Encoded Therapeutics Presents Promising New Data for CNS Gene Therapy Programs at the ASGCT 28th Annual Meeting
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, today announced the results of preclinical studies that underscore the strength of the Company's vector engineering platform and demonstrate continued progress of programs for Angelman syndrome, Alzheimer's disease / tauopathies and chronic pain. Three poster presentations are being highlighted at the 28 th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, LA, May 13 – 17, 2025.
"The data we will present at ASGCT underscore the power and versatility of Encoded's vector engineering platform to target a wide array of validated disease pathways with potential one-time treatments," said Stephanie Tagliatela, co-founder and Chief Scientific Officer at Encoded. "We look forward to sharing these promising results as we advance our maturing pipeline of CNS programs."
Poster Presentation Details
ETX201: An AAV9-based Vectorized miRNA Therapeutic Candidate for Angelman Syndrome. Poster # 547. Presenter, Sirika Pillay, PhD
Date & Time: Tuesday, May 13, 6 – 7:30 PM CT
Location: Poster Hall I2
Recent data further support the potential for a one-time vectorized miRNA therapy for Angelman syndrome. The current findings demonstrate potent, dose-dependent, and selective target engagement, with gene-specific knockdown of UBE3A-ATS and paternal UBE3A upregulation in a genome-wide transcriptomic analysis. ETX201 distributes widely throughout the brain in NHPs, with ETX201 miRNA transcript present in CSF and serum. In addition, ETX201 exhibits dose-dependent upregulation of paternal UBE3A across multiple disease-relevant brain regions, translating to an approximate 20 percent increase in total UBE3A RNA, as well as increased protein levels.
Potent and Specific AAV9-miRNA Candidates Demonstrate Robust Reduction of Microtubule-associated Protein Tau (MAPT) in Non-Human Primates. Poster # 1440. Presenter, Veda Tatavarty, PhD
Date & Time: Wednesday, May 14, 5:30 – 7 PM CT
Location: Poster Hall I2
The first data from NHP studies of Encoded's vectorized miRNA cassettes show MAPT knockdown in key disease-relevant brain regions following AAV9-based delivery, including: up to 34 percent knockdown of bulk MAPT transcript in the cortex, 32 percent in the hippocampus, and up to 24 percent reduction in tau protein levels in the hippocampus. Consistent with these findings, our vectorized miRNA cassettes show high on-target specificity and processing fidelity and demonstrate target engagement in adult humanized tau (hTau) mice, including greater than 50 percent knockdown of MAPT transcript and human tau protein using an IV-delivered tool capsid. Together, these data demonstrate potent and selective knockdown of MAPT and highlight the potential to achieve enhanced target engagement by pairing Encoded's vectorized miRNA cassettes with novel capsids designed for superior biodistribution in the brain.
Developing an AAV-based Gene Therapy for Chronic Pain Through Identification of Potent and Selective Artificial miRNA Candidates to Knockdown SCN9A. Poster # 1925. Presenter, Chao Tai, PhD
Date & Time: Thursday, May 15, 5:30 – 7 PM CT
Location: Poster Hall I2
New data in chronic pain include the first demonstration of preclinical efficacy for Encoded's SCN9A -targeted vectorized miRNA candidates in a rat pain model, showing a durable analgesic effect and up to 70 percent Scn9a knockdown in DRG tissue. These data on our SCN9A -targeting miRNA candidates confirm potent and dose-dependent knockdown observed in vitro, with transcriptome-wide selectivity and favorable miRNA processing characteristics.
About Encoded Therapeutics
Encoded Therapeutics is a clinical-stage genetic medicines company developing potentially one-time, disease-modifying therapies for severe CNS disorders. Our proprietary vector engineering technology combines novel regulatory elements and payloads with AAV vectors, unlocking innovative solutions for intractable CNS conditions. Our lead clinical-stage program, ETX101 for Dravet syndrome, targets the underlying cause of the disorder through selective upregulation of SCN1A for potentially long-lasting benefit. Encoded's second program, ETX201, is a development-stage vectorized miRNA-based gene therapy designed to restore expression of UBE3A in individuals with Angelman syndrome. In parallel, we are advancing potentially best-in-class programs for common CNS conditions, including chronic pain and Alzheimer's disease / tauopathies. Harnessing our proprietary technology platform and expertise, we can efficiently advance programs from discovery through clinical development. Encoded is committed to pioneering breakthrough treatments for CNS disorders. For more information, please visit www.encoded.com.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
Yahoo
4 hours ago
- Yahoo
FDA clears first blood test to help diagnose Alzheimer's disease
The test is for those aged 55 and older who are already exhibiting signs and symptoms of Alzheimer's disease.
San Francisco Chronicle
5 hours ago
- San Francisco Chronicle
New Alzheimer's blood tests make diagnosis easier — but they're not right for everyone
The number of Americans with Alzheimer's disease, the most common cause of dementia in the U.S., is projected to rise significantly in the coming decades. The devastating disease has no cure, but the past several years have brought promising developments in new drug therapies and blood tests that help doctors treat and diagnose the disease. Here's what to know about the blood tests that diagnose Alzheimer's, one of which got full FDA approval in May. How they work Several commercially available blood tests have become available the past few years, most recently from biotech company Fujirebio Diagnostics, which got full approval from the FDA last month. Each test measures slightly different things, but overall they look for abnormal levels of certain proteins — amyloid and tau — in the blood. The accumulation of amyloid and tau proteins in the brain, often called plaques, are hallmark signs of Alzheimer's. If someone has tau plaques in the brain, for instance, some of that will leak out of the brain into the blood, resulting in a blood test result showing higher than normal levels of tau. In patients with symptoms of cognitive impairment, the tests predict an Alzheimer's diagnosis with about 90% accuracy. Why they're important The blood tests are a notable development because they are less expensive and less invasive than other types of testing that doctors have long used to diagnose patients with Alzheimer's. One is a PET scan, which involves injecting a patient with a radioactive tracer that binds to amyloid or tau in the brain so the presence of the proteins can be seen in a scan. It can cost several thousand dollars, compared to several hundred dollars for a blood test. The other is a spinal tap, which is painful or uncomfortable for many people. This method measures different forms of amyloid and tau in the spinal fluid. 'Up until these blood tests came out, a physician was stuck doing a PET scan or spinal tap, so not great options,' said Dr. Frank Longo, a professor of neurology at Stanford Medicine. 'The big breakthrough is finding something to measure in the blood that's about 90% accurate of what's going on in the brain, about as accurate as spinal fluid or a PET scan.' Doctors use the blood test as part of a broader medical evaluation that also includes a patient history, neurological exam and other testing. The blood test can help rule out Alzheimer's as the cause of a patient's cognitive impairment and potentially avoid unnecessary further testing. If someone's blood test comes back normal, for example, they may not have to undergo the PET scan or spinal tap, and their doctor can look into other potential causes of the cognitive impairment. 'That's the biggest practical thing it's doing now,' Longo said. It's important to distinguish Alzheimer's from other types of dementia because there are Alzheimer's treatments that have come out the last few years that help slow the progression of Alzheimer's. But patients must be in relatively early stages of the disease, with mild impairment or mild dementia, to be eligible. Some of the therapies have significant side effects, so patients would not want to start them unless they knew it was Alzheimer's, and not something else, causing the dementia. They're not for everyone The tests are approved only for people who already have symptoms of cognitive impairment and are of a certain age — 55 and over or 60 and over, depending on the test. They are not approved for healthy adults with normal cognition who want to diagnose or rule out Alzheimer's, or who are simply curious if they are at risk for developing Alzheimer's. They must be ordered by a doctor. They're becoming more common, but may not be covered by insurance Some of the blood tests have been available for a year or two, Longo said, but they were under an earlier and more limited type of FDA approval, not the full approval that the agency granted last month to the Fujirebio test. So they are poised to become more common. 'A year ago, most of my colleagues and I were not ordering these,' Longo said. 'I'd say less than 5% or 10% of the time we were ordering these. Now people are starting to order them in symptomatic patients. It's not rare now. They're starting to be recognized more.' Some primary care doctors are starting to order the tests as well, said Dr. Armen Moughamian, medical director of Sutter Health's Ray Dolby Brain Center at CPMC in San Francisco. The center treats patients with memory disorders. 'It's definitely a minority, but I've been seeing it done,' he said. As with many new medical tests, insurers may not cover them yet, but that usually evolves over time. Full FDA approval may help make the case for the tests to be covered, Longo said. 'There is some uncertainty and lack of clarity about whether insurance and Medicare pay,' Moughamian said. 'That creates some hesitancy for providers to order it.' They may one day be used to screen and diagnose healthy people earlier In people with Alzheimer's, amyloid plaques begin to form in the brain as many as 20 years before they show symptoms. Right now, newer therapies for Alzheimer's — like lecanemab and donanemab, which slow the progression of the disease — are approved only for people with symptoms. Those symptoms are mild cognitive impairment or mild dementia, the early stages of symptomatic Alzheimer's. This means people who have not yet developed symptoms, but who have amyloid plaques in the brain, cannot get these treatments. So if a blood test could be used in pre-symptomatic people, it could mean better screening and earlier diagnosis to larger groups of people. 'What we want to push for is earlier diagnosis because new therapies are available when we catch people in earlier stages,' Moughamian said. Moughamian is leading two clinical trials that examine whether drugs that remove amyloid in the brain, donanemab and another drug remternetug, can work in people who have amyloid plaques but have not yet developed symptoms. They are using an Alzheimer's blood test to see whether patients are eligible for the trial.
Chicago Tribune
6 hours ago
- Chicago Tribune
Jim Taylor: What my wife's experience with Alzheimer's has taught me
After several unexplained memory slips, there came a day when my wife, Geri, didn't recognize her own face in the mirror. That's when we knew it was time for her to get checked out. It was 2012, and Alzheimer's was a feared diagnosis. At the time, billions of dollars of investments into research and development had failed to produce treatments that could prevent, slow or cure the disease. Getting a definitive diagnosis would be extremely difficult, but the alternative was living with years of the landscape for Alzheimer's diagnosis and treatment has taken a great leap forward. It is increasingly possible to manage the disease and live a fulfilling life. We have reached a historic moment with the FDA's approval of the first blood biomarker tests for Alzheimer's. This long-awaited breakthrough means physicians can now detect early signs of Alzheimer's — which accounts for 70% of all cases of dementia — using a simple blood test that can be done during a regular check-up with your PET scans remain important for confirming a diagnosis, they are only available at specialized centers, typically in urban medical centers, and they are expensive. Blood biomarker tests now offer an easy first step in the diagnostic journey. They provide fast answers for people experiencing memory problems and can even spot early signs of cognitive decline years before symptoms appear. Without these tests, most people have a long, challenging path to wife Geri's path to diagnosis was anything but simple. In 2012, a neurologist confirmed she had mild cognitive impairment, a common precursor to Alzheimer's. It was a life-altering event. Over the next few years, we knew we had to dig deeper into the cause of her condition, to uncover any potential medical options. Eventually we found a clinical trial for an experimental Alzheimer's drug, and she received a PET scan to determine whether she qualified for the trial. Her brain scan detected amyloid plaque, the telltale sign of Alzheimer's. The diagnosis was difficult to face, but it meant we didn't have to struggle with uncertainty. We could act.I understand the fear that surrounds an Alzheimer's diagnosis, but catching it early helps. Changes in the brain begin years before memory problems become noticeable. The earlier the diagnosis, the more options people have. Research shows that anti-amyloid therapies are more effective when administered earlier: In one clinical trial, patients with early Alzheimer's showed 35% slowing of cognitive decline, compared with those on the placebo. These treatments can help people maintain their independence longer and make the most of their lucid was fortunate to participate in a clinical trial that significantly slowed her disease progression. The seven-year trial period was a game-changer for us. The regular infusions were a source of hope as we saw her benefit from the medication. This precious time allowed Geri to develop coping strategies to manage her disease. Together, we traveled across the country giving talks about living with Alzheimer's disease, and Geri needed very little assistance. We cherished this time together — years made possible because we sought answers early. Following the FDA's landmark approval of blood biomarker tests, the next step is making these tests widely available. Hospitals and health care systems across the country should ensure primary care physicians are aware of these tests and understand how to use them. Public education campaigns can raise awareness with people who have concerns about cognitive impairment and their detection gives people meaningful choices, and most importantly it gives people time. Time to benefit from lifestyle changes, participate in groundbreaking clinical trials, and access treatments when they can make the greatest difference. Although facing a potential Alzheimer's diagnosis is daunting, waiting only limits a person's options. If you're concerned that you or a loved one might have signs of cognitive impairment, please don't stay in the dark. Blood biomarker tests offer real hope in a new era of Alzheimer's care. The sooner we embrace these advances, the more precious time we can preserve.
