Understanding Hepatitis C: Symptoms, Causes, and Treatment Options
Table of Contents
Hepatitis C continues to pose a big global challenge, even compared to Hepatitis E and Hepatitis B. According to estimates, somewhere between 57 and 71.1 million people around the world live with chronic hepatitis C [1, 3]. Each year, roughly 300,000 individuals die from serious complications like advanced liver disease and liver cancer. Experts often label HCV as the top cause of chronic liver disease internationally. That's why it's so essential to raise awareness and support prevention strategies [10].
The virus mostly spreads through blood contact [8]. Years ago, before tests became strict, blood transfusions were a leading cause of infection. But with better screening today, the risk of infection from donated blood is way down. Another issue is the re-use of needles and syringes in healthcare settings or among people who use drugs, as that can directly pass HCV from one person to another. People who inject drugs are especially at risk if they share needles or other equipment [3]. Understanding these pathways helps experts tackle this ongoing public health concern.
Many people with hepatitis C don't have any noticeable symptoms early on [2]. Acute hepatitis C, which is the initial phase, can sneak in without showing any big red flags. On average, it takes about seven weeks for signs to appear, and only around one-third of people see any real problems in the acute stage, such as tiredness, joint aches, or a slight fever. If the virus isn't cleared, the infection might become chronic [9]. About 85% of acute cases progress to chronic hepatitis C if not treated [7]. Over years or decades, this prolonged infection can damage the liver to the point of triggering problems like jaundice (yellowish skin and eyes), fluid buildup in the belly, or weakness.
HCV is basically a thread of RNA, which is genetic material, that specifically attacks liver cells. One of its sneakier moves is dodging the immune system; in most infected individuals, the immune system never fully clears it [4]. What's interesting is that HCV usually doesn't kill liver cells directly. Instead, your immune system's response does much of the damage. Over time, this ongoing battle between the virus and the immune defenses leads to scars in the liver, which can bring on more severe issues down the line [10].
If hepatitis C isn't controlled, it tends to wear down the liver bit by bit. A major concern is cirrhosis, which is when the liver develops a lot of scar tissue, making it tougher for the liver to handle its usual tasks. Cirrhosis can show up after 20 or 30 years of chronic infection, but it can happen sooner, too, depending on other factors. In worse cases, cirrhosis progresses to decompensated liver disease, meaning the liver can't keep up with what your body needs. Another risk is hepatocellular carcinoma (HCC), a dangerous liver cancer that can arise in people with long-term HCV [1, 6].
Certain things can make hepatitis C more severe or speed up its complications. People who get infected later in life sometimes experience liver damage at a faster rate than those infected at younger ages. Gender and race may also influence how quickly the disease develops, though researchers are still finding out exactly why. Drinking alcohol is another major factor—it puts added stress on the liver, possibly pushing liver damage along faster [4]. Knowing these risks helps doctors and patients pinpoint strategies to prevent further harm.
The upside is that awareness, testing, and effective therapies have come a long way lately. Modern antiviral medications can do a great job of clearing the virus and heading off more serious damage [5]. Getting diagnosed early is key, because it gives people a chance to start treatment before cirrhosis or other advanced problems set in. Prevention is also super important. Simple but crucial measures, like sterilizing medical tools, improving safe injection practices, and rigorously screening blood donations, drastically reduce transmission. Around the globe, ongoing medical research aims to curb or even completely eliminate hepatitis C as a health threat in the future.
Hepatitis C is a wide-reaching liver infection that can quietly linger without causing immediate trouble. If nobody catches it early on, the infection can stick around for years, eventually leading to serious complications like cirrhosis and liver cancer. Because HCV mainly travels through blood contact, reducing exposure to infected blood and staying informed about how the virus spreads can make a big difference. Recognizing the symptoms, understanding your own risk factors, and getting tested as needed are all vital steps. Medical advances give hope that, through early detection and better treatments, we can slash new cases, successfully cure existing infections, and lessen the worldwide toll of hepatitis C.
[1] Martinello, M., Solomon, S. S., Terrault, N. A., & Dore, G. J. (2023). Hepatitis C. Lancet (London, England), 402(10407), 1085–1096. https://doi.org/10.1016/S0140-6736(23)01320-X
[2] Simmonds P. (2013). The origin of hepatitis C virus. Current topics in microbiology and immunology, 369, 1–15. https://doi.org/10.1007/978-3-642-27340-7_1
[3] Spearman, C. W., Dusheiko, G. M., Hellard, M., & Sonderup, M. (2019). Hepatitis C. Lancet (London, England), 394(10207), 1451–1466. https://doi.org/10.1016/S0140-6736(19)32320-7
[4] Kohla, M., & Bonacini, M. (2006). Pathogenesis of hepatitis C virus infection. Minerva gastroenterologica e dietologica, 52(2), 107–123. https://pubmed.ncbi.nlm.nih.gov/16557183/
[5] Manns, M. P., & Maasoumy, B. (2022). Breakthroughs in hepatitis C research: from discovery to cure. Nature reviews. Gastroenterology & hepatology, 19(8), 533–550. https://doi.org/10.1038/s41575-022-00608-8
[6] Puchades Renau, L., & Berenguer, M. (2018). Introduction to hepatitis C virus infection: Overview and history of hepatitis C virus therapies. Hemodialysis international. International Symposium on Home Hemodialysis, 22 Suppl 1, S8–S21. https://doi.org/10.1111/hdi.12647
[7] Hoofnagle J. H. (1997). Hepatitis C: the clinical spectrum of disease. Hepatology (Baltimore, Md.), 26(3 Suppl 1), 15S–20S. https://doi.org/10.1002/hep.510260703
[8] Dustin, L. B., Bartolini, B., Capobianchi, M. R., & Pistello, M. (2016). Hepatitis C virus: life cycle in cells, infection and host response, and analysis of molecular markers influencing the outcome of infection and response to therapy. Clinical microbiology and infection : the official publication of the European Society of Clinical Microbiology and Infectious Diseases, 22(10), 826–832. https://doi.org/10.1016/j.cmi.2016.08.025
[9] Delahooke T. E. (2004). Hepatitis C: What is the nature of the problem?. Journal of viral hepatitis, 11 Suppl 1, 5–11. https://doi.org/10.1111/j.1365-2893.2004.00569.x
[10] Boyer, N., & Marcellin, P. (2000). Pathogenesis, diagnosis and management of hepatitis C. Journal of hepatology, 32(1 Suppl), 98–112. https://doi.org/10.1016/s0168-8278(00)80419-5
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Yahoo
2 days ago
- Yahoo
Opinion: Closing Alberta harm-reduction hubs is going backwards
In 2018, a middle-aged man arrived on my patient panel. I will call him Jon. Jon was being treated in our clinic for hepatitis C but did not yet have his own family physician. When I met with him for our first visit, he told me the story of how he had contracted, years prior while incarcerated, this chronic viral infection of the liver. Supplies for tattoos and drug use were contraband at the time, so he and other inmates had shared needles and syringes. By the mid-1990s, studies were advancing our understanding of harm reduction. Based on growing scientific evidence, Canada began to implement programs that were known then as needle exchanges and have since evolved into low-barrier, wraparound services. Many today think of harm reduction as being about drug use only — supervised consumption sites for example. But harm reduction is nothing new. It was historically integral to the HIV/AIDS movement of the late 1980s and early '90s, when communities of men who have sex with men and people who use drugs (PWUD) demanded access to care that helped them reduce their risk of disease transmission. It worked. Today's modern care includes a whole spectrum of public health interventions that sharply decrease the risk of sexually transmitted and blood-borne infections (STBBI). These include things as simple as access to condoms or new syringes. The gold standard is to provide these on demand. Anything less, like having to exchange your used syringe for a new one, increases infections. In Alberta, harm-reduction services are often co-located with primary health care, including pregnancy and wound care as well as treatment for substance use and other mental health disorders. These centres are resource hubs for people who find themselves in the most painful of life's trying moments. Red Deer's Turning Point Society, founded in 1988 in response to the HIV/AIDS epidemic, is the only resource of this kind in all of central Alberta. This week, it was announced that all provincial funding for Turning Point will be discontinued on Nov. 1. What will closures like this mean for Albertans? Without access to wraparound services that include best harm-reduction practices, blood-borne and sexually transmitted infections including hepatitis, HIV, gonorrhea, chlamydia and syphilis will rise. Hospital use and costs will also increase due to more skin, organ and bone infections, more ICU stays, and even amputations. The cost of infections like these is a high price to pay compared to a harm-reduction kit that costs around a dollar. There is a common conception that the neighbourhoods around resource hubs such as Turning Point are more likely to have many discarded needles and syringes or that they are 'honeypots' for drug dealers. Studies have shown these not to occur. In fact, the hubs' cleanup and outreach crews often ensure that the area is more tidy and safer than they would be otherwise. My patient Jon came a long way since I first met him in 2018. He was eventually cured of his hepatitis C. He continued to use injection drugs and was careful to never share equipment — he knew the risks. He relied on our centre — one of Calgary's resource hubs — not only for his drug use supplies but also for getting tested regularly for STBBIs, screened for chronic diseases as he aged, and mental health supports. We have all, in fact, come a long way since the days before harm reduction. Watching, from Calgary, the defunding of central Alberta's resource hub, I fear for other similar centres — places like Alpha House in Calgary and George Spady in Edmonton. What is to become of the places that have always, as the evidence guides, provided low-barrier, wraparound care? That are doing what studies show is safest, healthiest, and most cost-effective. They have not only their clients' and patients' best interests in mind, but also the public's. By shuttering low-barrier and harm-reduction services, we are going backwards, not forwards. As citizens, we must object to the UCP's misguided and regressive restructuring and fight for places like Turning Point, Alpha House, and George Spady. History and science have shown that these are the places doing the work that helps us all. Dr. Bonnie Larson is a clinical assistant professor of family medicine at the University of Calgary. Letters welcome We invite you to write letters to the editor. A maximum of 150 words is preferred. Letters must carry a first and last name, or two initials and a last name, and include an address and daytime telephone number. All letters are subject to editing. We don't publish letters addressed to others or sent to other publications. Email: letters@ Bookmark our website and support our journalism: Don't miss the news you need to know — add and to your bookmarks and sign up for our newsletters here. You can also support our journalism by becoming a digital subscriber. Subscribers gain unlimited access to The Edmonton Journal, Edmonton Sun, National Post and 13 other Canadian news sites. Support us by subscribing today: The Edmonton Journal |The Edmonton Sun.
Vox
08-08-2025
- Vox
How 'the Grim Reaper effect' stops our government from saving lives
is a senior correspondent and head writer for Vox's Future Perfect section and has worked at Vox since 2014. He is particularly interested in global health and pandemic prevention, anti-poverty efforts, economic policy and theory, and conflicts about the right way to do philanthropy. Last summer, the Congressional Budget Office released a report under the unassuming name 'Budgetary Effects of Policies That Would Increase Hepatitis C Treatment.' I read it because I am the type of person who is interested in the budgetary effects of policies that would increase hepatitis C treatment. Embedded in the report, though, was a point that will be important for just about anything the federal government tries to do to save the lives of Americans. Hep C is a nasty viral infection whose effects are, for a virus, unusually long-lasting. Untreated, it causes serious liver damage over the course of decades, leading to much higher rates of cirrhosis and liver cancer, all of which is very expensive to treat. But in the 2010s, a number of extremely effective antivirals, which randomized trials show cure upwards of 95 percent of chronic infections, came on the market. Like most new drugs, these antivirals are under patent and quite expensive; as of 2020, the cost of an eight-to-twelve week course of the drugs, usually enough to cure an infection, was between $11,500 and $17,000. Yet CBO concludes that the drugs are so effective, and the costs of treating patients with hep C who haven't been cured are so massive, that expanding treatment with these drugs reduces federal spending on hep C treatment and associated complications overall. Doubling the number of Medicaid patients getting the drugs would increase federal spending by $4 billion over 10 years. But over the same decade, the federal government would save $7 billion through reduced need for treatments like liver transplants and ongoing care for chronic cases. Put like that, this starts to sound like one of the rarest discoveries in federal budgeting: a free lunch. That means a policy that is good on its own merits (saving lives and preventing debilitating chronic disease) but also saves the government money. But the most interesting part of the report to me comes at the end. 'An increase in hepatitis C treatment could also affect the federal budget in other ways—for example, by leading to improved longevity and lower rates of disability,' the authors note. The latter point is pretty straightforward: If hepatitis C leads to disabilities that make people eligible for disability insurance and subsidized health coverage, then reduced hep C means lower spending on those programs. But (and this is me speculating, so blame me and not the CBO if I'm wrong) that effect is probably swamped by that of 'improved longevity.' Simply put: curing hep C means people live longer, which means they spend more years collecting Social Security, Medicare, and other benefits. That could mean that whatever cost savings the actual hep C treatment produces might be wiped out by the fact that the people whose lives are being saved will be cashing retirement checks for longer. I like to call it the Grim Reaper effect. The US runs a large budget deficit. It also provides far more generous benefits to seniors than to children or working-age adults. Per the Urban Institute's regular report on government spending for children, the ratio of per capita spending on senior citizens to per capita spending on children is over 5 to 1. Put together, the deficit and the elder-biased composition of federal spending implies something that is equally important and macabre: helping people live longer lives will, all else being equal, be bad for the federal budget. In an increasingly aging country, hep C is not the first place where the Grim Reaper effect has been felt, and it won't be the last. I don't have an easy fix for the situation, but it feels important to at least understand. Logan's Run economics One of the first and clearest cases of this longevity dilemma in budgeting came with cigarettes. The history of mass cigarette smoking in the US is surprisingly short. Per the CDC, American adults were only smoking 54 cigarettes annually per capita as of 1900. By 1963, that number had grown to 4,345. The development of automatic rolling machines, milder forms of tobacco, and mass marketing meant millions of working and middle-class Americans became pack-a-day smokers. But while the per capita average floated around 4,000 from the late '40s to the early '70s, it then began a precipitous decline. In 2022, the most recent year for which the Federal Trade Commission released data, Americans bought 173.5 billion cigarettes, or 667 per adult, less than a sixth of the peak, while fewer than 12 percent of American adults now smoke. Cigarettes are, of course, deadly, but they kill with a lag, usually after decades of regular smoking. That meant that in the late 1980s and 1990s, the US started to hit peak cigarette deaths, as adults who came of age during the smoking era started to get lung cancer and emphysema en masse, at numbers that less-addicted subsequent generations wouldn't match. The male death rate from lung cancer peaked in 1990, and the female death rate peaked in 1998. A flurry of economic research at the time tried to make sense of what this meant for the federal budget. Smoking harms your health. But it also shortens your lifespan. A useful 1998 Congressional Budget Office report noted that most research found that, over their lives, smokers spend more in health care costs (including more that goes on the federal tab) than non-smokers, even accounting for their shorter lifespans. But that picture changed once you added in pensions and other non-health spending. Economists John Shoven, Jeffrey Sundberg, and John Bunker in 1989 estimated that the average male smoker saved Social Security $20,000 (about $60,000 today) in benefits not paid. The figure for women, who live longer than men on average but earn less in wages and thus in Social Security, was about half that. 'It seems likely that the Federal budget currently benefits from smoking,' two Congressional Research Service researchers concluded in 1994, when the 'benefits' of early death to Social Security and Medicare were included. Malcolm Gladwell, in a thoughtful 1990 treatment of the problem in the Washington Post, was catchier: 'Not Smoking Could be Hazardous to Pension System.' Decades later, the CBO did a fuller analysis of the budgetary consequences of smoking in the aftermath of the large cigarette tax increase President Obama signed in early 2009 and proposals for further hikes. At first blush, the revenue raised from a cigarette tax should be easy to estimate: multiply annual cigarette sales by the amount of the tax. But obviously raising the price of the good will reduce the amount people buy; one major reason for cigarette taxes, after all, is to deter smoking. The CBO used a price elasticity of -0.3, meaning that a 10 percent increase in cigarette prices reduces the number sold by 3 percent. But the 2012 report was meant to go a step or two further, according to then-director Doug Elmendorf, who explained the backstory in a recent conversation with me. 'The effects of making people healthier are good for those people, obviously, but also perhaps good for the federal budget because the federal government pays for a lot of health care. If you're healthier, you don't need so much health care.' But at the same time, 'It was clear that if people were healthier, they would live longer, and that could have budgetary costs. It wasn't obvious offhand what the balance of those effects would be.' The 2012 CBO report tried to put all these effects together: the effect of lower smoking on reducing health-care spending (including government-funded spending) due to a healthier population, the effect on Social Security and other benefit spending from resulting longer lifespans, the effect of lower smoking rates on wages, and tax revenue from those wages. (The latter is often not included in formal CBO scores, as it tips closer to 'dynamic' scoring where the effect of legislation on the overall economy is included.) Over the first 10 years after a hike in the cigarette tax, they found that having a healthier population was more of a blessing than a curse, budget-wise. The health effects of a cigarette tax hike reduced federal health spending by over $900 million over a decade, even after accounting for people living longer and claiming more years of Medicare. By contrast, retirement programs only spent $183 million more because people lived longer. Swamping all that was a $2.9 billion increase in tax revenue from a healthier population capable of working and earning more. But that's just the 10-year effect. As the decades pass, the effect of longevity would grow and grow. First, Medicare costs would start to rise, as the cost of a longer-lived population began to swamp the cost savings of that population being healthier overall. (Even people who've been healthy for a long time can run up major health spending at the end of their now longer lives.) Social Security costs would keep rising, too. Fifty years in, these costs would overwhelm the benefits, and the cigarette tax's health effects would start costing the budget, on average. The point isn't 'cigarette taxes are good' or 'cigarette taxes are bad.' The point is that even a policy that saves lives isn't necessarily a slam dunk from the hard-eyed perspective of budget policy. Recent years provided a possibly even darker example. In 2022, the Medicare Trustees pushed back the date they expected the program's Hospital Insurance Trust Fund to be depleted by two years. They had several reasons, but a major one was that Covid-19 had killed hundreds of thousands of Medicare patients prematurely. Not only that, but 'Medicare beneficiaries whose deaths were identified as related to COVID had costs that were much higher than the average Medicare beneficiary prior to the onset of the pandemic.' Put another way: Covid killed off Medicare's sickest, and most expensive, enrollees. That meant the program was left with an overall healthier population, which by itself lowered medical costs by 2.9 percent in 2021. Similarly, a paper by a team of health economists earlier this year estimated that the 1.4 million excess deaths in the US due to Covid had the net effect of boosting the Social Security trust fund to the tune of $156 billion. That represented $219 billion in benefits that no longer needed to be sent, minus $44 billion in lower payroll tax revenues and $25 billion in new benefits to surviving family members. It all reminds one of Logan's Run, in which people are killed off upon hitting age 30 lest they take up too many of society's resources. That movie is a dystopia — but as a budget proposal, it'd score very well. It's good to save lives, actually The economists and agencies doing this math are, of course, only doing their jobs. We need to know what government programs will cost over the near- and long-run. These effects on health and life and death matter to those calculations. 'Members of Congress regularly thought that we were ghoulish for talking about how, if people live longer, there'll be higher benefits for Social Security,' Elmendorf recalls. 'But it's not ghoulish. Obviously, we want to live longer and members of Congress should try to help all Americans live longer. CBO's job — an analyst's job in general — is just to be honest about the likely effects.' But the fact that increased human longevity on its own worsens the budget picture should lead to some reflection. For one thing, it suggests that sometimes we should embrace policies simply because they're the right thing to do, even if they don't pay for themselves. Recall the hepatitis C treatments that prevent expensive long-term expenses for Medicaid, but might add on new costs by extending the benefits' lifespans. It's possible that, upon taking the latter into account, expanding access to hep C drugs costs the government money on net. It's a free lunch no longer. That's not a reason not to embrace the policy, though. Lots of things the government does cost money. The military doesn't pay for itself. K–12 schools don't pay for themselves. Smithsonian Museums don't pay for themselves. That doesn't mean those aren't important functions that it makes sense to put some of our tax dollars toward. Hep C treatment, I think, fits in that list, even if it's not literally free from a budget standpoint. Congress should also allow agencies like the CBO to do more to symmetrically account for the positive budgetary effects of longevity, along with the negatives. People who live longer, after all, often earn wages in those new years of life, wages that generate income and payroll tax revenues for the federal government. Moreover, people at the end of their careers are earning more money and hence paying more taxes than young people, meaning life extension helping people in their 50s and 60s might be especially good for tax revenue. The problem is that the CBO generally considers 'how many workers paying taxes are there' to be an economic effect and only considers it in special 'dynamic' scores of legislation, in which the economic consequences of them are taken into account. Dynamic scoring has been a topic of great controversy for decades, going back at least to the Bush II administration, but the rule Congress sets for CBO on when to use dynamic scoring results in CBO applying dynamic scoring very rarely in practice. A middle ground option, though, would be something called 'population change' scoring, in which CBO considers the direct effects of a change in the population (through longer lifespans, say, or immigration) on the level of employment and tax revenue, without doing a full, more complicated dynamic score. That would make its accounting of the effects of longer lives less biased: the budgetary benefits would be counted alongside the costs. We should also consider the aspects of our budget situation that make the longevity effect a reality. One is the US's long-standing, bipartisan choice to run massive budget deficits, even during relative boom times. One arithmetic consequence of that choice is that it makes the continued existence of every American a net loss for the country's books. That's not the main reason to avoid large deficits during booms, but it's a somewhat toxic byproduct all the same. The other aspect driving this effect is the choice to invest government resources very heavily in seniors relative to other age groups. This is due in large measure to the US choice to provide universal health care for seniors but not other age groups, and due to our lack of investment in very young children and working-age adults compared to other rich nations. There is no law of nature saying the US has to weigh its priorities that way. As long as we do, the numbers will imply that it's better for the budget for people to die before they get old.
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
