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6 days ago
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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
Yahoo
17-06-2025
- Business
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Clearside Biomedical Announces Multiple Presentations to be Featured at the Clinical Trials at the Summit Meeting
- Broad Use of Suprachoroidal Injection Platform Highlighted Across Retinal Indications - - CLS-AX Combines the Flexibility of Anti-VEGF Therapies with the Long-Lasting Benefits of a Tyrosine Kinase Inhibitor - ALPHARETTA, Ga., June 17, 2025 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD) ('Clearside' or the 'Company'), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), announced today that the use of its SCS delivery platform will be featured in multiple presentations at the Clinical Trials at the Summit (CTS) Meeting on June 21, 2025 in Las Vegas, Nevada. Victor Chong, MD, MBA, Chief Medical Officer and EVP, Head of Research and Development, commented, 'Our proprietary SCS Microinjector® platform is an established, in-office solution for delivering vision-preserving therapies to the back of the eye. The pipeline of promising clinical programs using our platform underscores its versatility across multiple treatment modalities. We remain committed to redefining care with therapies that offer both extended durability and flexible dosing to address serious retinal diseases.' Presentations Related to Clearside and its Development Partners Title: Suprachoroidal CLS-AX for nAMD: Phase 3 Program UpdatePresented by: Sobha Sivaprasad, MD, Professor of Retinal Clinical Research, Consultant Ophthalmologist, Moorfields Eye Hospital, London, UK Partner: REGENXBIOTitle: Update on One-Time Suprachoroidal ABBV-RGX-314 for the Treatment of DRPresented by: Anna Abolian, OD, Senior Medical Director, Clinical Development Lead, REGENXBIO Inc. Partner: Aura BiosciencesTitle: Bel-sar - a New Treatment Paradigm in Choroidal Melanoma: Update on the Global Phase 3 CoMpass TrialPresented by: Jennifer Lim, MD, Professor of Ophthalmology, Director, Retina Service,Department of Ophthalmology and Visual Sciences, University of Illinois School of Medicine About CLS-AX (axitinib injectable suspension) Clearside is developing CLS-AX as a longer-acting therapy for the treatment of retinal diseases. CLS-AX (axitinib injectable suspension) is a proprietary suspension of axitinib for suprachoroidal injection. Axitinib is a tyrosine kinase inhibitor (TKI), currently approved as an oral tablet formulation to treat advanced renal cell carcinoma, that achieves pan-VEGF blockade, directly inhibiting VEGF receptors-1, -2, and -3 with high potency and specificity. Clearside believes this broad VEGF blockade may have efficacy advantages over existing retinal therapies by acting at a different level of the angiogenesis cascade and may benefit patients who sub-optimally respond to current, more narrowly focused anti-VEGF therapies. Suprachoroidal injection of this proprietary suspension of axitinib has demonstrated meaningful potential in Phase 1/2a and Phase 2b wet AMD clinical trials in which CLS-AX was well tolerated and demonstrated a positive safety profile. With suprachoroidal administration of axitinib, there is the potential to achieve prolonged duration and targeted delivery to affected tissue layers by compartmentalizing axitinib behind the retina, thereby limiting drug exposure to the front of the eye. About Clearside's Suprachoroidal Space (SCS®) Injection Platform and SCS Microinjector® Clearside's patent protected, proprietary suprachoroidal space (SCS®) injection treatment approach offers unprecedented access to the back of the eye, where sight-threatening disease often occurs. The Company's unique platform is inherently flexible and intended to work with established and new formulations of medications. Clearside's patented SCS Microinjector® can deliver a wide variety of drug candidates into the suprachoroidal space, providing targeted delivery to potentially improve efficacy and compartmentalization of medication to reduce or eliminate toxic effects on non-diseased cells. The SCS Microinjector is comprised of a syringe with a custom-designed hub and two 30-gauge hollow microneedles of varying lengths, each approximately one millimeter, optimizing insertion and suprachoroidal administration of drugs. About Clearside Biomedical, Inc. Clearside Biomedical, Inc. is a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®) to improve patient outcomes. Clearside's SCS injection platform, utilizing the Company's patented SCS Microinjector®, enables an in-office, repeatable, non-surgical procedure for the targeted and compartmentalized delivery of a wide variety of therapies to the macula, retina, or choroid to potentially preserve and improve vision in patients with sight-threatening eye diseases. Clearside is developing its own pipeline of small molecule product candidates for administration via its SCS Microinjector. The Company's lead program, CLS-AX (axitinib injectable suspension), is in development for the treatment of neovascular age-related macular degeneration (wet AMD). Planning for a Phase 3 program is underway. In addition, Clearside is evaluating various small molecules for the potential long-acting treatment of geographic atrophy (GA). Clearside developed and gained approval for its first product, XIPERE® (triamcinolone acetonide injectable suspension) for suprachoroidal use, which is available in the U.S. through a commercial partner. Clearside also strategically partners its SCS injection platform with companies utilizing other ophthalmic therapeutic innovations. For more information, please visit or follow us on LinkedIn and X. Cautionary Note Regarding Forward-Looking Statements Any statements contained in this press release that do not describe historical facts may constitute forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as 'believe', 'expect', 'may', 'plan', 'potential', 'will', and similar expressions, and are based on Clearside's current beliefs and expectations. These forward-looking statements include statements regarding the clinical development of Clearside's product candidates, and the potential benefits of CLS-AX and XIPERE®, Clearside's suprachoroidal delivery technology and Clearside's SCS Microinjector®. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements. Risks and uncertainties that may cause actual results to differ materially include uncertainties inherent in the conduct of clinical trials, Clearside's reliance on third parties over which it may not always have full control, Clearside's ability to raise additional capital, and other risks and uncertainties that are described in Clearside's Annual Report on Form 10-K for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission (SEC) on March 27, 2025, and Clearside's other periodic reports filed with the SEC. Any forward-looking statements speak only as of the date of this press release and are based on information available to Clearside as of the date of this release, and Clearside assumes no obligation to, and does not intend to, update any forward-looking statements, whether as a result of new information, future events or otherwise. Investor and Media Contacts:Jenny Kobin Remy Bernarda ir@ Source: Clearside Biomedical, 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
Yahoo
05-06-2025
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REGENXBIO REPORTS NEW POSITIVE FUNCTIONAL DATA FROM PHASE I/II AFFINITY DUCHENNE® TRIAL OF RGX-202
RGX-202 demonstrating consistent evidence of positively changing disease trajectory for Duchenne All dose level 2 participants exceeded external natural history controls on all functional measures Biomarker data demonstrate consistent, robust microdystrophin expression and transduction levels across all treated ages One new participant aged 2 years at dosing had expression level at 118.6% compared to control Favorable safety profile continues with no serious adverse events or adverse events of special interest observed Webcast to be held at 8:00 a.m. today ROCKVILLE, Md., June 5, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial. Updates include positive functional, safety and biomarker data for RGX-202, REGENXBIO's potential best-in-class, investigational gene therapy for Duchenne muscular dystrophy. The functional data demonstrate consistent benefit among dose level 2 participants at 9 and 12 months following treatment with RGX-202. "Today's findings support the potential of RGX-202 to positively change the disease course for Duchenne and meaningfully benefit patients living with this degenerative disease. At the same dose being used in the pivotal trial, RGX-202 participants exceeded natural history across all key measures, including the North Star Ambulatory Assessment, which is striking," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "We are particularly encouraged by the outperformance observed in older patients. The continued, positive data further strengthen our commitment to rapidly bring this potentially transformative therapy to market and support our planned Biologics License Application submission under accelerated approval in mid-2026." "These findings suggest that the microdystrophin expression observed with RGX-202 is leading to meaningful functional improvements, even in individuals with DMD who are expected to experience functional decline," said Aravindhan Veerapandiyan, M.D., of Arkansas Children's Hospital. "These Phase I/II results, demonstrating functional improvements and favorable safety profile, underscore the potential of RGX-202 as a treatment option for individuals with DMD. It is both encouraging and essential to have innovative therapies that can help preserve muscle integrity and substantially delay disease progression. I'm enthusiastic about the continued development of RGX-202 and the promise it holds for the Duchenne community." AFFINITY DUCHENNE Phase I/II Interim Data Updates (data cut: May 7, 2025) Functional DataToday, REGENXBIO announced positive interim functional results from the first five participants, aged approximately 6 to 12 years at dosing, receiving RGX-202 at dose level 2 (2x1014 GC/kg). Based on these patients' age at dosing and baseline function, four out of five patients are expected to be in the decline phase of their disease trajectory. Results were measured against external natural history controls that were strictly matched for age and baseline function1. RGX-202 continues to demonstrate evidence of positively impacting disease trajectory with dose level 2 participants demonstrating improved performance on North Star Ambulatory Assessment (NSAA) and timed function tests (Time to Stand, 10 Meter Walk-run, Time to Climb), exceeding external natural history controls. At 9 months, RGX-202 participants demonstrated improvement in function and exceeded external controls on all measures. On NSAA, RGX-202 recipients improved an average of 4 points from baseline and 4.8 points compared to natural history. [Figure 1] Four out of the five participants reached 12-months post dosing. Results at 12 months are similar to those seen at 9 months. RGX-202 participants demonstrated improved performance on timed function tests and NSAA, exceeding external natural history controls at 12 months. All participants within this cohort demonstrated improvement on all timed function tests compared to baseline. On NSAA, RGX-202 recipients improved an average of 4.5 points from baseline and 6.8 points compared to natural history. [Figure 2] Additionally, dose level 2 participants' timed task velocity changes exceeded minimal clinically important difference (MCID) benchmarks at 12 months, a measure referenced by the FDA in the approval of an available gene therapy. Biomarker DataBiomarker data from the Phase I/II study continues to support consistent, high expression and transduction of RGX-202 microdystrophin. New data from an additional patient, aged 2 at dosing, had a microdystrophin expression level of 118.6% compared to control. The primary endpoint in the pivotal phase of AFFINITY DUCHENNE is the proportion of participants whose RGX-202 microdystrophin expression is ≥10% at Week 12. RGX-202 was appropriately localized to the sarcolemma, demonstrating that the differentiated construct with the inclusion of the C-Terminal (CT) domain is appropriately targeting the muscle. Mean at 12 Weeks (min, max) Dose Level 1 1x1014 GC/kg Dose Level 2 2x1014 GC/kg Age range (number with data) 4-7 (2) 8-11 (1) 1-3 (2) 4-7 (2) 8-11 (5) RGX-202 Microdystrophin % normal control (Western Blot) 60.6 (37.8, 83.4) 10.4 120.5 (118.6, 122.3) 54.3 (31.5, 77.2) 39.7 (20.8, 75.7) VCN copies/nucleus (qPCR) 9.8 (7.4,12.1) 5.4 24.8 (20.4, 29.1) 30.1 (4.9, 55.4) 17.8 (12.0,30.7) Positive Fibers % (Immunofluorescence) 79.32 34.6 82.12 50.3 (29.4, 71.1) 45.7 (21.3,70.6) RGX-202 also continues to demonstrate the highest reported vector genome copies (4.9-55.4) measured by qPCR across approved or investigational gene therapies. Safety and Tolerability Data RGX-202 was well tolerated with no serious adverse events (SAEs) and no AEs of special interest (AESIs). Common drug-related AEs included nausea, vomiting and fatigue. All are typically anticipated with gene therapy administration. A proactive, short-course immune modulation regimen in combination with a differentiated construct and industry-leading product purity levels of more than 80% full capsids may contribute to a favorable safety profile for RGX-202. Phase I/II AFFINITY DUCHENNE Trial: RGX-202 Treatment Emergent Adverse Events Dose Level 1 Dose Evaluation (1x1014 GC/kg) Dose Level 2 Dose Younger Boys (2x1014 GC/kg) Dose Level 2 Evaluation / Expansion (2x1014 GC/kg) Total n=13 Age Range (number dosed) 4-11 (n=3) 1-3 (n=3) 4-11 (n=7) All Ages SAE 0 0 0 0 AESI Central or peripheral neurotoxicity 0 0 0 0 Drug-induced liver injury 0 0 0 0 Thrombocytopenia 0 0 0 0 Myocarditis 0 0 0 0 Myositis 0 0 0 0 AFFINITY DUCHENNE Pivotal TrialREGENXBIO is enrolling participants in the pivotal portion of the Phase I/II/III AFFINITY DUCHENNE trial of RGX-202. The trial is expected to enroll approximately 30 patients aged 1+ in the U.S. and Canada by 2025, with more than half already enrolled to support the pivotal dataset. The pivotal trial is expected to support a Biologics License Application (BLA) submission using the accelerated approval pathway in mid-2026. REGENXBIO expects to share top-line data in the first half of 2026 and plans to include biomarker, functional, and safety data in its submission. Webcast DetailsREGENXBIO will host a webcast featuring REGENXBIO management and Dr. Veerapandiyan to discuss today's developments at 8:00 a.m. EST. The live webcast can be accessed here and in the Investors section of REGENXBIO's website at An archived replay of the webcast will be available for approximately 30 days following the presentation. 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. In preclinical studies, the CT domain has been shown to protect the muscle from contraction-induced stress and improve its ability to repair itself. Additional design features 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 using REGENXBIO's 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@ ____________________________________ 1 For NSAA, the EC matched subjects of one treated subject did not have data at Month 9 or Month 12. The delta was based on the mean of RGX-202 participants' changes from baseline minus stratum-based mean change from baseline of EC matched participants. 2 One sample could not be evaluated View original content to download multimedia: SOURCE REGENXBIO Inc. Sign in to access your portfolio
Yahoo
02-06-2025
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REGENXBIO to Host Webcast Discussing Interim Functional Data from the Phase I/II AFFINITY DUCHENNE® Trial of RGX-202
Event will feature Aravindhan Veerapandiyan, M.D., principal investigator of the AFFINITY DUCHENNE® trial ROCKVILLE, Md., June 2, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a webcast to discuss new interim functional data from the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, the company's next-generation investigational gene therapy for the treatment of Duchenne muscular dystrophy. The webcast will feature AFFINITY DUCHENNE principal investigator Aravindhan Veerapandiyan, M.D., Arkansas Children's Hospital. Webcast detailsTitle: AFFINITY DUCHENNE® Trial of RGX-202: Phase I/II Interim Functional DataDate/Time: Thursday, June 5, 2025, at 8:00 a.m. EDT Access: The live webcast can be accessed here and in the Investors section of REGENXBIO's website at An archived replay of the webcast will be available for approximately 30 days following the presentation. 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 Contacts: Dana CormackCorporate Communicationsdcormack@ Investors:George E. MacDougallInvestor RelationsIR@ View original content to download multimedia: SOURCE REGENXBIO Inc.
Yahoo
21-05-2025
- Business
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Coave Therapeutics Establishes New Scientific Advisory Board to Accelerate Innovation in Genetic Medicines for Eye and CNS Diseases
Paris, France – May 21, 2025 – Coave Therapeutics ('Coave'), a company pioneering the future of genetic medicines, today announces the evolution of its Scientific Advisory Board (SAB). This renewed SAB reflects Coave's strategic shift from a platform-focused approach to the development of a therapeutic pipeline, as the Company advances multiple gene therapy programs. The SAB brings together internationally recognized leaders in adeno-associated viral (AAV) vector biology, gene therapy development for ophthalmology and central nervous system (CNS) diseases, and innovative approaches to extra-hepatic gene delivery. The SAB members include: Olivier Danos, PhD (Chair) – Chief Scientific Officer at REGENXBIO (Rockville, MD, USA) Dr. Danos is a pioneer in gene therapy with over 30 years of academic and industrial leadership, including roles at Biogen, Genethon and University College London, and now leads REGENXBIO's scientific strategy Robin Ali, PhD – Professor of Human Molecular Genetics at King's College London (London, UK) and Founder of MeiraGTx Dr. Ali is a world-leading expert in gene therapy for retinal diseases and a pioneer of translational research advancing vision restoration therapies Aravind Asokan, PhD – Professor at Duke University School of Medicine and Director of the Danaher Beacon for Gene Therapy Innovation (Durham, NC, USA) Dr. Asokan specializes in AAV vector biology and synthetic virology, developing engineered AAVs to enable next-generation gene therapies Juliette Hordeaux, DVM, PhD – Chief Scientific Officer at GEMMA Biotherapeutics (Philadelphia, PA, USA) and formerly Executive Director of Translational Research at the Gene Therapy Program, University of Pennsylvania (Philadelphia, PA, USA) Dr. Hordeaux is a recognized leader in the development of AAV-based CNS-targeted gene therapies, focused on translating preclinical innovations to clinical-stage programs Federico Mingozzi, PhD – Chief Executive Officer of Nava Therapeutics (Philadelphia, PA, USA) Dr. Mingozzi brings extensive expertise in gene therapy R&D and translation, having previously served as Chief Scientific Officer at Spark Therapeutics and earlier led programs at Genethon, INSERM and the Children's Hospital of Philadelphia Amy Pooler, PhD – Senior Vice President, Research and Development at Life Edit Therapeutics (Durham, NC, USA), previously Vice President, Head of Research at Sangamo Therapeutics (Brisbane, CA, USA) Dr. Pooler has extensive experience in translational neuroscience and genetic medicine, with leadership roles in developing CNS-targeted therapies at Sangamo and Life Edit The SAB members will provide strategic scientific and clinical guidance to support Coave's R&D initiatives, leveraging its ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform to unlock improved precision and safety in genetic medicine. Their combined expertise strengthens Coave's leadership in developing AAV-based therapeutics targeting diseases beyond the liver, including the eye and CNS. Lolita Petit, CSO of Coave Therapeutics commented, 'We are absolutely delighted to welcome this exceptional group of world-leading experts to our Scientific Advisory Board. Their collective expertise across AAV biology, gene therapy development and translational research will be invaluable as we continue to enhance our ALIGATER™ platform and advance our therapeutic pipeline. Their support reinforces our commitment to delivering next-generation genetic medicines with improved precision and safety and accelerates our mission to transform the lives of patients with serious unmet medical needs.' *** Photos of SAB members available on request About ALIGATER™Coave's proprietary ALIGATER™ (Advanced Vectors-Ligand Conjugates) platform is a breakthrough technology addressing key limitations in the delivery of genetic payloads to extra-hepatic tissues, including limited tissue specificity, delivery efficiency and safety. ALIGATER™ enables conjugation of targeting ligands, such as small molecules, peptides, or antibody fragments, on AAV or non-viral vectors, offering superior delivery efficiency, tissue specificity and safety profile for a broad range of diseases. Importantly, the platform streamlines the manufacturing process by avoiding prior AAV capsid modifications. These capabilities will enable Coave to develop best-in-class gene therapies designed for specific indications. About Coave Therapeutics Coave Therapeutics is a genetic medicine company pioneering the development of innovative solutions to enhance the precision, safety, efficacy and manufacturability of genetic medicines. With its proprietary ALIGATER™ platform, Coave is at the forefront of addressing challenges in gene therapy delivery to extra-hepatic tissues, creating a robust pipeline targeting CNS, neuromuscular and eye diseases. Headquartered in Paris, France, Coave Therapeutics is backed by leading international life sciences investors. For more information about the science, pipeline, and people, please visit and follow us on LinkedIn. CONTACTS Coave Therapeutics Rodolphe Clerval, CEO contact@ MEDiSTRAVA Sylvie Berrebi, Mark Swallow coavetx@ in to access your portfolio
