Cell therapy weekly: Experimental hemophilia gene therapy to progress despite adverse immune response in two patients

This week: Regeneron and bluebird join forces to develop immune-oncology therapeutics and UK trial will target Crohn’s disease

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Aug 09, 2018
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The news highlights:

Safety concerns over hemophilia A gene therapy causes share price drop
Regeneron and bluebird join forces to develop cell therapies for cancer
£2 million UK stem cell trial will target Crohn’s disease
BioLife Solutions receives US$20 million investment to support strategic growth

Safety concerns over hemophilia A gene therapy causes share price drop

Shares in Spark Therapeutics (PA, USA) dropped up to 32% following an announcement in their second quarter 2018 financial results that two patients in a Phase I/II trial of its experimental gene therapy SPK-8011 for hemophilia A had a concerning immune response. The two patients were in the cohort receiving a dose of 2x1012 vg/kg and experienced a drop in factor VIII, an essential blood-clotting protein, activity levels to less than 5%, with one patient requiring hospitalization. However, across all 12 participants in the trial, at all three doses, beginning four weeks after vector infusion, there has been a 97% reduction in annualized bleeding rate and a 97% reduction in annualized infusion rate.

“These early data further support the dramatic impact on patient outcomes that can result from factor activity levels above 12% and bring us closer to our goal of one day eliminating spontaneous bleeding altogether, while potentially freeing patients with hemophilia A from the need for regular infusions.” Katherine A. High, president and head of research and development at Spark Therapeutics.

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Regeneron and bluebird join forces to develop cell therapies for cancer

Regeneron Pharmaceuticals, Inc. (NY, USA) and bluebird bio, Inc. (MA, USA) will collaborate in the development and commercialize novel immune-oncology cell therapies. In a deal worth over US$100 million, the cost of research and development of six targets up to the point of IND submission will be equally shared. bluebird bio's proprietary technologies use a customized lentiviral vector to modify T cells so that they can recognize tumor-specific proteins expressed by cancer cells and kill them upon engagement. Regeneron's VelociSuite® technologies, including VelocImmune® and Veloci-T, enable the creation of fully-human antibodies and T cell receptors.

"Like Regeneron, bluebird is a science-focused company looking to push the limits of what novel technologies can do in drug discovery and development," said George D. Yancopoulos, President and Chief Scientific Officer of Regeneron.

"With Regeneron's proven targeting technologies, in combination with our deep expertise in cell biology and vector technology, as well as clinical experience with leading CAR T cell drug products, we hope to rapidly advance novel cellular therapies with the potential to transform the lives of people with cancer." said Philip Gregory, Chief Scientific Officer of bluebird bio.

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£2 million UK stem cell trial will target Crohn’s disease

A study led by Queen Mary University of London and Barts Health NHS Trust (London, UK) is evaluating the safety and effectiveness of stem cells in the treatment of Crohn's disease. The patients’ immune systems will be wiped out and replaced using stem cell transplants in a bid to reduce gut inflammation. ASTIClite builds on the previous ASTIC trial, which benefited many participants but had a high incidence of adverse events.

Helen Bartlett, a Crohn’s disease patient who had stem cell therapy at John Radcliffe Hospital (Oxford, UK) said: “Living with Crohn’s is a daily struggle. I’ve been in and out of hospital for the last twenty years, operation after operation, drug after drug, to try to beat this disease. It’s frustrating, it’s depressing and you just feel so low. I cannot describe how much better I feel since the treatment. I still have problems and I’m always going to have problems, but I’m not in that constant pain.”

Professor Tom Walley, Director of the NIHR Evaluation, Trials and Studies programs, which funded the trial, said: “Stem cell therapies are an important, active and growing area of research with great potential. There are early findings showing a role for stem cells in replacing damaged tissue.  In Crohn’s disease this approach could offer real benefits for the clinical care and long term health of patients.”

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BioLife Solutions receives US$20 million investment to support strategic growth

BioLife Solutions, Inc. (WA, USA) has received US$20 million of investment from Casdin Capital LLC (NY, USA) to support their growth strategy of acquiring relevant and complementary services and technology for cell and gene therapy manufacturing.

Eli Casdin, Managing Director of Casdin Capital, remarked, "As active investors in cellular therapy companies and across the life science continuum, we recognize that the supplier side of the industry is critical but highly fragmented. There exists now a tremendous investment opportunity to consolidate and integrate these businesses, creating a broad and trusted platform partner for therapeutic developers."

Mike Rice, BioLife President & CEO commented, " This….allows us the financial flexibility to initiate our strategy of acquiring complementary revenue-generating companies or technologies that will enable BioLife to increase our share of the expenditures being made for cell and gene therapy manufacturing tools and services."

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

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