US tests bomb that splits ships in half
The U.S. has successfully tested a lighter version of a bomb that can blast huge ships in half.
The U.S. Air Force (USAF) said Wednesday (June 4) they dropped a 500-pound QUICKSINK munition from a B-2 Spirit stealth bomber.
It follows a dramatic 2022 trial where a larger 2,000-pound variant was used to destroy a full-scale surface vessel.
The USAF say the smaller version of the precision-guided bomb offers a lower-cost anti-ship weapon, with more being able to be carried by bombers and fighter jets.
Col. Dan Lehoski, 53rd Wing commander, said: "QUICKSINK offers an affordable, game-changing solution to rapidly and efficiently sink maritime targets.
"AFRL's 500-pound QUICKSINK variant adds options for the warfighter and enhances operational flexibility."
The live-fire exercise was a collaborative effort between Air Force Materiel Command's Air Force Research Laboratory (AFRL) and Air Force Test Center and Air Combat Command's 53rd Wing.
Col. Matthew Caspers, AFRL Munitions Directorate commander, said: "QUICKSINK is the result of a Joint collaboration that rapidly prototyped an affordable concept for holding surface targets at risk."
It has not been reported what target the new variant was used against.
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Business Wire
6 days ago
- Business Wire
Alnylam to Present Progress in Transforming the Treatment of Cardiovascular Disease with RNAi Therapeutics at European Society of Cardiology Congress 2025
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Company will present new data from its hypertension and transthyretin amyloidosis (ATTR) programs at the upcoming European Society of Cardiology (ESC) Congress 2025, taking place in Madrid, Spain, from August 29 - September 1, 2025. These presentations will highlight the potential of RNAi therapeutics to transform the treatment of cardiovascular disease and reinforce Alnylam's commitment to advancing innovative therapies for patients living with rare and more prevalent conditions underserved by current treatment options. Highlights include data from Cohort A of the KARDIA-3 Phase 2 study evaluating the investigational RNAi therapeutic, zilebesiran, in patients with uncontrolled hypertension and high cardiovascular risk. This analysis will be presented as a late-breaking abstract in the Hot Line 4 session on August 30, 2025. Data from the Company's flagship TTR franchise will also be presented, including new analyses from the HELIOS-B Phase 3 study of AMVUTTRA ® (vutrisiran), which delivers rapid knockdown of transthyretin, in patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM). These presentations include results of 12-month follow-up from the ongoing open-label extension (OLE) period of HELIOS-B, which will provide further insights into the sustained longer-term benefits of treatment with AMVUTTRA, and an analysis examining the effect of AMVUTTRA on days lost to death and/or hospitalization (DLDH). ESC Congress Presentation Details Hypertension Abstracts KARDIA-3: Zilebesiran as add-on therapy in adults with hypertension and established cardiovascular disease or at high cardiovascular risk Hot Line 4 Session Presentation Time: Saturday, August 30, 2025, 4:30 – 4:45 pm (CEST), 10:30 – 10:45 am (EDT) Presenting Author: Neha Pagidipati, U.S. Room: Madrid, Main Auditorium Impact of zilebesiran, an investigational RNA interference therapeutic targeting hepatic angiotensinogen, on renin–angiotensin system biomarkers in patients with mild-to-moderate hypertension Moderated ePoster: Treatment of Hypertension: Randomized Controlled Trials Time: Saturday, August 30, 2025, 5:15 – 6:00 pm (CEST), 11:15 am – 12:00 pm (EDT) Presenting Author: Michael A Weber, U.S. Room: Station 4, Research Gateway ATTR Abstracts Vutrisiran reduces days lost to death and/or hospitalization versus placebo in patients with transthyretin amyloidosis with cardiomyopathy in the HELIOS-B trial Moderated ePoster: Multidisciplinary Care in Heart Failure Time: Sunday, August 31, 2025, 9:15 – 10:00 am (CEST), 3:15 – 4:00 am (EDT) Presenting Author: Mazen Hanna, U.S. Room: Station 3, Research Gateway HELIOS-B: 12-month results from the open-label extension period of vutrisiran in patients with transthyretin amyloidosis with cardiomyopathy Abstract Session: Therapeutic Advances in Amyloid Cardiomyopathy Time: Sunday, August 31, 2025, 11:15 – 11:25 am (CEST), 5:15 – 5:25 am (EDT) Presenting Author: Pablo Garcia-Pavia, Spain Room: Science Box 2 (Research Gateway) Investor Webcast Information Alnylam management will discuss the KARDIA-3 results in a live event which will be webcast on August 30, 2025, at 1:00 p.m. ET (7:00 pm CEST). The webcast will be available on the Investors section of the Company's website at An archived webcast will be available on the Alnylam website approximately two hours after the event. AMVUTTRA ® (vutrisiran) INDICATION AND IMPORTANT SAFETY INFORMATION Indications In the EU, AMVUTTRA ® (vutrisiran) is indicated for the treatment of: hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN). wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Important Safety Information Reduced Serum Vitamin A Levels and Recommended Supplementation Vutrisiran treatment leads to a decrease in serum vitamin A levels. Supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients taking vutrisiran. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness). Adverse Reactions Commonly reported adverse reactions with vutrisiran were injection site reactions and increase in blood alkaline phosphatase and alanine transaminase. About AMVUTTRA ® (vutrisiran) AMVUTTRA ® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. It is marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and it is also approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in the US, Europe, Brazil, Japan and UAE. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only RNAi therapeutic approved for the treatment of both the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR). For US Media only: For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit For EU Media: For the full EU Summary of Product Characteristics, see About Zilebesiran Zilebesiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of hypertension to reduce cardiovascular risk in high unmet need populations. Zilebesiran targets angiotensinogen (AGT), the most upstream precursor in the Renin-Angiotensin-Aldosterone System (RAAS), a cascade which has a role in blood pressure (BP) regulation. Zilebesiran inhibits the synthesis of AGT in the liver, potentially leading to durable reductions in AGT protein, and ultimately, in the vasoconstrictor angiotensin (Ang) II. Zilebesiran utilizes Alnylam's Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables infrequent biannual subcutaneous dosing, increased selectivity and the potential to achieve continuous control of BP to impact cardiovascular risk. The safety and efficacy of zilebesiran have not been established or evaluated by the FDA, EMA or any other health authority. Zilebesiran is being co-developed and co-commercialized by Alnylam and Roche. About ATTR Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide. 1-4 About Cardiovascular Disease and Hypertension Cardiovascular disease (CVD) is a global health crisis and a leading cause of death worldwide, responsible for approximately 20 million deaths annually. 5,6 Hypertension is the primary cause of and number one modifiable risk factor for CVD. 7 An estimated 1 in 3 adults worldwide have hypertension, and, despite wide availability of antihypertensives, up to 80% of all patients, and up to a third of treated patients, do not reach and maintain blood pressure (BP) targets. 8 Even when blood pressure appears well managed, continuous control of BP may remain suboptimal, leading to variability in BP during the 24-hour period and in the long-term, putting patients at greater risk of cardiovascular events and end organ damage. 9-15 These patients require novel approaches that not only reduce BP, but also lower overall cardiovascular risk. About RNAi RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. 16 Its discovery has been heralded as 'a major scientific breakthrough that happens once every decade or so,' and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. 17 By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today's medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. 16 This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases. About Alnylam Pharmaceuticals Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its ' Alnylam P 5 x25 ' strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. Alnylam Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam's expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam's expectations regarding the potential of RNAi therapeutics to transform the treatment of cardiovascular disease and genetic and other conditions; Alnylam's ability to advance innovative therapies for patients living with rare and more prevalent conditions underserved by current treatment options; the safety and efficacy of vutrisiran for the treatment of ATTR-CM and the safety and efficacy of zilebesiran for the treatment of hypertension; and Alnylam's ability to achieve its ' Alnylam P 5 x25 ' strategy should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam's ability to successfully execute on its ' Alnylam P 5 x25 ' goals; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam's product candidates; actions or advice of regulatory agencies and Alnylam's ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam's approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam's product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam's ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam's ability to maintain strategic business collaborations; Alnylam's dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the 'Risk Factors' filed with Alnylam's 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam's subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing Alnylam's views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements. 1 Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638. 2 Gertz MA. Am J Manag Care. 2017;23(7):S107-S112. 3 Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9. 4 Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31. 5 GBD 2021 Causes of Death Collaborators. Lancet. 2024;403:2100-2132. 6 Lindstrom M, DeCleene N, Dorsey H, et al. J Am Coll Cardiol. 2022;80:2372-2425. 7 Yusuf S, Joseph P, Rangarajan S, et al. Lancet. 2020;395:795-808. 8 NCD Risk Factor Collaboration (NCD-RisC). Lancet. 2021;398:957-980. 9 Ebinger JE, Driver M, Ouyang D, et al. eClinicalMedicine. 2022;48:101442. 10 Kario K. Prog Cardiovasc Dis. 2016;59:262-281. 11 Doumas M, Tsioufis C, Fletcher R, et al. J Am Heart Assoc. 2017;6:e006093. 12 Mezue K, Goyal A, Pressman GS, et al. J Clin Hypertens. 2018;20:1247-1252. 13 Rothwell PM, Howard SC, Dolan E, et al. Lancet. 2010;375:895-905. 14 Tatasciore A, Renda G, Zimarino M, et al. Hypertension. 2007;50:325-332. 15 Mokadem ME, Boshra H, El Hady YA, et al. J Hum Hypertens. 2019;34:641-647. 16 Elbashir SM, Harborth J, Lendeckel W, et al. Nature. 2001;411(6836):494-498. 17 Zamore P. Cell. 2006;127(5):1083-1086.


Business Wire
07-08-2025
- Business Wire
Precision BioSciences Reports Second Quarter 2025 Financial Results and Provides Business Update
DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced financial results for the second quarter ended June 30, 2025, and provided a business update. 'Our team continues to be very disciplined about executing our plans and is making strong progress advancing our clinical stage PBGENE-HBV program while rapidly advancing PBGENE-DMD toward the clinic,' said Michael Amoroso, Chief Executive Officer of Precision BioSciences. 'The early Phase 1 safety and efficacy data for PBGENE-HBV from the first cohort of the Phase 1 ELIMINATE-B trial establishes proof of activity for our novel gene editing approach for chronic Hepatitis B. Our data shows that we have a novel, safe and active drug in all patients treated with a durable effect in one third of patients reinforcing the mechanism of PBGENE-HBV to eliminate cccDNA. We are very pleased with the safety profile demonstrated in Cohorts 1 and 2 which has enabled the Data Monitoring Committee to endorse enrolling Cohort 3 this month to test the next higher dose. Concurrently, we are accelerating the development of our second program, PBGENE-DMD, and were proud to receive both Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (FDA), underscoring the significant unmet need for new therapeutic options for patients living with DMD.' 'Given the unmet need, opportunity and enthusiasm for PBGENE-HBV and PBGENE-DMD, we are taking proactive steps to invest fully in these two programs while extending our expected cash runway to the second half of 2027 through a significant reduction in our non-program related annual operating expenses. These actions are expected to enable commencement of a Phase 2 study for PBGENE-HBV and a potential pivotal trial for PBGENE-DMD. Our team remains committed to delivering transformative therapies in areas with significant unmet need and, with a longer cash runway, we believe we are now even better positioned to deliver the meaningful clinical data that is expected by patients and shareholders for both of our wholly-owned programs,' added Mr. Amoroso. Wholly Owned Portfolio PBGENE-HBV (Viral Elimination Program): PBGENE-HBV is Precision's wholly owned in vivo gene editing program under investigation in a global first-in-human clinical trial, which is designed to be a potentially curative treatment for chronic Hepatitis B infection. PBGENE-HBV is the first and only potentially curative gene editing program to enter the clinic that is specifically designed to eliminate the root cause of chronic Hepatitis B, cccDNA, while inactivating integrated HBV DNA. The ELIMINATE-B trial is investigating PBGENE-HBV at multiple ascending dose levels with three dose administrations per dose level in patients with chronic Hepatitis B. On August 6, 2025, Precision announced Phase 1 safety and efficacy data for Cohort 1, the lowest dose level in the ELIMINATE-B trial. Cohort 1 consisted of three patients each of whom received three planned administrations of 0.2 mg/kg of PBGENE-HBV dosed approximately eight weeks apart. The primary objective is to characterize the safety of PBGENE-HBV. PBGENE-HBV was well-tolerated in all three patients in Cohort 1. Across Cohort 1, no patient experienced above a Grade 2 treatment-related adverse event, a serious adverse event, or dose-limiting toxicity. No clinically significant lab abnormalities were observed, including liver enzymes and platelets. PBGENE-HBV demonstrated a substantial HBsAg reduction in all three patients in Cohort 1 with best response reductions of 56%, 69% and 47% compared to baseline HBsAg levels (ranging between 562-11,813 IU/mL) in patients one, two and three, respectively. One of three patients (33%) in Cohort 1 achieved a durable HBsAg reduction of approximately 50% from baseline that was maintained as of the data cutoff-date (July 28, 2025), which was seven months after initial dosing. This data provides evidence of the ability of PBGENE-HBV to drive a durable antiviral response by editing the viral DNA at the source of chronic Hepatitis B infection. There was no association between baseline HBsAg and efficacy in Cohort 1. Precision also announced initial safety data from Cohort 2 (0.4 mg/kg) in the ELIMINATE-B study. As of the data cutoff, one patient received three dose administrations with two weeks of follow-up, and two patients received one dose administration with four weeks of follow-up. In these patients, no adverse events above Grade 2 were observed. No serious adverse events or dose limiting toxicities were observed, and no cumulative adverse effects were observed. There were no clinically significant elevations of liver transaminases. One additional patient did not complete their dose due to a transient infusion-related serious adverse event that resolved within minutes. The Data Monitoring Committee determined that this transient reaction was not dose-related or dose-limiting. Given the favorable safety profile of Cohorts 1 and 2, the Data Monitoring Committee recently recommended initiation of Cohort 3. The Company is on track to complete dosing of all three patients across all dose administrations in Cohort 2 and commence dosing Cohort 3. The Company expects to provide a data update later in 2025. PBGENE-DMD (Muscle Targeted Excision Program): PBGENE-DMD is Precision's development program for the treatment of DMD. DMD is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein and affects approximately 15,000 patients in the U.S. alone. There are currently no approved therapies that can drive durable and significant functional improvements over time. PBGENE-DMD is designed to improve function for more than 60% of patients afflicted with DMD by employing two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene. The aim of this approach is to restore a near-full length functional dystrophin protein within the body that more closely resembles normal dystrophin as opposed to synthetic, truncated dystrophin approaches with minimal functional benefit. The FDA granted PBGENE-DMD Rare Pediatric Disease designation in June 2025 for the treatment of DMD, highlighting the significant unmet need for new therapeutic options. This was followed in July 2025 by the FDA granting PBGENE-DMD Orphan Drug Designation, re-enforcing the need for new and better treatments. With the Rare Pediatric Disease designation, Precision may be eligible to receive a Priority Review Voucher upon FDA approval of PBGENE-DMD. In preclinical data presented at the ASGCT annual meeting in May 2025, PBGENE-DMD demonstrated significant and durable functional improvement in a humanized DMD mouse model. Following AAV delivery, PBGENE-DMD restored the body's ability to produce a functional dystrophin protein broadly across multiple muscle types, including cardiac and skeletal muscles. Over the course of nine months, mice treated with PBGENE-DMD showed increased dystrophin protein expression resulting in substantial and sustained functional muscle improvement. In addition, PBGENE-DMD-edited dystrophin mRNA transcript in muscle satellite stem cells, which are progenitor cells for new muscle cells, supports the potential for long-term durability. In July 2025, Precision announced new preclinical data building upon previous data shared at the ASGCT annual meeting. These data demonstrated that PBGENE-DMD produced a three-fold increase in dystrophin-positive muscle cells between three and nine months in the quadricep, gastrocnemius (calf), heart, and diaphragm. In the gastrocnemius specifically, up to 85% of cells were dystrophin-positive, indicating a high degree of productive gene editing. This broad increase in dystrophin-positive cells combined with the increased dystrophin protein detected in tissues further validates the improved muscle function that was observed over time and may be attributable to edited satellite cells. Precision is advancing the final U.S. investigational new drug (IND)-enabling toxicology studies with an anticipated IND and/or clinical trial application (CTA) filing targeted by the end of 2025 with initial clinical data expected in 2026. PBGENE-3243 (Mutant Mitochondrial DNA Elimination Program): PBGENE-3243 is a first-of-its-kind potential treatment for m.3243-associated mitochondrial disease that is designed to specifically target and eliminate mutant m.3243G mitochondrial DNA, thereby eliminating the root cause of the disease. Precision has paused development of PBGENE-3243 to prioritize its two lead programs, PBGENE-HBV and PBGENE-DMD. Partnered In Vivo Gene Editing Programs iECURE-OTC (Gene Insertion Program): Led by iECURE, ECUR-506 is an ARCUS-mediated in vivo gene editing program currently in a first-in-human Phase 1/2 trial (OTC-HOPE) evaluating ECUR-506 as a potential treatment for neonatal onset ornithine transcarbamylase (OTC) deficiency. Preliminary data from the study presented at ASGCT in May demonstrated a complete clinical response from three months post-exposure to the end of study at six months, as defined by the study protocol. The patient is now more than one year of age and is eating appropriate levels of protein for a child of his age. iECURE has reported that a second infant with severe OTC deficiency was dosed in the first half of 2025. The OTC-HOPE study is ongoing in the U.K., the U.S., Australia, and Spain, and iECURE expects to complete enrollment in 2025 and anticipates complete data from the trial in the first half of 2026. PBGENE-NVS (Gene Insertion Program): Precision continues to advance its gene editing program with Novartis to develop a custom ARCUS nuclease for patients with hemoglobinopathies, such as sickle cell disease and beta thalassemia. The collaborative intent is to insert, in vivo, a therapeutic transgene as a potential one-time transformative treatment administered directly to the patient to overcome disparities in patient access to treatment with other therapeutic technologies, including those that are targeting an ex vivo gene editing approach. Non-Core Ex Vivo Programs Azer-Cel (azercabtagene zapreleucel allogeneic CAR T treatment for cancer): Imugene Limited, Precision's clinical stage partner developing azer-cel for oncology indications, announced updated clinical data in July 2025. In the updated data in patients diagnosed with relapsed/refractory diffuse large B-cell lymphoma (DLBCL), two additional patients achieved a Complete Response and three additional patients achieved a Partial Response. Imugene reported that, as a result of the new data, the best overall response rate for azer-cel in DLBCL reached 75% and the Complete Response rate reached 55%. Based on the updated response rate and maturing durability data, as well as having been awarded FDA Fast Track Designation for DLBCL, Imugene expects to request an end of Phase 1 meeting with the FDA in the fourth quarter of 2025 to present the data and discuss designs for a pivotal/registrational trial for azer-cel. Other Announcements Mark Sulkowski, M.D., Professor of Medicine at the Johns Hopkins University School of Medicine and renowned expert in hepatic and infectious diseases has expanded his advisory role with Precision BioSciences. In the newly created role, Head Clinical Development Advisor, Dr. Sulkowski will work closely with Precision's leadership and cross-functional teams to support clinical strategy across the development lifecycle for the Company's on-going PBGENE-HBV Phase 1 clinical trial as well as initiation of later stage trials. His advisory role will focus on optimizing clinical trials, including translational integration, and aligning scientific rationale with regulatory objectives. Quarter Ended June 30, 2025 Financial Results 'As Precision advances the ELIMINATE-B clinical trial and prepares to file an IND and/or CTA for the PBGENE-DMD program we have been closely managing our operating costs. Cost management is evident in our decision to pause development on PBGENE-3243 in the second quarter and is reflected in a $3.9 million reduction in our second quarter total operating expenses as compared to the same period last year,' said Alex Kelly, Chief Financial Officer of Precision BioSciences. 'We have also extended our expected cash runway to the second half of 2027 to enable meaningful clinical data readouts for PBGENE-HBV and PBGENE-DMD. In July 2025, we initiated an operating efficiency program, including reductions in early research, manufacturing and general & administrative operating expenses which are aimed at reducing our annual cash operating expenses in each of 2026 and 2027 by approximately $25 million compared to the 2025 annual cash expense level.' 'In addition to significantly reducing our operating expenses, Precision will continue to pursue less dilutive sources of cash to even further extend the cash runway, including business development collaborations for future or deprioritized ARCUS programs as well as opportunities to monetize non-core program royalties and milestones,' added Mr. Kelly. Cash, Cash Equivalents, and Restricted Cash: As of June 30, 2025, Precision had approximately $84.8 million in cash, cash equivalents, and restricted cash. Based on its expected cash runway, Precision believes it is sufficiently capitalized to reach important milestones for both programs, including commencement of a Phase 2 study for PBGENE-HBV and a potential pivotal trial for PBGENE-DMD. The Company expects existing cash and cash equivalents, upfront and potential near-term cash from CAR T transactions, along with expected operating efficiencies, operational receipts, and availability of Precision's at-the-market (ATM) facility to extend Precision's cash runway into the second half of 2027. Revenues: Total revenues for the quarter ended June 30, 2025, were less than $0.1 million, as compared to $49.9 million for the quarter ended June 30, 2024. The decrease was expected and primarily the result of $48.2 million of non-cash revenue recognized in the prior period which represented all remaining deferred revenue related to the Prevail Therapeutics Agreement at its conclusion in April 2024 under generally accepted accounting principles. The upfront cash from this collaboration was received and recorded on the balance sheet in January 2021. Research and Development Expenses: Research and development expenses were $12.8 million for the quarter ended June 30, 2025, as compared to $17.2 million for the quarter ended June 30, 2024. The decrease of $4.4 million was primarily due to decreases in PBGENE-HBV direct expenses due to lower manufacturing and toxicology expenses as the program transitioned to the clinic in the fall of 2024. PBGENE-3243, which has been paused, and other research related expenses also decreased in the comparative period, offset by an increase in PBGENE-DMD expenses as the program was accelerated in the second quarter of 2025. General and Administrative Expenses: General and administrative expenses were $9.1 million for the quarter ended June 30, 2025, as compared to $8.5 million for the quarter ended June 30, 2024 as an increase in employee-related costs, including non-cash employee-related costs, offset a decrease in other general and administrative expenses. Net Loss: Net loss was $23.5 million, or ($2.13) per share (basic and diluted), for the quarter ended June 30, 2025. Net income was $32.7 million, or $4.70 per share (basic) and $4.67 per share (diluted), for the quarter ended June 30, 2024. About Precision BioSciences, Inc. Precision BioSciences, Inc. is a clinical stage gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS® genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes. Using ARCUS, the Company's pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit The ARCUS® platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into gene to cause expression/add function), elimination (removing a genome e.g. viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV such as in the DMD program). Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the key advantages of ARCUS and its key capabilities and differentiating characteristics; expectations about operational initiatives, strategies, further development, or timing of additional updates or data releases of PBGENE-HBV and PBGENE-DMD, including timing of dose administrations and subsequent cohorts in the ELIMINATE-B trial; the unique design of PBGENE-HBV to eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional or complete cures; the suitability of PBGENE-HBV for the treatment of hepatitis and the targeting of the root cause of the disease; plans to escalate to higher dose levels and next cohorts in the ELIMINATE-B clinical trial; the potential of PBGENE-DMD to be a first-in-class in vivo gene editing approach addressing the majority of DMD patients; expectations on accelerated development of the PBGENE-DMD program; the design of PBGENE-DMD to improve function over time and address more than 60% of patients with DMD; the potential for PBGENE-DMD to provide durable functional improvement with a one-time lower dose of AAV; the expected timing and opportunities of regulatory processes (including IND-enabling studies and filings such as INDs or CTAs for PBGENE-HBV and PBGENE-DMD and the acceptance of these filings by regulatory agencies); the possibility and eligibility to receive a Priority Review Voucher upon FDA approval of PBGENE-DMD; the safety data and antiviral activity established after the administrations of PBGENE-HBV; translation of results in preclinical studies of ARCUS nucleases to clinical studies in humans; the preclinical and clinical development and demonstrated, potential and expected safety, efficacy, durability, and benefit of PBGENE-HBV and PBGENE-DMD, as well as our other product candidates and those being developed by partners; the complete enrollment of the OTC-HOPE study and timing of full data from the trial in the first half of 2026; expectations and announcements about achievement of key milestones; our expected cash runway and the sufficiency of our cash runway extending into the second half of 2027 to advance PBGENE-HBV and PBGENE-DMD through meaningful Phase 1 data readouts and to enable commencement of a Phase 2 study for PBGENE-HBV and a potential pivotal trial for PBGENE-DMD; the potential of PBGENE-3243 as a first-of-its-kind treatment for m.3243-associated mitochondrial disease; the intention to pursue less dilutive sources of cash to even further extend the cash runway; and expectations from partners, including iECURE and Imugene Limited, on clinical data and timing and opportunities of regulatory processes. In some cases, you can identify forward-looking statements by terms such as 'aim,' 'anticipate,' 'approach,' 'believe,' 'contemplate,' 'could,' 'design,' 'designed,' 'estimate,' 'expect,' 'goal,' 'intend,' 'look,' 'may,' 'mission,' 'plan,' 'possible,' 'potential,' 'predict,' 'project,' 'pursue,' 'should,' 'strive,' 'suggest,' 'target,' 'will,' 'would,' or the negative thereof and similar words and expressions. Forward-looking statements are based on management's current expectations, beliefs, and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators' or other licensees' ability to identify, develop and commercialize product candidates; pending and potential product liability lawsuits and penalties against us or our collaborators or other licensees related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators' or other licensees' development of product candidates; our or our collaborators' or other licensees' ability to advance product candidates into, and successfully design, implement and complete, clinical trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; delays or difficulties in our and our collaborators' and other licensees' ability to enroll patients; changes in interim 'top-line' and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; our or our licensees' ability to obtain orphan drug designation or fast track designation for our product candidates or to realize the expected benefits of these designations; our or our collaborators' or other licensees' ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the rate and degree of market acceptance of any of our product candidates; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate executives and personnel; effects of system failures and security breaches; insurance expenses and exposure to uninsured liabilities; effects of tax rules; effects of any pandemic, epidemic, or outbreak of an infectious disease; the success of our existing collaboration and other license agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of sustained inflation, supply chain disruptions and major central bank policy actions; market and economic conditions; risks related to ownership of our common stock, including fluctuations in our stock price; our ability to meet the requirements of and maintain listing of our common stock on Nasdaq or other public stock exchanges; and other important factors discussed under the caption 'Risk Factors' in our Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2025, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC's website at and the Investors page of our website under SEC Filings at All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise. Precision Biosciences, Inc. Balance Sheets Data (In thousands, except share amounts) June 30, 2025 December 31, 2024 Cash, cash equivalents, and restricted cash $ 84,806 $ 108,468 Working capital 56,691 80,009 Total assets 108,928 136,388 Total liabilities 74,874 79,995 Total stockholders' equity $ 34,054 $ 56,393 Common stock outstanding 11,636,981 8,202,715 Expand
Yahoo
07-08-2025
- Yahoo
If Another Home For Humanity Is Out There, This Spaceship Could Get Us There
Here's what you'll learn when you read this story: Project Hyperion competition winners dreamed up Chrysalis, a starship and space habitat that could make it to the planet Proxima b in just 400 years (which is ridiculously fast). The spacecraft is designed to run on a fusion engine, create artificial gravity, and carry about a thousand people while flying at around a tenth of the speed of light. Though Chrysalis remains a concept (for now), it could end up launching something unprecedented in the future. Headed towards the Proxima Centauri system, the starship Chrysalis traverses a seemingly endless expanse of space as it soars toward its final destination—the potentially habitable planet Proxima b. There, over a thousand passengers who have been living in the airborne habitat (the descendants of a crew that launched from Earth four centuries ago) will build a new frontier for humanity. Chrysalis sounds as if it flew straight out of a scene in Isaac Asimov's Foundation. But despite its sci-fi features, this is an actual spacecraft concept that recently won the Project Hyperion design competition hosted by the Initiative for Interstellar Studies (i4is). The craft is the brainchild of an interdisciplinary team of Italian researchers—Giacomo Infelise, Veronica Magli, Guido Sbrogio, Nevenka Martinello, and Federica Chiara Serpe—who were challenged to come up with a floating habitat that would eventually touch down on the closest exoplanet to Earth, Proxima b. 'The presentation is rich and visually engaging, drawing comparisons to iconic works like Rama, and showcasing a clear passion for both design and storytelling,' the competition jury said of Chrysalis in a recent press release. 'Its overall spacecraft design seems to take inspiration from the gigantic world ship concepts of the 1980s.' Each team that embarked on this conceptual journey needed at least one architectural designer, one engineer, and one social scientist. Their mission was to figure out how to accommodate a thousand (give or take 500) people over the centuries it would take for the spacecraft to reach its destination. Like the fictional starships it was inspired by, Chrysalis would to produce artificial gravity through a rotation system, in order to try and counteract the detrimental effects of microgravity on the human body. Designing support systems for food, water, waste, and an atmosphere—as well as coming up with ways to provide livable conditions and meet basic needs—were also mandatory parts of the contest. Additionally, there would need to be methods of transferring knowledge from generation to generations in order to both keep culture alive and retain (and advance) technology. The spacecraft would also have to trek through space at a maximum velocity of a tenth of the speed of light, and make it to Proxima b in as close to 250 years as possible. The fastest spacecraft to date is NASA's Parker Solar Probe, which recently zoomed past the Sun at a blazing 692,000 kmph (430,000 mph). But even Parker would still be short of one trillion miles (1.6 trillion km) after 224 years of travel, and still have over 24 trillion miles to go. If Chrysalis ever becomes a reality, it should make it to Proxima b in 400 years, after a year-long acceleration period. It would take another year to decelerate once it reached the planet. Proxima b is 4.24 light-years away, and one light-year equates to about 9.5 trillion km (6 trillion miles). So, the journey from here to Proxima b would cover 39 trillion km (25 trillion miles) through the void. Doing that in 400 years is certainly nothing to sneeze at. Much like the chrysalis of a moth or butterfly blown to epic proportions, Chrysalis was imagined as a 58,000-meter-long (63,430 yards), 2.4 billion-metric-ton (2.65 US tons) cylindrical structure. Its narrower front end would mitigate the risk of impacts from micrometeoroids, space junk, and anything else that could cause a damaging collision. The minimized front end is also meant to reduce stress on the spacecraft as it accelerates and decelerates. Powered by the nuclear fusion of helium and deuterium isotopes, its propulsion system would be a Direct Fusion Drive (DFD)—an engine that is still very much in the conceptual phase. It is supposed to give the spacecraft a simultaneous burst of electrical power and thrust. There is a reason that some floating off-earth habitats in science fiction are shown rotating in space. The front end of Chrysalis—which is imagined to function as the habitat—would have multiple levels in flexible modular shells that fit into each other and constantly rotate on one axis to maintain artificial gravity. From outer to inner, levels would be specialized for food production, ecosystems, communal spaces, housing, gardens, facilities, a warehouse, and the axial core. The Cosmo Dome at the front end is intended to be a bubble of microgravity that provides an incredible view to passengers as they experience weightlessness for a while. So, where do we go from here? Humans have yet to reach Mars (much less Proxima b), and whether such a massive concept can ever translate to reality remains to be seen. However, landing on the Moon was seen as an impossibility only a hundred years ago—the limits of humanity are possibly boundless. You Might Also Like The Do's and Don'ts of Using Painter's Tape The Best Portable BBQ Grills for Cooking Anywhere Can a Smart Watch Prolong Your Life?