All posts by PIER MARIA FORNASARI

PIER MARIA FORNASARI
2019-09-20

Suvodirsen Receives FDA’s Fast Track Status to Treat Duchenne Muscular Dystrophy

The U.S. Food and Drug Administration (FDA) has granted fast track designation to suvodirsen as a treatment for people with Duchenne muscular dystrophy (DMD). The agency’s decision — which will expedite the review process for suvodirsen — was based on experimental and preclinical data demonstrating the treatment’s potential therapeutic activity. Suvodirsen already had been granted orphan drug designation for the treatment of DMD

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PIER MARIA FORNASARI
2019-09-20

FDA Grants Orphan Drug Designation to Reneo Pharmaceuticals for REN001 for Treatment of Fatty Acid Oxidation Disorders

 Reneo Pharmaceuticals, a clinical stage pharmaceutical company, announced today that the U.S. Food and Drug Administration  (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to the company’s lead drug candidate, REN001, for the treatment of fatty acid oxidation disorders (FAOD). “There is a pressing need for effective treatments for patients suffering from

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PIER MARIA FORNASARI
2019-09-20

Medical Board of California meeting on stem cell clinics emphasized the rigorous stem cell-related clinic trials ongoing and in development and the risks coming from deviating from standard clinical trial and GMP practices.

Source The Niche Knoepfler Lab Stem Cell Blog This afternoon Dr. Knoepfler attended and gave public comment at the Medical Board of California stem cell task force meeting on unproven stem cell clinics. There were about 40 people attending. In part this meeting is a consequence of the national governing organization of state medical boards, the Federation of

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PIER MARIA FORNASARI
2019-09-20

Future and Emerging Technologies Flagships and Large Research Initiatives

European Commission, Digital Single Market has published a new Factsheet/Infographic on FET Flagships and Large Research Initiative. These visionary, long-term, large-scale research initiatives address major science and technology challenges in Europe. They involve hundreds of research teams with the aim of boosting the EU’s scientific and industrial landscape.

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PIER MARIA FORNASARI
2019-09-20

Cancer-Related Mutations Identified in Primed and Naive Human Pluripotent Stem Cells

Source Cell Stem Cell Human pluripotent stem cells (hPSCs) are known to harbor chromosomal aberrations, affecting their tumorigenic potential. We established a strategy to identify cancer-related point mutations in hPSCs, detecting recurrent mutations in over 20 genes, alongside those previously detected in p53. Importantly, naive hPSCs harbor, on average, four times more mutations than their

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PIER MARIA FORNASARI
2019-09-20

AveXis presents new data at EPNS continuing to show significant therapeutic benefit of Zolgensma® in prolonging event-free survival now up to 5 years of age in patients with spinal muscular atrophy (SMA) Type 1

New interim data from SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age‑appropriate major milestone gain Updated results from global STR1VE study demonstrate that Zolgensma® (onasemnogene abeparvovec-xioi) has significant therapeutic benefit in prolonging event-free survival in SMA Type 1 patients versus natural history Patients in START long-term follow-up study (cohort 2), who

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PIER MARIA FORNASARI
2019-09-19

Boston University researchers create new protocol to improve gene therapy tool production

Method is more efficient and economical compared with other procedures A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine (BUSM) researchers. Gene therapy is a clinical technique that introduce genes to treat disease. One approach is to use adeno-associated virus

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PIER MARIA FORNASARI
2019-09-19

Multiple myeloma treatment granted FDA Orphan Drug Designation

Orphan Drug Designation has been given to bispecific antibody candidate, GBR 1342, for the treatment of multiple myeloma. The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Glenmark Pharmaceuticals’ bispecific antibody candidate GBR 1342 for the treatment of patients with multiple myeloma who have received prior therapies. “Patients who are struggling with diseases like

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