The New Zealand Medicines and Medical Devices Safety Authority has authorized Intellia Therapeutics to launch a Phase 1/2 trial testing NTLA-2002, its investigational gene-editing therapy, in adults with hereditary angioedema (HAE). According to the company, patient enrollment is expected to start by the end of the year. Data…
A crowdfunding campaign aims to raise $45,000 to support “Rare,” a documentary film featuring the struggles and achievements of people living with rare diseases and their families. Sweis Entertainment and Digital Cave Media launched the campaign — allowing filmmakers to finish producing and to release the documentary — on Kickstarter.
CAMP4 Therapeutics’ platform that targets a class of RNA known as regulatory RNAs, or regRNAs, can improve the activity of the gene that is defective in most cases of hereditary angioedema (HAE). That is according to new preclinical data shared by scientists from CAMP4 at the 17th Annual…
A new U.S. initiative called Rare Disease Cures Accelerator–Data and Analytics Platform — dubbed RDCA–DAP — aims to accelerate treatment innovation across rare diseases by sharing existing patient data and promoting the standardization of new data collection. Launched during a virtual workshop in September, the U.S. Food and Drug…
Cycle Pharmaceuticals has launched Sajazir, an injectable form of icatibant, as a new treatment option for acute attacks of hereditary angioedema (HAE) in adults. Patients who use Sajazir can take advantage of Cycle Vita, the company’s recently created rare disease product support hub. Icatibant, also sold as Firazyr,…
Astria Therapeutics is the new name of the company that’s developing STAR-0215, an investigational kallikrein-inhibitor previously known as QLS-215, for the treatment of hereditary angioedema (HAE). Formerly called Catabasis Pharmaceuticals, the company said it changed its name to reflect its pledge of having patients serve as its guiding…
Football and science seem to be disparate fields of play at first glance, but the nonprofit Uplifting Athletes is finding common ground by leveraging the popularity of college gridiron games to fund research for rare diseases. Its nearly two dozen chapters — representing college football teams across the nation…
Orladeyo (berotralstat), a daily oral therapy to prevent swelling attacks in people with hereditary angioedema (HAE), is now available in England, Wales, and Northern Ireland for eligible patients ages 12 and older. That authorization came as the U.K.’s National Institute for Health and Care Excellence (NICE) recommended Orladeyo —…
A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest therapeutic needs, but one that is often left behind. Named the Institute for Life Changing Medicines, the project was…
The U.S. Food and Drug Administration (FDA) has lifted the hold it had placed on KalVista Pharmaceuticals’ Phase 2 KOMPLETE trial of KVD824 as a treatment to prevent swelling attacks in people with hereditary angioedema (HAE). KalVista said it is working closely with study investigators and clinical sites so…
Get regular updates to your inbox.