for patients with end-stage, chronic heart failure requiring left ventricular assist device (LVAD) surgery. Revascor is an investigational cell therapy comprised of 150 million allogeneic mesenchymal precursor cells (MPCs), being developed for injection into the cardiac myocytes of patients with severe chronic heart failure. FDA Orphan Drug designation qualifies Mesoblast’s product for various benefits that should assist in the drug development process.

Silviu Itescu, Chief Executive of Mesoblast, commented: “We are very pleased that the FDA has granted our heart failure cell therapy product candidate, Revascor, Orphan Drug designation…We look forward to our upcoming meeting with the FDA to discuss a potential approval pathway under the product’s existing Regenerative Medicine Advanced Therapy (RMAT) designation for this life-threatening condition”.

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How cells communicate; new possibilities for cell therapies

New technology, developed at the University of Connecticut, School of Dental Medicine (CT, USA), can record how cells communicate in real time. The technology was employed to record changes in protein secretions from bone marrow-derived stem cells — used to treat myocardial infarctions — over time. Subsequently, researchers were able to use this information to concoct a ‘protein cocktail’ that could repair the heart tissue, without using stem cells. This ‘cell-less’ therapy could reduce complications associated with conventional stem cell transplantations in the future.

Yasir Suhail, a postdoctoral fellow in the biomedical engineering department of the University of Connecticut, School of Dental Medicine, commented: “The findings solve a fundamental problem afflicting systems biology: measuring how cells communicate with each other…The platform technology will open new lines of inquiry into research, by providing a unique way to detect how cells talk to each other at a deeper level than what is possible today.”

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Krystal Biotech receives FDA RMAT designation for severe epidermolysis bullosa treatment

Following positive phase II clinical trial results, the FDA has granted Krystal Biotech (PA, USA) Regenerative Medicine Advanced Therapy (RMAT) designation for KB103, their lead candidate therapy, for patients with severe, generalized recessive dystrophic epidermolysis bullosa (RDEB). KB103 is a replication-defective, non-integrating viral vector that delivers functional COL7A1 human genes directly to patients’ skin cells. RMAT designation confers all benefits of the FDA's Fast Track and Breakthrough Therapy designations and may allow KB103 to be eligible for accelerated approval and earlier delivery to patients.

Peter Marinkovich, Associate Professor of Dermatology at Stanford University (CA, USA) and principal clinical trial investigator, commented: “New treatments for patients suffering from epidermolysis bullosa are desperately needed, especially one that provides a convenient, painless way to administer treatment for patients suffering with this debilitating disease and to reduce their travel burden…These exciting data in the Phase I/II trials are supportive of this very promising new approach for treating this debilitating disease.”

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

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