Precision BioSciences Aktie

Precision BioSciences has activated the first study site for PBGENE‑DMD; Arkansas Children’s Hospital has begun screening and enrolling ambulatory patients aged 2 to 7 with mutations in exons 45–55. PBGENE‑DMD is an in‑vivo gene‑editing therapy that uses two ARCUS nucleases in an AAV vector to correct the dystrophin gene and restore near‑functional dystrophin. The Phase 1/2 FUNCTION‑DMD study is assessing safety, tolerability, efficacy, dystrophin expression and functional outcomes; the program holds FDA orphan‑drug and fast‑track designations. Investors reacted positively, with the stock up about 71% year‑to‑date near its 52‑week high, while a $183 million market capitalization and ongoing cash burn could affect the company’s financial runway. » Más en de.investing.com

Precision BioSciences presented phase‑1 data from the ELIMINATE‑B study at the IC‑HBV symposium: PBGENE‑HBV was well tolerated at two dose levels and showed signals of efficacy with reductions in viral load. The gene therapy aims to eliminate cccDNA and inactivate integrated HBV DNA; recruitment for HBeAg‑negative patients is underway in Moldova, Hong Kong and New Zealand, with expansion to the USA and UK planned. The company received a US patent for PBGENE‑HBV through March 2042 and also reports FDA orphan‑drug designation for PBGENE‑DMD; financials show low revenues (USD 1.26 million) with a positive cash position, but investor warnings indicate rapid cash burn. » Más en de.investing.com

Precision BioSciences has been granted US patent No. 12,410,418 for the gene therapy PBGENE-HBV, providing protection until March 2042 and covering the ARCUS nuclease that targets cccDNA and integrated HBV DNA. The patent complements existing protections in Europe and Hong Kong. The phase‑1 ELIMINATE‑B study continues: cohort 3 is being dosed, repeated doses are ongoing in cohort 2, and recruitment is underway in Moldova, Hong Kong and New Zealand; US study sites are imminent. Initial data showed a marked viral load reduction and a favorable safety profile at the lowest dose; the company also received orphan drug designation for PBGENE‑DMD. » Más en de.investing.com