According to a story from gurufocus.com, the biopharmaceutical company Abeona Therapeutics, Inc., recently announced the presentation of positive interim findings from two different phase 1/2 clinical trials. These trials were…
The National Center for Advancing Translational Sciences (NCATS) of the National Institutes of Health (NIH) and the Center for Biologics Evaluation and Research (CBER) of the Food and Drug…
According to a story from BioBuzz, the innovation of a platform for the delivery of gene therapies could help trigger a golden age of development for this new class of…
As originally reported in Euronews, there is limited supply of expensive medicines for rare diseases, so it can be difficult to prioritize which patients will have their lives saved when…
According to an announcement from the Muscular Dystrophy Association, the organization has recently awarded a total of $1,076,232 in MDA Venture Philanthropy (MVP) funding towards the drug company AavantiBio, which…
Gene therapy has become a technological golden snitch: the end of an era for many genetic disorders if we can grasp the treatment’s potential. However, the technology comes with a…
Hemophilia A is the most common type of hemophilia, accounting for 8 of every 10 cases. Treatment in the past consisted of the administration of concentrated factor VIII through…
Mustang Bio, a biopharmaceutical company, has been working with St. Jude Children's Research Hospital to develop MB-107, a lentiviral gene therapy to treat X-linked severe combined immunodeficiency. Mustang Bio…
According to a story from globenewswire.com, the genetic medicines company Homology Medicines, Inc., has recently released promising early data from its phase 1/2 clinical trial. This clinical trial is testing…
According to a story from BioPortfolio, the drug developer Rocket Pharmaceuticals recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This study is…
According to a story from biopharmadive.com, the race to a hemophilia A gene therapy continues to tighten following a recent release of early data from Sangamo Therapeutics and Pfizer. The…
By Caitlin Seida from In The Cloud Copy People living with the severe hemophilia A may soon see a new treatment option available thanks to the work of BioMarin Pharmaceuticals.…
Jojo, a ten-year-old girl, has become the first person to ever receive a new gene therapy for the treatment of GM1 gangliosidosis. The treatment, called AXO-AAV-GM1, was used in…
The University of Massachusetts Medical School has recently announced preliminary results from their trial for Tay-Sachs disease. This trial is investigating a gene therapy and researchers are excited about it's…
According to a story from BioSpace, the gene therapy companies Sarepta Therapeutics, Inc. and StrideBio, Inc. have recently announced the completion of a licensing and collaboration agreement that could lead…
According to a story from the Times of Israel, a team of researchers associated with the University of Massachusetts appear to have discovered a potential breakthrough in regards to the…
According to a story from BioSpace, the genetic medicines company Homology Medicines, Inc., has recently released preclinical data supporting the development of two experimental gene therapies. One, called HMI-202, is…
According to a story from pmlive.com, the gene therapy company Bluebird Bio's latest recently released results from the company's phase 2/3 clinical trial demonstrates further encouraging data for its Lenti-D…
As originally reported in Biobuzz, the story of rare disease treatment took a turn in the age of modern technology. Rare diseases have traditionally been inaccessible to the naked eye,…
Genes themselves are a relatively new discovery, found only about a century ago. Gene therapy, a medical procedure that alters genes to address disease, is a concept a few decades…
As originally reported in the Washington Post: many parents of children with rare diseases are familiar with the agony of mystery surrounding their children’s medical conditions. The frustration of searching…
Genes themselves are a relatively new discovery, found only about a century ago. Gene therapy, a medical procedure that alters genes to address disease, is in its first 30 years.…
According to a publication from Charcot Marie Tooth News, biomedical law expert Timo Minssen recently gave a presentation at the New York Genome Center where he discussed the potential ethical…
According to a story from globenewswire.com, gene therapy, a current hot topic in the medical field, is continuing to make headlines. The drug company Amicus Therapeutics recently announced that a…
According to a press release from the Kentucky-based Apellis Pharmaceuticals, the Company has initiated a program to investigate the use of APL-9, an investigational drug, in gene therapies that involve…
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