Spark Therapeutics $ONCE has scored an historic FDA approval of Luxturna, the world’s first such AAV-delivered gene therapy designed to cure a rare eye disease triggered by a genetic mutation. The drug is OK’d for RPE65 mutation linked retinal dystrophy. The treatment uses a viral vector to insert the correct copy of a gene retinal cells need to create a protein that turns light into electric signals which can restore vision lost to the disease. As with the earlier pioneering approval of the world’s first CAR-T, FDA commissioner Scott Gottlieb did the honors in recognizing the importance of this approval. And he says the agency will make sure that the regulatory path is straight and clear for the rest of the field looking to following Spark’s footsteps. “We’re at a turning point when it comes to this novel form of therapy and at the FDA, we’re focused on establishing ...
“The sale of the PRV provides us with an important source of non-dilutive capital to help advance our pipeline of rare and ultra-rare therapies, and accelerates the availability of these potential therapies to patients,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx.
Curisium, a new healthcare technology that builds blockchain-based contracting technology for payers, providers, and life sciences companies, officially launche with the announcement that it has secured $3.5 million in seed funding.
A flexible and transparent power source inspired by the electric eel could be used to power electrical devices in the body, such as cardiac pacemakers, implantable sensors or even prosthetic organs. The prototype, described in Nature1 on 13 December, runs on a solution of salt and water, but researchers hope that future versions might get their energy from bodily fluids.
AstraZeneca today announced that the US Food and Drug Administration (FDA) has accepted a supplemental New Drug Application (sNDA) for the use of TAGRISSO® (osimertinib), a third-generation, irreversible epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) with clinical activity against central nervous system (CNS) metastases, in the 1st-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have EGFR mutations (exon 19 deletions or exon 21 (L858R) substitution mutations). The FDA has granted TAGRISSO Priority Review status, and previously granted Breakthrough Therapy Designation for TAGRISSO in the 1st-line treatment of patients with metastatic EGFR mutation-positive (EGFRm) NSCLC.
Teva Pharmaceutical Industries Ltd., (NYSE and TASE: TEVA) today announced the U.S. Food and Drug Administration (FDA) has accepted for review the company’s Biologics License Application (BLA) for fremanezumab, an anti-calcitonin gene-related peptide (anti-CGRP) monoclonal antibody for the preventive treatment of migraine. Additionally, the FDA has granted fast track designation for fremanezumab for the prevention of cluster headache.
Boston biotech resTORbio has pocketed another $40 million from a second-round financing that it hopes gives it enough cash to move its lead candidate for respiratory tract infections in elderly patients into phase 3.
There is no proven intervention for preventing late-life dementia. Researchers from the Minnesota Evidence-based Practice Center (EPC) reviewed published research to determine if physical activity, prescription medications, over-the-counter vitamins and supplements, or cognitive training interventions could help to prevent dementia in patients who did not have it at the time of the studies. The vast majority of research showed that none of the interventions worked. Findings from four systematic evidence reviews are published in Annals of Internal Medicine.
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