http://canpku.org/ Latest news from Canadian PKU and Allied Disorders Wed, 18 Mar 2026 00:00:00 +0000 en http://canpku.org/index.cfm?fuseaction=Feature.showFeature&featureID=20 Wed, 18 Mar 2026 00:00:00 +0000 From the web CARES - Advocacy/Plaidoyer http://canpku.org/index.cfm?fuseaction=Feature.showFeature&featureID=20 The Government of Canada is taking action to streamline access to medicines to save lives, reduce costs, and build our economy. Canada has a significant pharmaceutical sector, ranking 9th globally. Its industry represents 2% of global sales, supports over 80,000 high-skilled jobs, contributes $14 billion to the economy, and invests over $2 billion annually in research and development. Companies recognize the importance of the Canadian market and continue to bring new medicines to Canada. From April 2025 to February 2026, Health Canada authorized 56 new pharmaceutical drugs, 23 new biologic drugs, 134 generic pharmaceutical drugs and 12 biosimilars. 43 of these contained medicinal ingredients that have never been approved for sale in Canada before. Health Canada works with Canada's Drug Agency and the Institut national d'excellence en santé et en services sociaux to conduct aligned reviews to shorten the time between Health Canada authorizations and reimbursement recommendations for drug plans. In the last two years, these aligned reviews have shortened the time between a regulatory decision and reimbursement recommendation by more than 80%. The work of the Task Force will build on the Government of Canada's Red Tape Review, which seeks to reduce unnecessary regulatory burden through a more risk-based and agile regulatory system that further aligns with trusted international regulators and is easier for Canadians and businesses to navigate. http://canpku.org/index.cfm?fuseaction=Feature.showFeature&featureID=19 Fri, 09 Jan 2026 00:00:00 +0000 From the web PKU/PCU - Phenylketonuria/Phénylcétonurie C - I am a Clinician R - Treatments & Therapies / Traitements & Thérapies CARES - Research/Recherche http://canpku.org/index.cfm?fuseaction=Feature.showFeature&featureID=19 Aurora Therapeutics, founded by CRISPR pioneers Jennifer Doudna and Fyodor Urnov, has officially launched to transform personalized gene editing into a scalable approach for treating rare genetic diseases. Backed by Menlo Ventures with $16 million in seed funding, Aurora combines modular gene editors, AI-driven guide design, and innovative regulatory strategies to develop therapies that can address multiple mutations in parallel. The company’s first focus is phenylketonuria (PKU), aiming to provide durable, population-scale treatments for patients with rare mutations. Aurora’s leadership team brings extensive experience in rare disease drug development, while its model leverages emerging regulatory frameworks to make personalized therapies economically and operationally viable.