Latest news with #MichaelKelly
Yahoo
10-07-2025
- Business
- Yahoo
REGENXBIO Announces Publication of Preclinical Results Demonstrating Functional Benefits of Novel Microdystrophin Construct in RGX-202 Investigational Gene Therapy for Duchenne Muscular Dystrophy
Construct including CT domain demonstrated higher levels of microdystrophin protein, increased muscle force, and improved resistance to damage in mice lacking dystrophin REGENXBIO's next-generation investigational gene therapy, RGX-202, is the only microdystrophin construct that includes the CT domain Findings support the positive functional data seen in Phase I/II AFFINITY DUCHENNE® trial of RGX-202 ROCKVILLE, Md., July 10, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the publication of preclinical results comparing a microdystrophin gene therapy construct that included the C-terminal (CT) domain to a microdystrophin construct without the CT domain. The results, which were published in peer-reviewed journal Molecular Therapy Methods and Clinical Development, showed that the microdystrophin with the CT domain improved functional benefit compared to the microdystrophin without, supporting the potential of RGX-202 to drive functional improvements in patients with Duchenne Muscular Dystrophy. RGX-202 is the only investigational or approved microdystrophin gene therapy candidate for the treatment of Duchenne muscular dystrophy (Duchenne) that includes the CT domain, a key portion of dystrophin, making it the closest to naturally occurring dystrophin. "We specifically designed RGX-202 differently from other gene therapies with the goal of providing improved outcomes for patients, and this research further validates the potential therapeutic advantage of adding the CT domain and its importance in preventing the muscle breakdown associated with functional decline in Duchenne," said Olivier Danos, Ph.D., Chief Scientific Officer of REGENXBIO. "The positive interim results we've seen in the Phase I/II AFFINITY DUCHENNE® trial are reinforced by this preclinical research demonstrating how the novel construct of RGX-202 protects against muscle damage and supports the potential for durable, functional benefit for patients." "AAV gene therapy holds great promise for Duchenne, and the community is in need of treatment options that have the potential to improve function and quality of life for patients," said Michael Kelly, Ph.D., Chief Scientific Officer, CureDuchenne. "The CT domain is a critical part of the large DMD gene and these preclinical results highlight its role in muscle health. With the science behind the novel microdystrophin construct of RGX-202, combined with the interim safety and efficacy profile seen to date in clinic, I am very encouraged by the potential of RGX-202 to be a meaningful, differentiated gene therapy for the community." In this paper, titled "Enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain," two AAV vectors, one encoding a microdystrophin protein with the CT domain and one encoding an otherwise equivalent microdystrophin protein without the CT domain, were evaluated across three studies in mdx mice, a preclinical model of Duchenne, to measure muscle force, protein levels, and protection from contraction-induced muscle injury. Compared to the microdystrophin without the CT domain, the microdystrophin that included the CT domain was found to be maintained at higher levels in transduced muscles, recruited the dystrophin-associated protein complex more effectively to the muscle membrane, and increased muscle force and resistance to damage in mice lacking dystrophin. These are key factors in supporting the preservation of muscle health, as muscle damage leads to disease progression in Duchenne. These findings indicate that incorporation of the CT domain enhances the microdystrophin design by allowing for higher levels of microdystrophin to accumulate in the muscle – primarily attributed to the longer half-life of the extended microdystrophin – and may improve the functional benefit of microdystrophin gene replacement. Interim results from the Phase I/II AFFINITY DUCHENNE clinical trial of RGX-202 reported in June 2025 show that RGX-202 demonstrated consistent evidence of positively changing the disease trajectory of patients with Duchenne and a favorable safety profile. REGENXBIO is enrolling participants in the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial of RGX-202 and expects to submit a Biologics License Application (BLA) using the accelerated approval pathway in mid-2026. About RGX-202RGX-202 is a potential best-in-class investigational gene therapy designed for improved function and outcomes in Duchenne. RGX-202 is the only gene therapy approved or in late-stage development for Duchenne with a differentiated microdystrophin construct that encodes key regions of naturally occurring dystrophin, including the C-Terminal (CT) domain. Additional design features such as codon optimization may potentially improve gene expression, increase protein translation efficiency and reduce immunogenicity. RGX-202 is designed to support the delivery and targeted expression of microdystrophin throughout skeletal and heart muscle using the NAV® AAV8 vector and a well-characterized muscle-specific promoter (Spc5-12). RGX-202 is manufactured by REGENXBIO using its proprietary, high-yielding NAVXpress® suspension-based platform process. About Duchenne Muscular DystrophyDuchenne is a severe, progressive, degenerative muscle disease, affecting 1 in 3,500 to 5,000 boys born each year worldwide. Duchenne is caused by mutations in the Duchenne gene which encodes for dystrophin, a protein involved in muscle cell structure and signaling pathways. Without dystrophin, muscles throughout the body degenerate and become weak, eventually leading to loss of movement and independence, required support for breathing, cardiomyopathy and premature death. ABOUT REGENXBIO is a biotechnology company on a mission to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the field of AAV gene therapy. REGENXBIO is advancing a late-stage pipeline of one-time treatments for rare and retinal diseases, including RGX-202 for the treatment of Duchenne; clemidsogene lanparvovec (RGX-121) for the treatment of MPS II and RGX-111 for the treatment of MPS I, both in partnership with Nippon Shinyaku; and surabgene lomparvovec (ABBV-RGX-314) for the treatment of wet AMD and diabetic retinopathy, in collaboration with AbbVie. Thousands of patients have been treated with REGENXBIO's AAV platform, including those receiving Novartis' ZOLGENSMA®. REGENXBIO's investigational gene therapies have the potential to change the way healthcare is delivered for millions of people. For more information, please visit FORWARD-LOOKING STATEMENTSThis press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2024, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the SEC and are available on the SEC's website at All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Zolgensma® is a registered trademark of Novartis AG. All other trademarks referenced herein are registered trademarks of REGENXBIO. Contacts:Dana CormackCorporate Communicationsdcormack@ Investors:George E. MacDougallInvestor RelationsIR@ View original content to download multimedia: SOURCE REGENXBIO Inc. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Sunday World
27-06-2025
- Sunday World
Councillors shocked at footage of naked man outside asylum centre
Video footage of the incident shot by bystanders from the footpath and from passing vehicles has been widely spread on social media since it happened on Saturday, June 14. District councillors in Wicklow have called for greater lines of communication with the local engagement team after a man was recently found running naked outside an asylum centre in the town. Video footage of the incident shot by bystanders from the footpath and from passing vehicles has been widely spread on social media since it happened on Saturday, June 14. The footage captures a completely nude male running along the middle of the road outside the IPAS centre at the Grand Hotel. He appears to be in a disorientated and agitated state. The Grand Hotel in Wicklow town. Photo: Michael Kelly News in 90 Seconds - June 27th Speaking during the June meeting of Wicklow Municipal District, independent councillor John Snell called on his district colleagues to work collectively to help alleviate concerns when these incidents happen. He stressed the need for the Department of Children, Equality, Disability, Integration and Youth to provide information about what happened to the individual in question and asked newly elected cathaoirleach Graham Richmond to write to the relevant Minister seeking clarity on the matter. He also raised concern about how the footage appeared online before elected representatives were informed about what happened and suggested a better strategy is needed to monitor certain comments and activities online. In agreement councillor Paul O'Brien issued a word of caution about how the issue is dealt with and cited the difficulty in trying to manage social media accounts and open forums. Councillor Gail Dunne added it was important that residents in the town feel safe and that such incidents don't become a common reoccurrence. The male in question has since been arrested for public order offences and was brought to Wicklow Garda Station, where he was held in custody. He was charged with several offences, which are due before the courts. He is currently receiving care at Newcastle Hospital after being sectioned under the mental health act. Funded by the Local Democracy Scheme
Washington Post
24-06-2025
- Sport
- Washington Post
Navy's new athletic director has Annapolis roots and global ambition
Growing up with a parent who graduated from the Naval Academy, Michael Kelly attended countless football games at Navy-Marine Corps Memorial Stadium during his youth. As of Monday, Kelly became responsible for overseeing the Midshipmen football program as well as every other sport as Navy's new athletic director. The graduate of St. John's College High in Washington introduced himself to his new co-workers on his second day on the job during an informal gathering Tuesday at packed Akerson Theater, where the football team typically watches game film. Sitting in the front row were his parents, including his father, Dennis, a member of Navy's class of 1967. 'To come back to the DMV, it means a lot to me. Growing up in the area, playing basketball at St. John's for coach [Joe] Gallagher and being part of the whole scene here,' Kelly said. 'Back then, going to games when David Robinson was playing [basketball] here, seeing when Maryland and Georgetown were all great on the basketball side, to me it's all that. It's nostalgic to be home.' Kelly replaces Chet Gladchuk, who announced March 31 he would be retiring after a 24-year tenure highlighted by a dramatic resurgence in football prosperity. Among Gladchuk's most consequential final duties was taking a lead role in the national search for his replacement. Also figuring prominently in the search was Chad Chatlos, a 1993 Navy graduate who serves as managing director for TurnkeyZRG, the executive recruiting firm the academy retained for the process, which lasted close to 10 weeks and comprised 23 candidates, according to Gladchuk. 'In the final analysis, it was across the board — the farther we got, the closer we got to a decision, it became clearer and clearer and clearer that there was one gentleman that just absolutely stood out amongst all,' Gladchuk said of Kelly. 'We looked at his background, and it was extraordinary. He's done everything.' Kelly most recently was the athletic director at South Florida, where among his most notable accomplishments over seven years was almost doubling the department's budget to $100 million. The Bulls expanded to 21 varsity sports under his watch and claimed 21 team championships in the American Athletic Conference, where Navy is a current member in football. Kelly's previous jobs included serving as the chief operating officer of the College Football Playoff, managing site selection for the national championship game and the national semifinals, and as senior associate commissioner for the ACC from 2007 through 2013, playing a prominent role in negotiating the ACC's television rights contract in 2010. 'I think the job at the ACC and then also my job at the CFP kind of helps me — not only helps us navigate major business deals as it relates to where we might move Army-Navy, where we put Navy-Notre Dame, things of that nature,' Kelly said, 'because that's kind of what I was involved in and then certainly the whole television aspect of it. How can we maximize the value of the tradition and the excellence of Navy football and really all Navy sports?' The Midshipmen are coming off a 10-3 record in football capped by a dramatic victory in the Armed Forces Bowl over Oklahoma. The Midshipmen earned double-digit wins for the sixth time in program history in the second season under Coach Brian Newberry, who sat in the second row during Kelly's introductory address inside the football facility Tuesday. 'For me it's been all about meeting as many people as I possibly can here internally and certainly talking to donors and sponsors and others, too,' Kelly said. 'So that's really what I'm focused on this week, is to just introduce myself, tell them how excited I am to be here and being able to be ready, spending this whole month mainly focused on that so we can hit the ground running when the true summer camps start and getting ready for what I expect will be a very successful fall for Navy football and for all of our fall sports.'
Associated Press
14-06-2025
- Business
- Associated Press
Stay in Your Lane Debuts Industry-First AI-Verified Dating App for Safer, Authentic Connections
Stay in Your Lane launches a free dating app featuring AI verification, live video chat, profile videos, and real-time authenticity checks for safe connections. United States, June 14, 2025 -- Revolutionizing Dating Apps with Advanced Safety Protocols Michael Kelly, Owner of Stay in Your Lane, proudly announces the launch of a premium dating app designed to prioritize safety, privacy, and authenticity. Leveraging advanced AI, live video features, and user-centric functionality, the app addresses key flaws in traditional dating platforms, offering users a secure and genuine online dating experience. Online dating has grown in popularity but continues to be undermined by fake profiles, catfishing, and privacy concerns. Stay in Your Lane offers a groundbreaking solution by placing user safety and authenticity at the heart of its platform. The app's AI-driven facial recognition system verifies every user to ensure profiles are genuine and match the individual behind them. This eliminates fake accounts and promotes accountability. To further enhance the experience, Stay in Your Lane integrates a live video chat feature, allowing users to connect face-to-face before meeting in person. This added layer of security ensures members can confidently interact, knowing they're speaking to the actual person behind the profile. Michael Kelly, Owner of Stay in Your Lane, emphasized the app's transformative vision: 'Our product is the authenticity of our members. We've reimagined the online dating experience to make it safer, more secure, and truly authentic. This app addresses the problems users face while promoting meaningful connections.' Enhancing User Experience with Innovative Features Stay in Your Lane combines advanced safety measures with unique tools to create an unparalleled online dating experience. Michael Kelly shared the app's approach to building a trustworthy community: 'We ask our members to be patient as we grow our membership. After all, it's free to use while we focus on delivering a platform that prioritizes authenticity and safety.' Addressing Gaps in Current Platforms Stay in Your Lane takes a proactive stance in solving problems overlooked by traditional dating platforms. Its free access model—complete with messaging and video chat—lowers barriers to entry while reinforcing trust through mandatory verification and real-time connections. The app monetizes premium services such as boosts and super likes to enhance visibility and engagement, ensuring the platform remains accessible while supporting continued growth. These combined measures establish Stay in Your Lane as a leader in the next generation of online dating. A Community Focused on Authentic Relationships Stay in Your Lane fosters a space where users can prioritize genuine, meaningful relationships. By ensuring profiles are authentic and interactions are secure, the app empowers members to build connections without distractions or concerns about safety. Michael Kelly explained the platform's mission: 'We've created Stay in Your Lane to stand apart from the crowd. This isn't just another dating app—it's a solution to the safety and authenticity issues that have frustrated users for years.' The Future of Online Dating Starts Here Stay in Your Lane is available for download now, offering a premium experience for those seeking secure and authentic connections. The app's free features, combined with optional premium services, ensure accessibility while fostering a growing community of verified members. To learn more or join the community, visit About Stay in Your Lane Stay in Your Lane is a premium dating app founded by Michael Kelly, focused on creating a safer, more authentic online dating environment. With advanced AI verification, live video chat, privacy-first features, and real-time authenticity protocols, the app addresses key challenges in online dating while fostering genuine connections. Media Contact Michael Kelly Owner, Stay in Your Lane Contact Info: Name: Michael Kelly Email: Send Email Organization: Stay in Your Lane Website: Release ID: 89162305 In case of encountering any inaccuracies, problems, or queries arising from the content shared in this press release that necessitate action, or if you require assistance with a press release takedown, we urge you to notify us at [email protected] (it is important to note that this email is the authorized channel for such matters, sending multiple emails to multiple addresses does not necessarily help expedite your request). Our responsive team will be readily available to promptly address your concerns within 8 hours, resolving any identified issues diligently or guiding you through the necessary steps for removal. The provision of accurate and dependable information is our primary focus.
New York Times
10-06-2025
- Sport
- New York Times
‘I understand': After suspension for betting, A's pitcher Michael Kelly tries to start over
ANAHEIM, Calif. — It took Michael Kelly until he was 31 years old to establish himself as a big league pitcher. There were eight years in the minor leagues — five before he advanced past A-ball. Another season in independent ball, where he had a 5.34 ERA. Then three more years in the minor leagues, punctuated by a couple of call-ups. Advertisement It was a slog, a fight. It meant everything just to make it to where he was in 2024 — a 2.59 ERA with the then-Oakland A's, spanning 31 1/3 innings. He'd finally earned a spot. Then, on June 4 of last year, he got popped for betting just $99.22 on baseball, the bets made when he was a minor leaguer. He was suspended for a year. His betting history was made public in a press release by the league. This was now what he was best known for. And his career was, once again, put on hold. 'Regardless of what it looks like, how it was, I think fans and teams and players, everyone needs to trust the integrity of the game,' Kelly said, saying that he accepted responsibility for the actions that led to his suspension. 'So, because of that, I understand why they did what they did, and they went with the suspension. 'They sent the message, and everyone needs to trust the integrity of the game. And I understand that completely.' Kelly was suspended along with three other players — Diamondbacks reliever Andrew Saalfrank, Padres pitcher Jay Groome and Phillies infielder José Rodriguez. Padres infielder Tucupita Marcano was banned from the sport that same day for betting on Pirates games, while he was on their big-league roster. Saalfrank, who has spent time in the big leagues, was brought back by his organization and is currently pitching in the Arizona Complex League. Both Groome and Rodriguez were non-tendered by their respective clubs and are free agents. Kelly's bets were minuscule, all things considered. They occurred over 12 days, from October 5, 2021, to October 17, all while he was a minor leaguer in the Astros' system. He placed 10 bets involving nine big-league games. Three of the games he bet on involved the Astros. He won a net of $28.30 on his bets. 'I really didn't dwell on it much,' Kelly said, when asked how he can reconcile all this happening over such a relatively small amount of money. 'I focused on what I can control. Being where my feet were, and staying in the moment. I'm not one to look back and dwell on something.' Advertisement Kelly didn't want to discuss how receiving his suspension played out, noting that he'd rather focus on the present. He said he spent his time creating a training facility with two former big leaguers, Josh Tomlin and Steven Hill. The facility was designed to train kids and coach young players. After the initial shock, Kelly heard from many of his teammates. Family supported him, he said. He started the facility because he knew he wanted to stay involved in the game. But still, he missed being on the field. When he returned, he said, 'There was some excitement. Warm welcomes, for sure.' When asked what he wanted to prove in his career, following his suspension, Kelly responded that 'For me, it's just showing my love for the game. … Competing and being the best competitor that I can be. A professional for guys on the team, guys I'm playing against.' Kelly returned to the Athletics on June 5, the first day he was eligible. The team recently lost 20-of-21 games and was in desperate need of bullpen arms. He pitched in his first game back on the roster, throwing 1 1/3 scoreless frames against the Twins. It meant a lot to Kelly. He had to get through a lot just to step on a major league mound for the first time three years ago. He understood why he had to miss the last year, as embarrassing as it was. What he also recognizes, however, is that it was about more than 99 dollars and 22 cents. It was about betting on baseball, and the serious risk to the integrity of the sport that gambling can cause. But he can't control that, not now. So he focuses on what he can impact: getting back out there, pitching again after that self-inflicted mistake. It was so important to him. 'It was everything that I was hoping it was going to be,' Kelly said. 'Just being out there and competing and helping the team, and doing my part. It was everything I could hope for.'
