is engaged in the development of gene therapies for rare, frequently terminal, diseases, including neurometabolic disorders, primary immune deficiencies, and blood disorders. The gene therapy approach uses blood stem cells to deliver corrected genetic information directly into the patient’s body.
Orchard’s pipeline demonstrates the diversity of disorders amenable to gene therapy – the company has no less than 12 drug candidates in development. Among these candidates, Libmeldy (OTL-200) stands out.
Libmeldy is in commercialization stages as a treatment for MLD (metachromatic leukodystrophy), a rare, mutation-based genetic disorder of the nervous system, received its approval for medical use in the EU in December 2020.
“We look forward to the company’s commercial execution in the EU and an eventual 2022 approval in the US. Last month ORTX received IND clearance from the FDA for the program paving the way for discussions with the US regulators to decide a suitable path forward toward a BLA filing.”
with possibly two gene therapies approved in the next 12-18 months and a pivotal study beginning in a third (MPS-I), we think ORTX shares are undervalued at these levels," the analyst concluded.
Wedbush analyst David Nierengarten rates ORTX as Outperform (i.e. Buy), and his $15 price target indicates a potential for 241% growth in the year ahead.
Only Buy ratings, 3, in fact, have been issued in the last three months. Therefore, ORTX gets a Strong Buy consensus rating. At $15, the average price target indicates shares could appreciate by 241% in the year ahead.
finance.yahoo.com/ne...tocks-174115335.html
Orchard’s pipeline demonstrates the diversity of disorders amenable to gene therapy – the company has no less than 12 drug candidates in development. Among these candidates, Libmeldy (OTL-200) stands out.
Libmeldy is in commercialization stages as a treatment for MLD (metachromatic leukodystrophy), a rare, mutation-based genetic disorder of the nervous system, received its approval for medical use in the EU in December 2020.
“We look forward to the company’s commercial execution in the EU and an eventual 2022 approval in the US. Last month ORTX received IND clearance from the FDA for the program paving the way for discussions with the US regulators to decide a suitable path forward toward a BLA filing.”
with possibly two gene therapies approved in the next 12-18 months and a pivotal study beginning in a third (MPS-I), we think ORTX shares are undervalued at these levels," the analyst concluded.
Wedbush analyst David Nierengarten rates ORTX as Outperform (i.e. Buy), and his $15 price target indicates a potential for 241% growth in the year ahead.
Only Buy ratings, 3, in fact, have been issued in the last three months. Therefore, ORTX gets a Strong Buy consensus rating. At $15, the average price target indicates shares could appreciate by 241% in the year ahead.
finance.yahoo.com/ne...tocks-174115335.html