In 2018, Pfizer initiated a Phase Ib clinical trial of its gene therapy treatment for Duchenne muscular dystrophy (DMD). The AAV9 gene therapy, PF-06939926, is designed to provide DMD patients with a form of the dystrophin gene at the root of the condition, thereby countering the progressive muscle degeneration and weakness that characterizes the disease. Pfizer planned to dose 15 patients with the gene therapy, which must be stored at -70C. Continue reading →

Personalized medicine has taken a big step forward with the launch of non-profit n-Lorem Foundation, which will create patient-tailored antisense oligonucleotide (ASO) therapeutics for people with rare diseases at no cost to the patients. This comes at the same time as custom gene therapies for rare disease patients are being developed, including some combined with CRISPR. Continue reading →

As we mark the start of the 2020s, its interesting to reflect on the advances weve seen in research into muscle-wasting conditions over the past decade and what the next 10 years might bring. Continue reading →

-Enrollment ongoing in clinical trials of CTX001 for patients with severe hemoglobinopathies- -Enrollment ongoing in clinical trial of CTX110, targeting CD19+ malignancies- -Enrollment has begun in clinical trial of CTX120, targeting B-cell maturation antigen (BCMA)- ZUG, Switzerland and CAMBRIDGE, Mass., Feb. 12, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics(CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, today reported financial results for the fourth quarter and full year ended December 31, 2019. In 2019, CRISPR Therapeutics achieved important milestones and momentum across key programs. Continue reading →