United States

CRISPR/Cas9 gene editing appeal filed

Tuesday, August 1, 2017

CRISPR Therapeutics, Intellia Therapeutics, Caribou Biosciences and ERS Genomics announced that The Regents of the University of California, the University of Vienna and Dr. Emmanuelle Charpentier (collectively UC), co-owners of foundational intellectual property relating to CRISPR/Cas9 genome engineering, submitted an appellate brief to the U.S. Court of Appeals for the Federal Circuit seeking reversal of a decision by the U.S. Patent and Trademark Office’s Patent Trial and Appeal Board (PTAB) in an interference proceeding relating to CRISPR/Cas9 gene editing technology. In the appeal, UC requests reversal of the PTAB’s decision terminating the interference between certain CRISPR/Cas9 patent claims owned by UC and claims of the Broad Institute, Harvard University and the Massachusetts Institute of Technology (collectively, Broad).

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Regulatory Update, August 2017

Tuesday, August 1, 2017

FDA Draft Q&A Guidance on Electronic Records and Electronic Signatures

In the June 21, 2017, Federal Register, the FDA announced the availability of a draft guidance for industry titled, ‘‘Use of Electronic Records and Electronic Signatures in Clinical Investigations under our regulations—Questions and Answers.’’ This document pertains to sponsors, clinical investigators, institutional review boards (IRB), CRO and other interested parties who use electronic records, electronic signatures and electronic systems in FDA-regulated clinical investigations and who send certain required information to the FDA or others who keep certain required records and make them available during FDA inspections.

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The All of Us Research Program announces first community partner awards

Tuesday, July 25, 2017

The National Institutes of Health announced its first four community partner awards to begin building a national network of trusted leaders to motivate diverse communities to join the All of Us Research Program, part of the Precision Medicine Initiative. This initial group of awardees will receive a combined $1.7 million this fiscal year, with future support planned pending the availability of funds. These awardees will raise awareness about the program among seniors, Hispanics and Latinos, African Americans and the LGBTQ community, to complement other outreach efforts of the program. NIH anticipates making additional funding awards for community partners in the future, drawing on the lessons learned from this initial set of awardees.

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Survey: Discussions about clinical trials should be part of standard of care

Tuesday, July 25, 2017

An overwhelming majority of Americans (86%) agree that healthcare professionals should discuss clinical trials with patients diagnosed with a disease as part of their standard of care, according to a new national public opinion survey commissioned by Research!America. And three-quarters of respondents (75%) agree that taking part in clinical trials is as valuable to our health care system as giving blood. But Americans are split on whether it’s important for everyone to take part in a clinical trial if they are asked—44% agree while 45% disagree and 12% are not sure. A higher percentage (74%) said they would participate if they are asked by someone they trust.

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CSL Behring receives Orphan Drug exclusivity for Haegarda

Monday, July 24, 2017

CSL Behring announced that the FDA has granted the company seven years of orphan drug exclusivity for Haegarda (C1 Esterase Inhibitor Subcutaneous [Human]), the first and only subcutaneous treatment option for prevention of hereditary angioedema (HAE) attacks. Haegarda was approved by the FDA on June 22, 2017 for routine prophylaxis to prevent HAE attacks in adolescent and adult patients, and marketing exclusivity will continue through June 22, 2024.

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FDA accepts Amgen’s Aimovig BLA for migraine

Monday, July 24, 2017

Amgen announced that the FDA has accepted for review the Biologics License Application (BLA) for Aimovig (erenumab) for the prevention of migraine in patients experiencing four or more migraine days per month. If approved, Aimovig is expected to be the first-and-only monoclonal antibody targeting the calcitonin gene-related peptide (CGRP) receptor, specifically designed for the prevention of migraine.

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