Page 3 - Lorem Foundation News Today

Published: May 21, 2021 By Alex Keown Biopharma and life sciences companies strengthen their leadership teams and board with these Movers & Shakers.  AavantiBio – Jessie Hanrahan was named chief regulatory officer of AavantiBio. Hanrahan will oversee global regulatory affairs for AavantiBio’s diversified pipeline of gene therapy programs targeting rare diseases with significant unmet medical needs. Most recently, she was Vice President of Regulatory Science at bluebird bio, Inc. Pr...

Share Rare Daily Staff n-Lorem Foundation, a nonprofit that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living with ultra-rare diseases, said it entered into a partnership with Ultragenyx Pharmaceutical to bolster n-Lorem’s efforts. Ultragenyx joins Ionis Pharmaceuticals, Biogen, Charles River Labs, Covance by Labcorp, and Korea Institute of Toxicology as industry partners supporting n-Lorem’s work. Ultragenyx focuses on developing products for ser...

The technology to find treatments for rare and ultra-rare diseases exists today. The world needs a drug development paradigm for them all.

Share this article SAN DIEGO, Jan. 26, 2021 /PRNewswire/ --  n-Lorem Foundation, the first and only nonprofit organization whose mission is to create experimental antisense oligonucleotide (ASO) treatments for patients who have diseases caused by extremely rare mutations (1-30 patients worldwide) for free, for life, celebrates its first anniversary and the extraordinary progress made in 2020 toward its mission of offering immediate hope and rapid treatment to the millions of patients affected ...

Published: Jan 10, 2021 By Gail Dutton   n-Lorem Foundation has taken on a challenge that many nations consider too great: treating patients with ultra-rare diseases (which affect 30 or fewer people) for free, for life.  It received FDA guidance January 5 th and is in the midst of preparing the first package of investigational new drug (IND) applications – usually with an n-of-1, for individual patients. Patient dosing is expected to begin later this year. “No one has ever done th...

Share this article Share this article CARLSBAD, Calif., Dec. 17, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA-targeted therapeutics, announced today that its founder and executive chairman Stanley T. Crooke, M.D., Ph.D., will retire from Ionis and its board of directors effective June 2021. Dr. Crooke is stepping down so that he may focus on his scientific interests and the nonprofit organization he founded, the n-Lorem Foundation. Dr. Crooke will continue...

/PRNewswire/ -- n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living...