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Rare Disease Day 2020

Tomorrow we celebrate Rare Disease Day to raise awareness for the 300 million individuals worldwide who live with rare diseases.


Passage Bio Announces Pricing of Initial Public Offering


CRISPR “Minigene” Approach Stops Genetic Liver Disease in Mice

A new CRISPR gene-editing technique prevented a genetic liver disease known to be driven by hundreds of different mutations and improved clinical symptoms in mice, Penn Medicine researchers reported in new proof-of-concept study published online in Science Advances. The findings suggest a promising CRISPR tool that could potentially treat patients with a rare metabolic urea-cycle disorder caused by a deficiency the enzyme, ornithine transcarbamylase (OTC), as well as other hereditary diseases triggered by different mutations on the same gene.


FDA Continues Strong Support of Innovation in Development of Gene Therapy Products

This is a pivotal time in the field of gene therapy as the FDA continues its efforts to support innovators developing new medical products for Americans and others around the world. To date, the FDA has approved four gene therapy products, which insert new genetic material into a patient’s cells. The agency anticipates many more approvals in the coming years, as evidenced by the more than 900 investigational new drug (IND) applications for ongoing clinical studies in this area. The FDA believes this will provide patients and providers with increased therapeutic choices.


Rare Disease CDKL5 Deficiency Disorder Granted WHO Disease Classification

The rare genetic disease CDKL5 Deficiency Disorder (CDD) has been designated with a new disease code in the International Classification of Diseases (ICD), the medical classification list from the World Health Organization (WHO). The CDKL5 Deficiency Disorder diagnostic code will be incorporated in the October 1, 2020 classification revision.


Passage Bio to Present at Barclays Gene Editing & Gene Therapy Summit

Passage Bio, a genetic medicines company developing AAV-delivered gene therapies for the treatment of rare monogenic central nervous system diseases, today announced that Jill Quigley, chief operating officer, will present at the Barclays Gene Editing & Gene Therapy Summit on Wednesday, November 13th, 2019 at 9:30 a.m. ET in New York, NY.