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Precision BioSciences, Inc., based in Durham, North Carolina, is a pioneering biotechnology company dedicated to enhancing life through advanced genome editing. The company employs its proprietary genome editing platform, ARCUS, to develop innovative product candidates aimed at treating human diseases and offering solutions in food and agriculture.
ARCUS, distinguished by its compact size, enables the delivery of gene edits to a broader range of cells and tissues using both viral and non-viral methods. This versatility makes it a robust tool in Precision BioSciences' mission to address various health conditions and agricultural challenges.
The company has recently made significant strides in its projects, partnering with multiple organizations to expand its research and product development capabilities. These collaborations have been pivotal in propelling forward the potential applications of ARCUS technology.
Precision BioSciences is actively engaged in several cutting-edge projects that underscore its commitment to innovation. The company's financial health is reflected in its consistent investment in research and development, ensuring sustained growth and advancement in its technology.
Latest News:
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Precision BioSciences, Inc. (Nasdaq: DTIL) has announced the publication of a new paper in Nature Communications detailing preclinical results using its ARCUS nuclease to target and reduce mutant mitochondrial DNA (mtDNA) in vivo. The study led by Dr. Carlos T. Moraes demonstrated effective shifts in mtDNA heteroplasmy and notable safety in mouse models. MitoARCUS achieved up to 60% heteroplasmic shifts in vitro and showed no off-target editing in vivo. This innovative approach to gene editing may hold significant therapeutic promise for mitochondrial diseases, with further research anticipated.
Precision BioSciences (Nasdaq: DTIL) has appointed Alex Kelly as Chief Financial Officer, a position he has held in an interim capacity since December 2020. Kelly joined the company in October 2020 as Chief Corporate Affairs Officer. He will oversee finance, corporate communications, and investor relations, reporting directly to CEO Matt Kane. Shane Barton will continue as the principal accounting officer. Matt Kane praised Kelly's contributions, emphasizing his pharmaceutical experience and role in advancing the company's strategic financial goals related to allogeneic CAR T and gene editing therapies.
Precision BioSciences Inc. (Nasdaq: DTIL) announced that Chief Scientific Officer Derek Jantz, Ph.D., will participate in a fireside chat at the Jefferies Virtual Healthcare Conference from June 1-4, 2021. The chat is scheduled for June 2, 2021, at 1:30 PM ET. Investors and interested parties can access the live webcast on the company's website, with an archived replay available for 30 days. Precision BioSciences focuses on developing allogeneic CAR T and in vivo gene correction therapies using its ARCUS® genome editing platform, aimed at curing genetic and infectious diseases.
Precision BioSciences (Nasdaq: DTIL) announced additional data from its Phase 1/2a study of PBCAR0191, an allogeneic CAR T candidate targeting CD19. The data will be presented at the 2021 ASCO Meeting, June 4-8. The study included 13 patients with relapsed/refractory non-Hodgkin lymphoma, showing 77% had aggressive forms. Results highlighted an acceptable safety profile, with no severe adverse effects reported. The company is also progressing towards the clinical start of PBCAR19B, aimed at enhancing CAR T persistence. A conference call on data review is scheduled for June 4, 2021, at 8:00 a.m. ET.
Precision BioSciences (Nasdaq: DTIL) reported financial results for Q1 2021, achieving revenues of $16.3 million, up from $7.0 million in Q1 2020. The company aims to advance its allogeneic CAR T and in vivo gene editing pipelines, recently reacquiring global rights for its CAR T programs from Servier. A Phase 1 study of PBCAR19B is set to begin in May 2021. Research and development expenses were $25.6 million, with a net loss of $18.7 million or $(0.33) per share, improved from a loss of $(0.52) per share in the previous year.
Precision BioSciences (Nasdaq: DTIL) revealed promising preclinical findings from a collaboration with the University of Pennsylvania at the 2021 ASGCT Annual Meeting. Their ARCUS® genome editing platform demonstrated a significant reduction in serum TTR levels after targeting the transthyretin gene linked to transthyretin amyloidosis (ATTR). In nonhuman primates, serum TTR levels dropped over 95% by day 21 post-treatment, with effects lasting up to 250 days. This suggests a potential 'one-time' treatment for ATTR, indicating the ARCUS technology's transformative potential for rare genetic diseases.
Precision BioSciences, Inc. (Nasdaq: DTIL) will report its first quarter 2021 financial results and provide a business update on May 13, 2021. The company focuses on developing innovative allogeneic CAR T and in vivo gene correction therapies utilizing its proprietary ARCUS® genome editing platform. This platform aims to enhance therapeutic safety and control, targeting genetic and infectious diseases for which current treatments are inadequate.
Precision BioSciences, Inc. (Nasdaq: DTIL) announced a preclinical study presentation at the ARVO Annual Meeting, focusing on ARCUS genome editing for autosomal dominant Retinitis Pigmentosa (adRP). The study demonstrates that ARCUS can rejuvenate rod photoreceptor structure and function in a pig model with the common P23H rhodopsin mutation. Significant improvements in rod-driven signals were observed, suggesting potential for late-stage treatment in humans. This research adds to growing preclinical support for ARCUS in addressing rare genetic conditions.
Precision BioSciences, Inc. (Nasdaq: DTIL) announced a poster presentation at the ASGCT Annual Meeting, scheduled for May 11-14, 2021. The poster titled "Translation of an AAV-delivered gene editing approach for transthyretin amyloidosis in animal models" will be presented by Dr. Jenny A. Greig from the University of Pennsylvania. This research highlights the use of ARCUS gene editing in treating transthyretin amyloidosis, a disease caused by misfolded transthyretin protein. Initial findings indicate a successful knock-out of the TTR gene, showing promise for permanent genomic edits.
Precision BioSciences has appointed Dr. Alan List as Chief Medical Officer, enhancing its leadership team. Dr. List brings significant expertise in hematology and oncology, having led clinical programs for multiple FDA-approved drugs. He will oversee the development of allogeneic CAR T therapies, including the lead therapy PBCAR0191 and next-gen PBCAR19B. Dr. List's prior role as a strategic advisor will support a smooth transition from outgoing CMO Chris Heery, who will remain as a consultant. This appointment comes as Precision progresses with several clinical trials.