Prevail Therapeutics Inc. (Prevail), a gene therapy company developing AAV-based gene therapies for patients with neurodegenerative diseases, today announced the U.S. Food and Drug Administration (FDA) has accepted the Company’s Investigational New Drug (IND) application for its lead gene therapy program, PR001. PR001 is a potentially disease-modifying, single-dose, AAV9-based gene therapy being developed for the treatment of Parkinson’s disease patients with a GBA1 mutation (PD-GBA).
Prevail Therapeutics Announces IND Active... - Cure Parkinson's
Prevail Therapeutics Announces IND Active for Phase 1/2 Trial of its Gene Therapy PR001 to Treat Parkinson’s Disease Patients with GBA1 Muta
![Farooqji profile image](https://images.hu-production.be/avatars/d2bcede6695608a9d172b4c00f6b376c_small@2x_100x100.jpg)
Written by
![Farooqji profile image](https://images.hu-production.be/avatars/d2bcede6695608a9d172b4c00f6b376c_small@2x_100x100.jpg)
Farooqji
To view profiles and participate in discussions please or .
Read more about...
Not what you're looking for?
You may also like...
Dry Mouth, Gum Disease, Chapped Lips, Dry Eyes, Dry Skin, Eye Floaters And Some Possible Help From A Single Spray!
With winter just around the corner, this information may be most useful and timely! With winter...
Dopamine Dysregulation Syndrome
Definition here;
https://en.m.wikipedia.org/wiki/Dopamine_dysregulation_syndrome
How can docs be...
In Pursuit Of Restful Sleep And Help For Nocturia
One topic that seems to come up regularly on this forum is the issue of poor sleep or insomnia and...
Adding Sinemet to Mucuna
We're trying to go in the opposite direction of many here by adding Sinemet to Mucuna and are...
Rethinking Parkinson’s Disease: could dopamine reduction therapy have clinical utility. (The article)
The replies in Michel0220's contribution regarding Jonathan Sackner Bernstein's theory on dopamine...