Category REGENERATIVE MEDICINE

The Alliance for Regenerative Medicine Publishes First-Ever Rare Disease Report, Detailing Financial & Clinical Progress of 323 Rare-Disease Focused Cell & Gene Therapy Developers Worldwide

01 March 2019 P.M.Fornasari Rare diseases affect fewer than roughly five in 10,000 people worldwide. Approximately 80% of rare diseases have identified genetic origins, and are often debilitating; about 30% of children with a rare disease will die before their fifth birthday. There are roughly 30 million people in the U.S. living with a rare

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Reprogrammed’ stem cells to treat spinal-cord injuries for the first time

Approval from Japanese regulators means that trials of induced pluripotent stem cells can begin later this year. Scientists in Japan now have permission to inject ‘reprogrammed’ stem cells into people with spinal-cord injuries. An upcoming trial will mark the first time that induced pluripotent stem (iPS) cells have been used to treat spinal-cord injuries, after

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Chronic Low Back Pain Study Results Indicate Benefit With Tanezumab

Februray 25, 2019 P.M. Fornasari Tanezumab, an investigational subcutaneously-administered, non-opioid treatment, may benefit patients with moderate to severe chronic low back pain (CLBP), according to top-line results from a Phase 3 study. Study A4091059, a double-blind, placebo- and active-controlled study, included patients with moderate to severe CLBP who had persistent low back pain for more

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ASIPP Releases Guidelines for Use of Biologics in Management of Low Back Pain

February 25, 2019 P.M. Fornasari A task force of regenerative medicine experts established by the American Society of Interventional Pain Physicians (ASIPP) has formulated new guidelines for the safe and effective use of biologics in the management of low back pain. The guidelines, published in Pain Physician, are based on a review of the literature on the

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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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‘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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