Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR), according to NCBI, are sequences that replicate and infect bacterial cells. CRISPR has been in the news recently, but this time it has…
The FDA grants designations, such as the Rare Pediatric Disease and Orphan Drug designations, to aid in the drug development process. Recently, LEXEO Therapeutics has received these two designations for…
In a news release from June 29, 2021, biotechnology company Neurogene Inc. shared that its adeno-associated virus (AAV) encoding a codon-optimized human CLN5 transgene (hCLN5) received Orphan Drug designation from…
A recent announcement in Biospace by Capsida Biotherapeutics and CRISPR Therapeutics of their newly-formed partnership offers renewed hope for amyotrophic lateral sclerosis (ALS) and Friedreich’s Ataxia patients. The companies believe…
Although it is still considered an experimental technique, gene therapy shows promise for treating a wide variety of genetic diseases or disorders. In a recent news release from the Sanford…
Gene therapies are becoming an increasingly popular treatment for genetic diseases, especially rare ones. Now, medical professionals are trying a new strategy with gene therapy: adding acetaminophen, also known by…
According to a story from bbc.com, five month old Arthur was born with the rare disease spinal muscular atrophy (SMA). He is one of the first people in the UK…
From May 11 to 14, 2021, the American Society of Gene & Cell Therapy (ASGCT) Virtual Annual Meeting took place. During the 24th annual meeting, a variety of stakeholders met…
In a press release from late May 2021, AAV gene therapy company and Bayer AG subsidiary Asklepios BioPharmaceutical, Inc. ("AskBio") shared that the FDA approved its Investigational New Drug (IND)…
During the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting, which took place virtually from May 11-14, 2021, biotechnology company M6P Therapeutics ("M6P") presented preclinical data…
There are multiple gene mutations associated with severe combined immunodeficiency (SCID), a rare genetic disorder which severely inhibits - or stops - immune function. But according to New Atlas, an…
While some therapies offer potential benefits to patients, not all therapies show efficacy within the clinical trial environment. Recently, shares MedCityNews, this occurred with biotechnology company Biogen Inc.'s ("Biogen") gene…
According to corporate news shared on Street Insider, biopharmaceutical company BridgeBio Pharma, Inc. ("BridgeBio") is making ground with its AAV5 gene therapy, BBP-631. Recently, the FDA granted Fast Track designation…
According to a recent article in USA Today, the decades-old vision of gene therapy has become a reality for a Danish couple. Thomas Felborg, his wife Daria Rokina brought…
On April 28th, the virtual World Orphan Drug Congress USA 2021 was held. The program featured a variety of subjects relevant to the development of orphan drugs and the rare…
According to a recent news release, CTX001, a gene-edited therapy for patients with transfusion-dependent beta thalassemia, received Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The treatment was…
Gene therapy is a recent development in the industry, and it is one that has brought hope to many people who live with a genetic condition without a viable treatment…
In a recent press release, biotechnology and gene therapy company LEXEO Therapeutics ("LEXEO") shared that its gene therapy LX1001 received Fast Track designation from the FDA. Overall, LX1001 is…
A variety of gene mutations cause retinitis pigmentosa (RP) - but how do those genes affect the degeneration of rod and cone photoreceptors in the eyes? According to Medical XPress,…
Sometimes it can be difficult to find or afford certain medications designed for your condition. However, this may soon be changing for Canadians with spinal muscular atrophy (SMA). On March…
According to a story from the National Hemophilia Foundation, the drug company uniQure has provided an update in regards to a case of hepatocellular carcinoma (HCC) that emerged in a…
In a recent press release, clinical-stage gene therapy company Rocket Pharmaceuticals, Inc. ("Rocket") shared that its investigational gene therapy candidate, RP-L201, received Priority Medicines (PRIME) designation from the European…
Rocket Pharmaceuticals, Inc. ("Rocket") has dedicated its mission to developing gene therapy solutions for rare childhood conditions. Recently, the company shared the publication of preliminary data from a Phase…
bluebird bio has created a gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD), which is now making its way through a clinical development program. According to Businesswire, bluebird has…
According to Charcot-Marie-Tooth News, the CMT Research Foundation has announced that they will provide additional funding for the development of a gene therapy for Charcot-Marie-Tooth disease Type 1 (CMT1A). The…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
