Precision BioSciences Secures Orphan Drug Designation for DMD Treatment, DTIL Stock News

Precision BioSciences has received orphan drug designation from the FDA for its Duchenne muscular dystrophy treatment. This designation offers potential benefits like tax credits and market exclusivity upon approval, which could aid in the treatment's development and commercialization. Analysts forecast an average target price of $21.50, with a high estimate of $30.00 and a low estimate of $13.00.

Precision BioSciences (NASDAQ:DTIL) has received Orphan Drug Designations from the FDA for its nuclease treatments targeting Duchenne muscular dystrophy (DMD). The designations were granted for the company’s adeno-associated virus serotype 9-delivered DMD19-20L.431 and DMD35-36L.457 nucleases, which are being developed to treat DMD [1].

Orphan Drug Designation is given to drugs and biologics intended for rare diseases that affect fewer than 200,000 people in the United States. This status provides certain benefits to drug developers, including tax credits for qualified clinical trials and potential market exclusivity. DMD is a rare genetic disorder characterized by progressive muscle degeneration and weakness due to alterations in a protein called dystrophin that helps keep muscle cells intact [2].

The designation from the FDA is a significant milestone for Precision BioSciences, as it offers potential benefits that could aid in the treatment's development and commercialization. Analysts forecast an average target price of $21.50, with a high estimate of $30.00 and a low estimate of $13.00 [3].

Separately, Medera Inc. and the University of Kansas Medical Center have announced the successful treatment of the first patient in the MUSIC-DMD Phase 1b clinical trial, which is investigating a gene therapy approach for DMD-associated cardiomyopathy. The trial is evaluating the safety and efficacy of AAV1.SERCA2a, an adeno-associated virus serotype 1 (AAV1) gene therapy that delivers the SERCA2a gene directly to heart muscle cells [4].

References:
[1] https://in.investing.com/news/stock-market-news/precision-biosciences-gets-fda-orphan-drug-designations-for-dmd-treatment-93CH-4916498
[2] https://www.investing.com/news/stock-market-news/precision-biosciences-gets-fda-orphan-drug-designations-for-dmd-treatment-93CH-4140350
[3] https://www.biospace.com/press-releases/medera-and-university-of-kansas-medical-center-announce-first-patient-treated-in-pioneering-gene-therapy-trial-for-duchenne-muscular-dystrophy-associated-cardiomyopathy
[4] https://www.biospace.com/press-releases/medera-and-university-of-kansas-medical-center-announce-first-patient-treated-in-pioneering-gene-therapy-trial-for-duchenne-muscular-dystrophy-associated-cardiomyopathy