FDA Encourages Development of Regenerative Medicines

The agency clarified the process for development programs for regenerative medicine therapies. Feb 20, 2019 By Pier Maria Fornasari FDA provided further details in February 2019 on how manufacturers may use expedited development programs for regenerative medicine therapies, clarifying that the category includes gene and Chimeric antigen receptor T cells (CAR-T) therapies, as well as cellular

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Institute for Clinical and Economic Review’s Assessment Finds Spinraza and Zolgensma Provide Substantial Health Benefits for People with Spinal Muscular Atrophy

Current pricing of Spinraza would require a substantial discount to meet traditional cost-effectiveness ranges; Zolgensma’s value-based price range is between $310,000-$900,000 using standard methodology but as high as $1.5 million using alternative measures of health gain —— As with all treatments for ultra-rare conditions, judgments of overall value require consideration of contextual issues and broader

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How gut bacteria controls gene expression through “interspecies communication”

Highlights •Microbiome-derived NO promotes widespread S-nitrosylation of the host proteome •Interspecies S-nitrosylation regulates miRNAs, gene expression and host development •Microbiota control host function by shaping the post-translational landscape Summary Bioactive molecules can pass between microbiota and host to influence host cellular functions. However, general principles of interspecies communication have not been discovered. We show here

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RESTORE encourages your contribution to promote the project. Support RESTORE consortium

Advanced Therapies (Advanced Therapies Medicinal Products and Biologised Medical Devices – ATMP/bio-MD) are a potential game changer in health care: They aim to transform the current focus on “treatment of disease” into one that concentrates on “regeneration of health”. The unifying goal of RESTORE is the implementation of newly developed Advanced Therapies in clinical routine

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‘Off-the-shelf’ CAR-Ts have an important role to play – but are they ready for prime time?

Allogeneic CAR-Ts could serve an important niche – particularly for patients who can’t receive autologous CAR-Ts – but the earliness of the data makes conclusions tough to draw. Since their transition from small, academic clinical trials to commercial production, CAR-T cell therapies have won recognition as a novel and highly effective therapeutic modality, particularly in

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Microsoft, Takeda, Eurordis take on rare diseases in children with report, pilots

The three companies announced a slew of pilots employing machine learning, collaborative digital tools and blockchain technology. A year after Microsoft, Takeda and Eurordis announced that they were teaming up on an innovation initiative to solve the issue of diagnosing children with rare diseases, the partners have unveiled an actionable recommendation report and three new

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Procter & Gamble Partners with Aether to Advance AI-Driven 3D Bioprinting

The 3D printing and AI startup Aether has reached a two-year joint agreement with Procter & Gamble to further develop its AI-driven 3D bioprinting system into an “intelligent robotic craftsman”. Procter & Gamble, the multinational consumer goods giant, has agreed to a two-year developmental agreement with the San Francisco-based 3D printing and AI startup Aether.

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AI applications in healthcare grow, opening channel prospects

The hype around AI applications in healthcare spans decades, but the latest wave of AI tech suggests real-world traction. Artificial intelligence is already promising big things at Massachusetts General Hospital and Brigham and Women’s Hospital in Boston, where researchers have been building machine learning models over the past couple of years. Those models now help

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