Cell therapy weekly: Potential second pregnancy in CRISPR storm

This week in cell therapy: Regulatory pathway in China mapped out for MPC-150-IM and £1.5 million grant will investigate AAV continuous manufacturing.

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Nov 29, 2018
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The news highlights:

Potential second pregnancy in CRISPR storm
Regulatory pathway in China mapped out for MPC-150-IM
£1.5 million grant will investigate AAV continuous manufacturing

Potential second pregnancy in CRISPR storm

He Jiankui, who announced earlier this week that he had used CRISPR-Cas9 to alter the genes of twin girls, giving them HIV resistance, revealed that there may be a second pregnancy. Speaking at the Human Genome Editing Summit (27–29 November, Hong Kong), He defended his work, commenting that all the participants had a “good education background” and were fully informed about the risks of the study.

However, preliminary investigation at the hospital named in the clinical trial registry, Shenzhen Harmonicare Medical Holdings Limited (Shenzhen, China), has indicated that signatures associated with the medical ethics application form are “suspected to have been forged, and no relevant meeting of the Medical Ethics Committee of the hospital in fact took place”.

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Regulatory pathway in China mapped out for MPC-150-IM

Following mixed results from the Phase II trial of MPC-150-IM for heart failure, Tasly Pharmaceutical Group (NY, USA) and Mesoblast Limited (Melbourne, Australia) have announced completion of the first Joint Steering Committee (JSC) meeting and that they will be meeting the National Medical Products Administration (NMPA) of China, formerly known as the China Food and Drug Administration, in early 2019.

Tasly Biopharma’s Chief Medical Officer and Chair of the JSC, Gloria Wang, said: “We are going to leverage Mesoblast’s extensive cardiovascular clinical experience and knowledge base in order to map out the most expeditious pathway to approval for the heart failure product in China.”

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£1.5 million grant will investigate AAV continuous manufacturing

Cobra Biologics (Keele, UK), Pall Corporation (NY, USA) and Cell and Gene Therapy Catapult (London, UK) have been jointly awarded a £1.5 million grant from Innovate UK to investigate continuous manufacturing of adeno-associated virus (AAV). AAV are a powerful tool in creating gene therapies but their production is often cited as a bottleneck in the widespread delivery of these treatments.

“Together we will develop innovative in-process analytical techniques and manufacturing approaches based on continuous chromatography platforms, to significantly increase process yields, and, in doing so, make these advanced therapies more accessible to patients,” commented Peter Coleman, Chief Executive Officer of Cobra Biologics.

“Yields for downstream processing of AAV are currently very low and the production process is costly in both time and consumables,” explained Mario Philips, Vice President & General Manager of Pall Biotech. “With this project, we hope to advance the AAV purification process and affect a 25% or more step change in purification yields.”

“As we move forward, we will create a scalable continuous process that increases efficiencies in time and cost, to make commercialization of gene therapies safer, faster, and cheaper than ever before,” added Keith Thompson, Chief Executive Officer of the Cell and Gene Therapy Catapult. “We are excited to play a key role in increasing patient access to these potentially life-changing therapies and to further establish best practices in manufacturing.”

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

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