February 28, 2026
Op-Ed Authored by Lord (David) Cameron, former UK Prime Minister and Chair of the OHC Advisory Council published in The Daily Telegraph on Saturday, Feb. 28, 2026 (Rare Disease Day) Rare diseases are not actually that rare. Collectively they affect 500 million people around the world. Some, such as Huntington’s disease...
Continue ReadingOctober 25, 2023
ABX1100 is a first-in-class molecule representing a new approach to reducing toxic levels of glycogen that build up in muscles of patients with Pompe Disease. Continue Reading
October 18, 2023
First Ten Years. Accelerating Discoveries into Medicines. Continue Reading
October 04, 2023
Monoclonal antibodies could provide a much-needed tool to protect vulnerable populations from severe COVID-19 Continue Reading
October 03, 2023
The Therapeutics Accelerator will identify, fund and advance innovative projects from academic partners in the U.K. and U.S. with the goal to deliver 40 new potentially life-changing therapies for rare diseases into clinical trials over 10 years. Continue Reading
September 29, 2023
Aleta hopes to initiate the registrational trial investigating ALETA-001 in B-cell malignancies in 2024/25. Continue Reading
September 23, 2023
Agreement enables the development of therapeutics, including AcuraStem's AS-202 Candidate, an antisense oligonucleotide that suppresses levels of the PIKFYVE kinase Continue Reading
September 14, 2023
New gene therapy company created and funded by Oxford Science Enterprises, Harrington Discovery Institute and Old College Capital in partnership with six leading scientists from the world-renowned UK Respiratory Gene Therapy Consortium (GTC) Continue Reading
September 14, 2023
Cell therapies derived from stem cells could one day prevent vision loss or even restore sight in retinal degenerative disease. Continue Reading
August 22, 2023
A Conversation with Jonathan S. Stamler, MD, President of Harrington Discovery Institute Continue Reading
July 05, 2023
New Orleans, LA – LSU Health New Orleans today announced that has exercised its option to receive an exclusive license to certain early-stage technology designed to slow the progression of DNA repeat expansion disorders.In DNA repeat expansion disorders, a segment of repeated DNA expands within a gene to cause disease. The... Continue Reading