Duchenne muscular dystrophy is a genetic, progressive condition that causes loss of muscle function over time. Its mostly seen in boys and men but can occur, often in a milder form, in girls, and is present at birth. While many new treatments are on the horizon, current treatment options for Duchenne may slow the progression of the disease and treat its symptoms, but cannot halt or reverse its course. Continue reading →

Shareholders of Pfizer (NYSE:PFE) have had an eventful year. A long-awaited spinoff and a global pandemic populated most of the headlines about the company, but those weren't the only developments that will affect the stock in 2021 and beyond. As we approach the holidays, it's useful to look back and ask how much you would have if you had invested in Pfizer at the beginning of the year Continue reading →

Eli Lilly has stepped into the gene therapy space after announcing a deal to acquire Prevail Therapeutics, a company focused on developing adeno-associated virus (AAV)-based gene therapies for neurodegenerative diseases. Lilly will acquire Prevail for $22.50 per share in cash, plus one $4 contingent value right dependent on the first regulatory approval of a product from Prevails pipeline. Continue reading →

CRISPR -- which stands for "clustered regularly interspaced short palindromic repeats" -- has been a hot area of research and investment since scientists discovered that this naturally occurring gene-editing function of bacteria could conceivably be used to treat genetic diseases. Several companies are using gene-editing in their attempts to cure illnesses caused by errors on a single gene such as sickle cell disease, hemophilia, and cystic fibrosis. One of them, CRISPR Therapeutics (NASDAQ:CRSP), has produced results that could not only make it a winner in single-gene disorders, but position it to tackle much more complex -- and profitable -- diseases in the years ahead Continue reading →

Eli Lilly has joined the ranks of big pharma companies building a position in gene therapy, paying $26.50 per share to take control of New York-based Prevail Therapeutics and its two clinical-stage development programs. Prevail has been snapped up just three years after being set up by OrbiMed's co-head of private equity Jonathan Silverstein, aiming to tackle Parkinsons disease (PD) caused by mutations in the glucocerebrosidase (GBA1) genea disease Silverstein was diagnosed with in 2016 Continue reading →