What does Sarepta Therapeutics do?
Sarepta is engineering solutions for rare diseases with science that is on the forefront of precision genetic medicine. Our platforms include: gene therapy, RNA technologies, and gene editing. We are in a daily race to save lives stolen or impacted by rare disease.
Is Casimersen gene therapy?
Casimersen is a type of gene therapy known as an antisense oligonucleotide, which can cause the protein-making machinery in a cell to skip over exon 45 in the dystrophin gene. This allows the cell to overcome some dystrophin mutations and make a shortened dystrophin protein that is partially functional.
Where is Sarepta based?
Cambridge, Massachusetts
Sarepta Therapeutics, Inc. (Nasdaq: SRPT) is a medical research and drug development company with corporate offices and research facilities in Cambridge, Massachusetts, United States.
What happens in exon skipping?
In molecular biology, exon skipping is a form of RNA splicing used to cause cells to “skip” over faulty or misaligned sections (exons) of genetic code, leading to a truncated but still functional protein despite the genetic mutation.
What are the two side effects of eteplirsen?
The recommended regimen is 30 mg per kg body weight once weekly by intravenous infusion. Side effects of eteplirsen include headache, fever, falls, abdominal pain, cough and nausea. Injection site reactions and hypersensitivity reactions including rash, pruritus, urticaria and skin exfoliation have occurred.
Is Sarepta a good place to work?
Management does everything possible for Sarepta to be very nice and pleasant place to work. Recruitment process is rather strict and meticuluos, therefore employees are highly knowledgible, intelligent, professional, patients dedicated, and nice decent people to work with.
How many employees does Sarepta have?
As of the end of 2018, Sarepta had 499 employees, including 255 working in R&D and 48 in sales. The company’s total headcount is now tracking to about 900 by the end of 2019, Ingram told investors during the firm’s second-quarter earnings call in August.
Is Casimersen FDA approved?
Today, the U.S. Food and Drug Administration granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping (Exons are pieces of DNA that provide information for making proteins …
Where is Sarepta in the Bible?
Sarepta (near modern Sarafand, Lebanon) was a Phoenician city on the Mediterranean coast between Sidon and Tyre, also known biblically as Zarephath.
What diseases can exon skipping cure?
Exon skipping is a treatment approach for people whose Duchenne muscular dystrophy (DMD) is due to certain mutations in its causative gene. Duchenne is the most common and severe type of muscular dystrophy (MD), marked by progressive muscle degeneration.