Latest news with #MuscularDystrophyAssociation
Yahoo
16 hours ago
- General
- Yahoo
Firefighters hope to ‘Fill the Boot' in support of Muscular Dystrophy Association
PORTLAND, Ore. (KOIN) — Drivers along a busy road near Washington County will notice firefighters with boots in hand. The off-duty crews with Tualatin Valley Fire & Rescue are taking part in the annual 'Fill the Boot' fundraiser, asking motorists to donate money to help support the Muscular Dystrophy Association (MDA). Firefighters will be along Beaverton-Hillsdale Highway and Canyon Road, and the on-ramps and off-ramps to Highway 217 in Beaverton between 8 a.m. and 4 p.m. on Friday, June 6. Copyright 2025 Nexstar Media, Inc. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.
Yahoo
12-04-2025
- Entertainment
- Yahoo
Colman Domingo has wished Eric Dane well amid his ‘Euphoria' co-star's health battle
Colman Domingo has wished Eric Dane well amid his fellow 'Euphoria' actor's health battle. The star's co-star on the show was recently diagnosed with amyotrophic lateral sclerosis (ALS), and the 55-year-old actor has now opened up about his shock at hearing Eric's sad news. He told People about how he learned of his friend's diagnosis the same day it was shared with the public: "I haven't been able to speak with him yet.' Colman added about how he is backing Eric: 'I just got the news yesterday too, and I'm wishing him well. I look forward to working with him.' Colman plays Ali in 'Euphoria' – a mentor to and sponsor for Zendaya's drug-addled character Rue, while Eric plays troubled Cal Jacobs, the patriarch of the Jacobs family. 'Euphoria' was renewed for a third season in February 2022, and production began just two months ago. Eric, 52, revealed his ALS diagnosis in an exclusive interview with People on Thursday (10.04.25.) He said: 'I have been diagnosed with ALS. 'I am grateful to have my loving family by my side as we navigate this next chapter. 'I feel fortunate that I am able to continue working and am looking forward to returning to the set of 'Euphoria' next week.' The rare and progressive disease causes paralysis of the muscles, affecting speech, walking, and other motor functions. Eric has two children, Billie Beatrice, 15, and Georgia Geraldine, 13, with his wife, Rebecca Gayheart. ALS, also known as Lou Gehrig's disease, is an illness that typically leads to progressive loss of muscle control, including the ability to speak, eat and breathe. The Muscular Dystrophy Association estimates those diagnosed with the condition usually live three to five years after their diagnosis, though some patients survive for decades. Other celebrities diagnosed with ALS include Aaron Lazar, John Driskell Hopkins and Stephen Hawking. Eric is set to return to Euphoria for its third season, with filming scheduled to resume on Monday (14.04.25.) He also recently completed work on the Amazon crime drama series 'Countdown'. Fans can expect more drama for his character, Cal Jacobs, in the new 'Euphoria' series as Eric has hinted at a potential redemption arc following his character's arrest at the end of season two. He told Variety about the upcoming series: 'There's gonna be redemption... I can't imagine Cal's life from solitary confinement. It's tough to work Cal into the storyline when he's behind bars.'
Buzz Feed
11-04-2025
- Entertainment
- Buzz Feed
Here's Everything We Know About Eric Dane's ALS Diagnosis — And What It Means For 'Euphoria' Season 3
Eric Dane has been diagnosed with amyotrophic lateral sclerosis. The 52-year-old actor shared his diagnosis with People yesterday, saying: 'I have been diagnosed with ALS. I am grateful to have my loving family by my side as we navigate this next chapter.' For context, ALS (sometimes referred to as Lou Gehrig's disease) is a rare neurodegenerative disease that affects nerve cells in the brain and spinal cord, causing progressive paralysis of the muscles. There is no cure for the disease, which, over time, causes people to lose the ability to walk, speak, eat, and breathe independently. Notably, ALS does not typically affect a person's thinking ability. Every person's experience with ALS is unique, but according to the Muscular Dystrophy Association, the life expectancy after someone is diagnosed can be between three to five years, although some people live for decades. Eric has starred in many popular shows and movies, from Grey's Anatomy to Burlesque. However, he's now probably best known for his role in Euphoria as Cal Jacobs, the father of Jacob Elordi 's Nate Jacobs. Shooting for Season 3 of the hit HBO show is currently underway, and in his statement, Eric was pleased to confirm his involvement. 'I feel fortunate that I am able to continue working and am looking forward to returning to set of Euphoria next week,' he told People. 'I kindly ask that you give my family and I privacy during this time.' Eric is married to Rebecca Gayheart, and they share two teenage daughters: Billie Beatrice, 15, and Georgia Geraldine, 13. As you may know, Rebecca filed to divorce Eric in 2018. However, the couple remained close, and last month, she requested to dismiss her petition. On April 9, days before Eric's ALS diagnosis was made public, Rebecca gave an update on their marriage, telling E! News: 'We are best of friends. We are really close. We are great co-parents. We really figured out the formula to staying a family, and I think our kids are benefiting greatly from it, and we are as well.' 'I think it's important to not look at a relationship that ends as a failure. It's just a season. It wasn't a failure. It was a huge success,' she went on. 'We were married for, I mean, we are still married, but together for 15 years, and we had two beautiful kids, so I think that's a successful relationship, and that's how we look at it.'
Yahoo
19-03-2025
- Business
- Yahoo
New Novartis Phase III data demonstrate meaningful efficacy and safety results of intrathecal onasemnogene abeparvovec in broad patient population with SMA
New Novartis Phase III data demonstrate meaningful efficacy and safety results of intrathecal onasemnogene abeparvovec in broad patient population with SMA Treatment with investigational OAV101 IT led to statistically significant 2.39-point improvement on the HFMSE vs. 0.51 points in sham control arm Safety findings were consistent in both treatment-naïve and treatment-experienced patients These new data indicate the potential for OAV101 IT to be a clinically meaningful treatment option for a broad range of patients with SMA Novartis plans to file applications with regulatory agencies in H1 2025 Basel, March 19, 2025 – Novartis announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad population of patients aged two to <18 years with spinal muscular atrophy (SMA). In the Phase III STEER study, treatment with OAV101 IT led to a statistically significant 2.39-point improvement on the Hammersmith Functional Motor Scale Expanded (HFMSE), a gold standard for SMA-specific assessment of motor ability and disease progression, vs. 0.51 points in the sham control arm (P=0.0074).1-5 In the Phase IIIb STRENGTH study, treatment with OAV101 IT in patients who have discontinued treatment with nusinersen or risdiplam demonstrated stabilization of motor function over 52 weeks of follow-up. These data will be presented during the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference held in Dallas, Texas, from March 16–19, 2025. The results add to the growing body of evidence within the OAV101 IT development program, which has evaluated a broad population of over 170 patients with SMA, spanning a total of over 6.4 years across the STEER, STRENGTH and Phase I/II STRONG studies.6 'In the STEER study evaluating treatment-naïve patients, OAV101 IT demonstrated a statistically significant improvement in motor function across a broad SMA population,' said Crystal Proud, M.D., Pediatric Neurologist and a Principal Investigator at Children's Hospital of the King's Daughters. 'These results – paired with those in the STRENGTH study – support the potential for OAV101 IT to be a meaningful treatment option for people living with SMA with a goal of maintaining or improving motor function through a one-time therapy.' OAV101 IT is an investigational gene replacement therapy designed to directly address the genetic root cause of the disease by replacing the nonworking SMN1 gene with a single dose. It is the first investigational gene replacement therapy to provide clinical benefit in both children and young adults with SMA with a favorable safety profile, underscoring the potential of this therapy to provide patients the opportunity to avoid repeated treatment administration. 'The data presented today from our OAV101 IT program reinforce our belief in this therapy, which has the potential to have a meaningful impact on a broad range of people with SMA through its continuous benefit via a one-time dose,' said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer, Novartis. 'Together with patients, caregivers and healthcare professionals, we are committed to continuing to advance our mission to lead innovation in SMA treatment and broaden therapy options with our gene replacement therapies.' STEER StudyIn the registrational STEER study, efficacy and safety were studied in treatment naïve patients with SMA Type 2, aged two to less than 18 years who were able to sit, but had never walked independently. Results for OAV101 IT were compared against a sham control, a procedure designed to mimic the administration of an investigational drug, without delivering any active treatment. One hundred twenty-six (126) patients received either OAV101 IT (n=75) or a sham procedure (n=51). Mean (range) age at dosing was 5.89 (2.1–16.6) years in the treatment group and 5.87 (2.4–14.2) years in the sham arm. At the end of the 52-week period, all eligible patients had received both OAV101 IT and the sham procedure. Key findings: The trial met its primary endpoint of change from baseline to 52 weeks in HFMSE score, with OAV101 IT demonstrating a statistically significant 2.39-point improvement on the HFMSE vs 0.51 points in the sham group (overall difference, 1.88 points; P=0.0074). All secondary endpoints consistently favor OAV101 IT, despite not achieving statistical significance due to the pre-planned multiple testing procedure. The overall incidence of adverse events (AEs), serious AEs (SAEs), and AEs of special interest was similar between both groups. The most common AEs for both groups in the STEER study were upper respiratory tract infection and pyrexia. The most frequent SAEs were pneumonia and vomiting for the OAV101 IT group and pneumonia and lower respiratory tract infection for the sham group. Instances of transaminase increases were infrequent; most were low-grade and transient. There were no cases of Hy's law. STRENGTH StudyThe open label Phase IIIb STRENGTH study evaluated the safety, tolerability and efficacy of OAV101 IT in patients with SMA aged two to less than 18 years who had discontinued treatment with nusinersen or risdiplam. In the study, 27 patients were enrolled with a mean (range) age of 7.4 years (2.4-17.7). Mean duration of prior risdiplam and nusinersen treatment were 2.98 and 4.32 years, respectively. Key findings: OAV101 IT demonstrated a favorable safety profile that was consistent with STEER study. The motor endpoint of efficacy, HFMSE, demonstrated stabilization for the overall study population over 52 weeks. The increase from baseline to 52 weeks in HFMSE least squares (LS) total score was 1.05. All patients in the STRENGTH study experienced at least one AE. The most frequent AEs were common cold, pyrexia and vomiting. A total of 13 patients (48.1%) experienced AEs considered to be related to study treatment. No AEs leading to death or study discontinuation were reported. About OAV101 ITIntrathecal onasemnogene abeparvovec (OAV101 IT) is an investigational, one-time gene replacement therapy for patients with spinal muscular atrophy (SMA). Novartis has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of adeno-associated virus 9 (AAV9) gene replacement therapy for the treatment of all types of SMA; an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene replacement therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Généthon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA. About Spinal Muscular AtrophySpinal muscular atrophy (SMA) is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.11-13 The severity of SMA varies across a spectrum of types that generally correspond to the number of copies the individual has of the SMN2 gene, which produces a small fraction (~10%) of functional SMN protein compared with SMN1.12 Loss of motor neurons cannot be reversed, so patients with SMA with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities.13 DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as 'potential,' 'can,' 'will,' 'plan,' 'may,' 'could,' 'would,' 'expect,' 'anticipate,' 'look forward,' 'believe,' 'committed,' 'investigational,' 'pipeline,' 'launch,' or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise. About Novartis Novartis is an innovative medicines company. Every day, we work to reimagine medicine to improve and extend people's lives so that patients, healthcare professionals and societies are empowered in the face of serious disease. Our medicines reach nearly 300 million people worldwide. Reimagine medicine with us: Visit us at and connect with us on LinkedIn, Facebook, X/Twitter and Instagram. References Weber C, et al. 'Reading the palm' – A pilot study of grip and finger flexion strength as an outcome measure in 5q spinal muscular atrophy. Brain and Development. 2024;46(5):89-198 Coratti G et al. Determining minimal clinically important differences in the Hammersmith Functional Motor Scale Expanded for untreated spinal muscular atrophy patients: An international study. Eur J Neurol. 2024;31:e16309. Revised Hammersmith Scale for spinal muscular atrophy: A SMA specific clinical outcome assessment tool - PMC. O'Hagen JM, Glanzman AM, McDermott MP, Ryan PA, Flickinger J, Quigley J, et al. An expanded version of the Hammersmith Functional Motor Scale for SMA II and III patients. Neuromuscular disorders: NMD. 2007;17(9–10):693–7. Epub 2007/07/31. Hammersmith Functional Motor Scale and Motor Function Measure-20 in non ambulant SMA patients - PubMed. Mazzone E, De Sanctis R, Fanelli L, Bianco F, Main M, van den Hauwe M, et al. Hammersmith Functional Motor Scale and Motor Function Measure-20 in non ambulant SMA patients. Neuromuscular disorders: NMD. 2014;24(4):347–52. Epub 2014/02/05. 10.1016/ Basil T. Darras. Intravenous and Intrathecal Onasemnogene Abeparvovec Gene Therapy in Symptomatic and Presymptomatic Spinal Muscular Atrophy: Long-Term Follow-Up Study. Presented at the 29th International Annual Congress of the World Muscle Society, October 8–12, 2024, Prague, Czechia. Available at: Accessed February 2025. Finkel RS, et al. J Neuromuscul Dis. 2023;10(3):389-404. Available at: Accessed November 2024. Accessed November 2024. Anderton RS and Mastaglia FL. Expert Rev Neurother. 2015;15(8):895–908. Finkel RS, et al. Neurology. 2014;83(9):810-7. Lorson CL, et al. Hum Mol Genet. 2010;(15):111-8. # # # Novartis Media RelationsE-mail: Novartis Investor RelationsCentral investor relations line: +41 61 324 7944E-mail: Sign in to access your portfolio
Yahoo
19-03-2025
- Health
- Yahoo
Precision BioSciences Presents Preclinical Efficacy and Durability Data on PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy (DMD) at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference
- Potential first-in-class gene editing approach designed for dystrophin gene correction leading the body to produce a functional dystrophin protein applicable for majority of DMD patients (up to ~60%) - PBGENE-DMD restored dystrophin protein expression and significantly improved muscle function over time while demonstrating long-term durability in an in vivo DMD disease model – - PBGENE-DMD dystrophin gene correction observed in muscle satellite stem cells suggesting potential for permanent functional benefit – DURHAM, N.C., March 19, 2025--(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, including novel gene excision programs for high unmet need genetic diseases, today announced the presentation of preclinical data for its PBGENE-DMD development program for the treatment of Duchenne muscular dystrophy (DMD) during an oral presentation at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 16-19, 2025 in Dallas, TX. "While there has been much-needed progress in the DMD field recently, patients still lack treatments that offer significant durable functional improvement. These PBGENE-DMD preclinical data compellingly demonstrate the potential for gene correction in the body to natively produce near full length dystrophin and restore muscle function while offering durability through the editing of muscle satellite stem cells," said Dr. Cassie Gorsuch PhD, Chief Scientific Officer. "By precisely and directly excising the genetic root cause for DMD patients with defects between exon 45 and 55, our approach could provide more durable outcomes for these patients compared to microdystrophin gene therapies. Furthermore, this therapeutic approach is applicable for up to 60% of DMD patients, far more than exon skipping approaches currently approved or in development. The results presented today demonstrate the therapeutic potential of PBGENE-DMD to improve the lives of patients with DMD and support future clinical development of the first widely applicable gene editing approach." Presentation Details: Title: ARCUS-Mediated Excision of Exons 45-55 Leads to Functional Del45-55 Dystrophin and Restoration of Skeletal Muscle-Function for the Treatment of DMDOral Presentation Date and Time: Wednesday, March 19, 2025, 8:00 AM CTPoster Number: O159 In preclinical data to be presented today, PBGENE-DMD demonstrated significant functional improvement in a humanized DMD mouse model by employing two complementary ARCUS nucleases delivered in a single AAV to excise exons 45-55 of the dystrophin gene. This approach aims to restore the body's native production of a functional dystrophin protein that more closely resembles normal dystrophin than synthetic microdystrophins. This dystrophin gene correction approach which involves editing muscle satellite stem cells potentially enhances durability and functional outcomes compared to synthetic approaches. Since up to 60% of DMD cases are caused by defects between exons 45 and 55, this approach is more broadly applicable for the majority of DMD patients than exon skippers. Key findings from the study include: Functional dystrophin protein production: PBGENE-DMD restored the body's ability to produce a functional dystrophin protein across multiple muscles, including heart, diaphragm, and skeletal muscles at levels expected to provide therapeutic benefit. Enhanced Muscle Resilience: Treated mice exhibited a 66% improvement in resistance to eccentric injury, an indicator of enhanced muscle resilience, compared to untreated diseased counterparts. Long-Term Functional Improvement: In mice treated with PBGENE-DMD the maximum force output (MFO), a critical functional metric, reached up to 93% of the MFO in healthy control mice with improvement observed in PBGENE-DMD-treated mice between 3 and 6 months. Durable Outcomes: PBGENE-DMD-edited dystrophin mRNA transcript was detected in PAX7+ cells, a marker for muscle satellite stem cells, suggesting potential for durable therapeutic effects compared to standard gene therapy approaches. 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 like in the DMD program). About Duchenne Muscular Dystrophy (DMD) DMD is a genetic disease caused by mutations in the dystrophin gene that prevent production of the dystrophin protein. Dystrophin stabilizes the cell membrane during muscle contraction to prevent damage, and the absence of intact dystrophin protein leads to inflammation, fibrosis, and progressive loss of muscle function and mass. Over time, children with DMD will develop problems walking and breathing, eventually leading to death in their second or third decade of life due to progressive cardiomyopathy and respiratory insufficiency. DMD occurs in 1 in 3,500 to 5,000 male births with an estimated prevalence of 15,000 patients and incidence of 550 patients/year in the United States alone. Unmet need for DMD patients remains high as there are no approved therapies with curative intent that can drive durable and significant functional improvements. 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 clinical development and expected safety, efficacy and benefit of our product candidates (including PBGENE-DMD); the unique design of PBGENE-DMD utilizing two ARCUS nucleases delivered using a single AAV to restore native function to the dystrophin protein by excising exons 45-55 as a treatment; the suitability of PBGENE-DMD for the treatment of Duchenne Muscular Dystrophy by directly editing the genetic root cause of the disease to potentially provide more durable outcomes for the majority of patients compared to gene therapies and exon skippers currently approved or in development; the potential of PBGENE-DMD as a first-in-class gene editing approach targeting up to 60% of DMD patients; the expected timing of regulatory processes (including filings such as IND's and CTA's and studies for PBGENE-DMD and the acceptance of these filings by regulatory agencies); the robust safety, tolerability and efficacy signals observed through preclinical evaluation in transgenic mouse models, human cell models of DMD; the translatability of preclinical models to human clinical trials; the key advantages of ARCUS and its key capabilities and differentiating characteristics ; expectations about operational initiatives, strategies, and further development of PBGENE-DMD; expectations about achievement of key milestones; the assessment of whether to partner or internally develop the PBGENE-DMD clinical program; and anticipated timing of patient dosing and clinical data. 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,", "suggest", "strive," "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 September 30, 2024, 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. 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