RGX-121 would be the first and only potential one-time commercially-available therapy designed to directly address the underlying genetic cause of Hunter syndrome, if approved Commercial launch plans remain on track REGENXBIO plans to present updated pivotal data during the ICIEM meeting in September 2025 ROCKVILLE, Md. , Aug. 18, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug... Read More

RGX-202 in Duchenne muscular dystrophy on track for topline results 1H 2026 and BLA submission mid-2026 Pivotal trial enrollment accelerated, expected to complete in October 2025 ahead of previous guidance Clemidsogene lanparvovec (RGX-121) on track to be first gene therapy and one-time treatment for MPS II; FDA inspections completed successfully Pre-license inspection (PLI) of in-house manufacturing facility, quality... Read More

A pivotal Phase IIb/III clinical trial using suprachoroidal delivery for treatment of diabetic retinopathy will be initiated New Phase II ALTITUDE ® trial data demonstrate a durable safety and efficacy profile observed through two years with a single, in-office injection of surabgene lomparvovec in subjects with non-proliferative diabetic retinopathy REGENXBIO to receive $100 million upon first subject dosed in the Phase... Read More

ROCKVILLE, Md. , July 31, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Thursday, August 7 , at 8:00 a.m. ET to discuss its financial results for the second quarter ended June 30, 2025 , and operational highlights. Listeners can register for the webcast via this link . Analysts wishing to participate in the question and answer session should access the live call by... Read More

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... Read More

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... Read More

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... Read More

$150 million secured at closing extends cash runway into early 2027 REGENXBIO retains additional potential non-dilutive funding opportunities, including monetization of Priority Review Voucher (PRV) and milestones from AbbVie ROCKVILLE, Md. , May 19, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the closure of a non-dilutive, limited recourse royalty bond agreement of up to $250 million with Healthcare... Read More

PARAMUS, N.J. , May 14, 2025 /PRNewswire/ -- NS Pharma, Inc. (NS Pharma), a subsidiary of Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) announced today that the U.S. Food and Drug Administration has accepted for review the Biologics License Application (BLA) submission by REGENXBIO Inc. (REGENXBIO; Headquarters: Rockville, Maryland , USA; CEO: Curran M. Simpson , NASDAQ: RGNX) for RGX-121 ( clemidsogene lanparvovec) , a... Read More

FDA assigns PDUFA target action date of November 9, 2025 RGX-121 on track to be the first gene therapy and one-time treatment for MPS II Partner Nippon Shinyaku to lead commercialization upon potential approval REGENXBIO to lead commercial manufacturing and supply chain ROCKVILLE, Md. , May 13, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the U.S. Food and Drug Administration (FDA) has accepted for... Read More

RGX-202 in Duchenne muscular dystrophy on track for BLA submission mid-2026 Pivotal trial more than half enrolled, with completion expected in 2025 Additional Phase I/II functional data expected 1H 2025 Initiating commercial supply manufacturing in Q3 2025 FDA acceptance of Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121, partnered with Nippon Shinyaku) expected in May 2025 Pivotal data evaluating... Read More

ROCKVILLE, Md. , May 8, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, taking place in New Orleans , May 13 through 17, 2025 . The presentations highlight REGENXBIO's leading end-to-end capabilities across research and early development, late-stage clinical development, and gene therapy manufacturing. Presentations... Read More

ROCKVILLE, Md. , May 6, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following investor conferences: BofA Securities Health Care Conference 2025 Presentation: Tuesday, May 13, 2025 at 5:00 p.m. PT Location: Las Vegas, NV RBC Global Healthcare Conference Fireside Chat: Tuesday, May 20, 2025 at 11:30 a.m. ET Location: New York, NY H.C. Wainwright BioConnect Fireside Chat:... Read More

ROCKVILLE, Md. , April 30, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Monday, May 12 , at 4:30 p.m. ET to discuss its financial results for the first quarter ended March 31, 2025 , and recent operational highlights. Listeners can register for the webcast via this link . Analysts wishing to participate in the question and answer session should use this link . A... Read More

Positive biomarker data in patient aged 1-3 add to consistent, robust microdystrophin and transduction levels across all treated ages Patient aged 3 years at dosing had expression level at 122.3% compared to control With a differentiated novel construct and proactive short course immune modulation regimen, RGX-202 continues to demonstrate encouraging safety profile with no SAEs or AESIs Phase III portion of AFFINITY DUCHENNE... Read More

Biologics Licensing Application (BLA) for clemidsogene lanparvovec (RGX-121) submitted and on track for potential FDA approval 2H 2025; strategic partnership with Nippon Shinyaku aims to expand potential access and commercial opportunity in MPS II and MPS I Pivotal trial of RGX-202 for Duchenne Muscular Dystrophy progressing rapidly; enrollment completion expected in 2025 with BLA filing in mid-2026 AbbVie-partnered retinal... Read More

ROCKVILLE, Md. , March 10, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced new interim biomarker data from the Phase I/II portion of the AFFINITY DUCHENNE ® trial of RGX-202 for the treatment of Duchenne muscular dystrophy will be presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place in Dallas, TX , March 16-19, 2025 . Title: RGX-202, an investigational... Read More

ROCKVILLE, Md. , March 4, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the closing of its previously announced strategic partnership with Nippon Shinyaku. Under the terms of the agreement, REGENXBIO and Nippon Shinyaku will develop and commercialize RGX-121 ( clemidsogene lanparvovec) for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome and RGX-111 for Mucopolysaccharidosis I (MPS I),... Read More

ROCKVILLE, Md. , Feb. 26, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Thursday, March 13 , at 4:30 p.m. ET to discuss its financial results for the fourth quarter and full year ended December 31, 2024 , and recent operational highlights. Listeners can register for the webcast via this link . Analysts wishing to participate in the question and answer session should... Read More

ROCKVILLE, Md. , Feb. 25, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following investor conferences: TD Cowen's 45th Annual Health Care Conference Fireside Chat: Wednesday, March 5 at 1:10pm ET Location: Boston, MA Leerink Partners Global Healthcare Conference Fireside Chat: Tuesday, March 11 at 9:20am ET Location: Miami, FL Barclays 27 th Annual Global Healthcare Conference... Read More

ROCKVILLE, Md. , Jan. 30, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced data from its RGX-121 (clemidsogene lanparvovec) program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be shared at the 21 st Annual WORLD Symposium ™ 2025, taking place in San Diego, CA February 3-7, 2025 . The following presentations include an encore of the topline results from... Read More

REGENXBIO to receive $110 million upfront, potential milestone payments of up to $700 million and meaningful double-digit royalties on net sales, and lead manufacturing Nippon Shinyaku to lead commercialization of first potential gene therapies for Mucopolysaccharidosis II (MPS II) and Mucopolysaccharidosis I (MPS I) in U.S. and Asia REGENXBIO retains rights to RGX-121 Priority Review Voucher (PRV) with potential accelerated... Read More

Alignment achieved with FDA on AFFINITY DUCHENNE ® pivotal program and access to accelerated approval; BLA expected in 2026 Pivotal trial of RGX-202 is enrolling ambulatory patients aged 1 and above with first patient dosed Phase I/II data show RGX-202 recipients exceeding external natural history and established benchmarks... Read More

Event will feature Aravindhan Veerapandiyan, M.D., principal investigator of the AFFINITY DUCHENNE ® trial and Michael Kelly , PhD, Chief Scientific Officer of CureDuchenne ROCKVILLE, Md. , Nov. 14, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a webcast to discuss the AFFINITY DUCHENNE ® pivotal program and new clinical data, including the first functional data from the ongoing Phase... Read More

Advancement in Phase I/II AFFINITY DUCHENNE ® trial of RGX-202 for Duchenne Muscular Dystrophy; pivotal trial initiation and first functional data expected this month BLA submission for RGX-121 initiated and expected to complete in Q1 2025 Positive Phase II data support bilateral administration of subretinal ABBV-RGX-314; data consistent with that from multiple previous studies demonstrating favorable safety and efficacy... Read More

ROCKVILLE, Md. , Nov. 4, 2024 /PRNewswire/ -- REGENXBIO Inc . (Nasdaq: RGNX) today announced it will participate in the following investor conferences: UBS Global Healthcare Conference Fireside Chat: Wednesday, November 13 at 12:30 p.m. PT Location: Rancho Palos Verdes, CA Stifel 2024 Healthcare Conference Fireside Chat: Tuesday, November 19 at 11:30 a.m. ET Location: New York, NY Piper Sandler 36th Annual Healthcare... Read More

ROCKVILLE, Md. , Oct. 30, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Wednesday, November 6 , at 4:30 p.m. ET to discuss its financial results for the third quarter ended September 30, 2024 , and recent operational highlights. Listeners can register for the webcast via this link . Analysts wishing to participate in the question and answer session should use this... Read More

97% reduction in treatment burden at nine months after treatment with ABBV-RGX-314 Data consistent with that from multiple previous studies demonstrating favorable safety and efficacy profile Well tolerated with zero cases of intraocular inflammation in a setting of no prophylactic steroids Data highlight the potential of ABBV-RGX-314 to treat both eyes in wet AMD ROCKVILLE, Md. , Oct. 21, 2024 /PRNewswire/ -- REGENXBIO Inc.... Read More

ROCKVILLE, Md. , Oct. 18, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present new interim data evaluating subretinal delivery of ABBV-RGX-314 in patients with bilateral wet age-related macular degeneration (wet AMD) at the American Academy of Ophthalmology 2024 annual meeting. ABBV-RGX-314 is an investigational one-time AAV Therapeutic being developed in collaboration with AbbVie for the... Read More

ROCKVILLE, Md. , Sept. 23, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in Chardan's 8 th Annual Genetic Medicines Conference on Monday, September 30, 2024 . Chardan's 8 th Annual Genetic Medicines Conference Fireside Chat: Monday, September 30, 2024 at 8:30 a.m. ET Location: New York, NY A live webcast of the fireside chat can be accessed in the Investors section of REGENXBIO's... Read More

Life sciences veteran Mitchell Chan joins the company ahead of multiple catalysts across late-stage gene therapy programs Vit Vasista steps down following more than 15 years at the Company ROCKVILLE, Md. , Sept. 17, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that Mitchell Chan has been appointed as Executive Vice President and Chief Financial Officer, effective today. A veteran finance executive and... Read More

Data from pivotal dose level demonstrates long-term, sustained reductions in CSF levels of HS D2S6, a key biomarker of brain disease in MPS II 80% of patients who received the pivotal dose discontinued intravenous enzyme replacement therapy or remained treatment-naïve Submission of a rolling BLA using the accelerated approval pathway on track for Q3 2024 ROCKVILLE, Md. , Sept. 3, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq:... Read More

ROCKVILLE, Md. , Aug. 29, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate the following upcoming investor conferences: Morgan Stanley 22 nd Annual Global Healthcare Conference Fireside Chat: Thursday, September 5, 2024 at 5:35 p.m. ET Location: New York, NY Baird 2024 Global Healthcare Conference Fireside Chat: Wednesday, September 11, 2024 at 9:40 a.m. ET Location: New York, NY Live... Read More

ROCKVILLE, Md. , Aug. 27, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced data from its RGX-121 program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be shared at the SSIEM 2024 Annual Symposium, taking place in Porto, Portugal from September 3-6, 2024 . Abstract Title: CAMPSIITE™ phase I/II/III: Interim clinical update of RGX-121, an investigational gene... Read More

ROCKVILLE, Md. , Aug. 8, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the H.C. Wainwright 4 th Annual Ophthalmology Virtual Conference on Thursday, August 15, 2024 . H.C. Wainwright 4 th Annual Ophthalmology Virtual Conference Panel: The Evolving Therapeutic Landscape of AMD Date/Time: Thursday, August 15, 2024 at 9:00 a.m. EDT Location: Virtual A live webcast of the panel can be... Read More

Company remains on track for its first BLA filing in 2024 and is accelerating progress toward pivotal trial initiation for Duchenne (H2 2024) and diabetic retinopathy (H1 2025) New, positive data from Phase II AFFINITY DUCHENNE ® trial of RGX-202 demonstrates consistent high expression of microdystrophin across treated patients in all age groups Successful End-of-Phase II meeting with FDA for RGX-202 supports plans for using... Read More

Robust microdystrophin expression observed in new data from pivotal dose Patients aged 5.8 and 8.5 years at dosing had expression levels at 77.2% and 46.5% of control, respectively Consistent high expression of microdystrophin across treated patients in all age groups continues to support plans for accelerated approval Expects to initiate pivotal trial in Q4 2024 ROCKVILLE, Md. , Aug. 1, 2024 /PRNewswire/ -- REGENXBIO Inc.... Read More

ROCKVILLE, Md. , July 25, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Thursday, August 1, 2024 , at 4:30 p.m. ET to discuss its financial results for the second quarter ended June 30, 2024 , and recent operational highlights. To access the live call by phone, dial (646) 307-1963 (domestic) or (800) 715-9871 (international) and enter the passcode 4849384. To access... Read More

Company to begin enrollment of patients aged 1-3 years Expects data from younger cohort to be part of pivotal plans and BLA filing for broad label End-of-Phase II meeting with FDA scheduled for late July to finalize pivotal program design Based on recent commercial landscape, confirmed accelerated approval pathway remains available given ongoing unmet need and RGX-202 differentiated design Remains on track to initiate... Read More

Aligned with FDA on content of BLA and plans for submission: Submission of a rolling BLA using the accelerated approval pathway expected to start in Q3 2024 Confirmatory trial expected to begin in H2 2025 FDA confirmed RGX-121 commercial bulk drug is comparable to clinical material Positive biomarker, neurocognitive and systemic data will be part of BLA submission ROCKVILLE, Md. , June 18, 2024 /PRNewswire/ -- REGENXBIO Inc.... Read More

Curran Simpson , Chief Operating Officer, Appointed as President and CEO Co-Founder Kenneth T. Mills to Step Down as President and CEO; Named Chairman of the Board ROCKVILLE, Md. , June 12, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that Curran Simpson , Chief Operating Officer, has been appointed as President, Chief Executive Officer and a member of the Board of Directors, effective July 1, 2024.... Read More

ROCKVILLE, Md. , June 5, 2024 /PRNewswire/ -- REGENXBIO Inc . (Nasdaq: RGNX) today announced it will participate in the Goldman Sachs 45th Annual Global Healthcare Conference on Wednesday, June 12, 2024 . Goldman Sachs 45th Annual Global Healthcare Conference Fireside Chat: Wednesday, June 12, 2024 at 8:00 a.m . EDT Location: Miami, FL A live webcast of the fireside chat can be accessed in the Investors section of... Read More

ROCKVILLE, Md. , May 10, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following upcoming investor conferences: BofA Securities Health Care Conference 2024 Fireside Chat: Wednesday, May 15, 2024 at 2:20 p.m. ET Location: Las Vegas, NV Stifel 2024 Tailoring Genes Genetic Medicines Forum Fireside Chat: Tuesday, May 28, 2024 at 4:10 p.m. ET Location: Virtual A live webcast of the... Read More

Company remains on track for its first BLA filing in 2024 and is accelerating progress toward pivotal trial initiation for Duchenne (H2 2024) and diabetic retinopathy (H1 2025) Dose level 2 selected as pivotal dose for RGX-202 treatment of Duchenne New positive interim efficacy and safety data announced for second boy at DL2 AFFINITY DUCHENNE ® trial expansion phase is underway with third and fourth boys dosed at DL2... Read More

ROCKVILLE, Md. , May 1, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Wednesday, May 8, 2024 , at 4:30 p.m. ET to discuss its financial results for the first quarter ended March 31, 2024 , and recent operational highlights. Listeners can register for the webcast via this link . Analysts wishing to participate in the question and answer session should use this link.... Read More

ROCKVILLE, Md. , April 24, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in Chardan's 8th Annual Genetic Medicines and Cell Therapy Manufacturing Summit on Monday, April 29, 2024 . Chardan's 8th Annual Genetic Medicines and Cell Therapy Manufacturing Summit Fireside Chat: Monday, April 29, 2024 at 10:30 a.m. EDT Panel: Gene Therapy: Positioning for CMC Success at Every Stage at 4:00... Read More

A single ABBV-RGX-314 gene therapy treatment has the potential to become a new standard-of-care option among anti-VEGF treatments by sustaining vision health long term and overcoming the clinical challenges of managing wet AMD due to the treatment burden of chronic anti-VEGF injections Patients who received therapeutic doses resulted in stable or improved vision and retinal anatomy up to 2 years Additional long-term... Read More

ROCKVILLE, Md. , March 27, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in Cantor Fitzgerald's Virtual DMD and Other Dystrophy Days on Wednesday, April 3, 2024 . Cantor Fitzgerald's Virtual DMD and Other Dystrophy Days Fireside Chat: Wednesday, April 3, 2024 at 9:50 a.m. EDT Location: Virtual A live webcast of the fireside chat can be accessed in the Investors section of REGENXBIO's... Read More

ROCKVILLE, Md. , March 11, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate the following upcoming investor conferences: Leerink Partners 2024 Global Biopharma Conference Date: Tuesday, March 12, 2024 Location: Miami, FL 1x1 investor meetings only Barclays 26th Annual Global Healthcare Conference Fireside Chat: Wednesday, March 13, 2024 at 4:05 p.m. ET Gene Therapy Regulatory Path in... Read More

ROCKVILLE, Md. , March 6, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy, today announced the pricing of an underwritten upsized public offering of 4,565,260 shares of its common stock at the price of $23.00 per share and 1,521,740 pre-funded warrants at a price of $22.9999 per warrant, in each case... Read More

ROCKVILLE, Md. , March 6, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it intends to offer and sell, subject to market conditions, $125,000,000 of its common stock in an underwritten public offering. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or the actual size or terms of the offering. In addition,... Read More

New three-month assessment in first patient at dose level 2 demonstrates robust microdystrophin expression Patient aged 12 years at dosing had expression level at 75.7% of control Early evidence of strength and motor function improvement observed On track to initiate pivotal trial in second half of 2024 Webcast this morning, Tuesday, March 5 , 8:30 a.m. ET , with principal investigator ROCKVILLE, Md. , March 5, 2024... Read More

Company to host webcast on Tuesday, March 5, 2024 at 8:30 a.m. EST Interim clinical data will be presented by Aravindhan Veerapandiyan, M.D., primary investigator of the AFFINITY DUCHENNE trial, at the MDA Clinical & Scientific Conference on Wednesday, March 6, 2024 at 12:00 p.m. EST ROCKVILLE, Md. , Feb. 29, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a live webcast to discuss new... Read More

Focus on clinical stage AAV Therapeutic product candidates addressing large commercial opportunities and value generation Prioritized pipeline is expected to further progress to pivotal stage and first BLA filing in 2024 New updates planned for Duchenne and in-office delivery retinal disease programs starting in March $314 million in cash, cash equivalents and marketable securities as of December 31, 2023 , expected to fund... Read More

ROCKVILLE, Md. , Feb. 21, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Tuesday, February 27, 2024 , at 4:30 p.m. ET to discuss its financial results for the fourth quarter and full year ended December 31, 2023 , and recent operational highlights. Listeners can register for the webcast via this link . Analysts wishing to participate in the question and answer... Read More

Results support BLA submission in 2024 using the accelerated approval pathway Primary endpoint of patients achieving reduction in CSF biomarker of MPS II disease was met with statistical significance (p value of 0.00016) Patients treated with RGX-121 have showed continued improvement in neurodevelopmental skill acquisition up to four years and discontinued intravenous enzyme therapy Company plans to discuss these results as... Read More

Completed enrollment in cohort 2 of the Phase I/II AFFINITY DUCHENNE ® trial of RGX-202, a potential one-time AAV Therapeutic for the treatment of Duchenne that includes an optimized transgene for a novel microdystrophin New three-month assessment in third patient at dose level 1 demonstrates largest increase in microdystrophin expression Patient aged 6.6 years old had expression level at 83.4% of control On track to... Read More

Pivotal topline results from the CAMPSIITE ® trial of RGX-121 for the treatment of MPS II to be presented Conference call Wednesday, February 7, 4:30 p.m. ET ROCKVILLE, Md. , Jan. 31, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations on its program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be shared at the 20 th Annual WORLD Symposium ™... Read More

A single ABBV-RGX-314 gene therapy treatment has the potential to become a new standard-of-care option among anti-VEGF treatments by sustaining vision health long term and overcoming the clinical challenges of managing wet AMD due to the treatment burden of chronic anti-VEGF injections ABBV-RGX-314 continues to be well tolerated in over 100 patients from three dose levels with no drug-related serious adverse events... Read More

ROCKVILLE, Md. , Jan. 10, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present data from the Phase II AAVIATE ® trial of suprachoroidal ABBV-RGX-314 for the treatment of wet age-related macular degeneration (AMD) at Hawaiian Eye and Retina 2024 Meeting being held January 13-19, 2024 in Maui, HI . ABBV-RGX-314 is an investigational one-time AAV Therapeutic being developed in collaboration... Read More

ROCKVILLE, Md. , Jan. 8, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the U.S. District Court for the District of Delaware granted Sarepta Therapeutics, Inc. (Sarepta) summary judgment on invalidity in a patent infringement suit arising from Sarepta's manufacture and use of cultured host cell technology covered by a University of Pennsylvania ( Penn ) patent that Sarepta uses to make clinical and... Read More

First patient received dose level 2 of RGX-202, a potential one-time AAV Therapeutic for the treatment of Duchenne that includes an optimized transgene for a novel microdystrophin On track for pivotal dose determination and initiation of pivotal program in 2024 ROCKVILLE, Md. , Nov. 29, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the first patient received RGX-202 at dose level 2 in the Phase I/II... Read More

Announces pipeline prioritization and corporate restructuring to focus on clinical stage AAV Therapeutic product candidates addressing large commercial opportunities and value generation Highest priority programs are ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duchenne, and RGX-121 for the treatment MPS II Restructuring plan,... Read More

ROCKVILLE, Md. , Nov. 7, 2023 /PRNewswire/ -- REGENXBIO Inc . (Nasdaq: RGNX) today announced it will participate the following upcoming investor conferences: Stifel 2023 Healthcare Conference Fireside Chat Date: Tuesday, November 14, 2023 Fireside Chat Time: 3:00 p.m. ET Location: New York, NY Piper Sandler 35 th Annual Healthcare Conference Fireside Chat Date: Wednesday, November 29, 2023 Fireside Chat Time: 12:30 p.m. ET... Read More

ABBV-RGX-314 continues to be well tolerated in 50 patients from dose levels 1 and 2 (Cohorts 1-3) with no drug-related serious adverse events Dose level 2 prevented disease progression and reduced vision-threatening events in non-proliferative diabetic retinopathy patients at 1 Year 70.8% of patients achieved Diabetic Retinopathy Severity Scale improvement vs. 25.0 % in control 0% of patients worsened ≥2 steps vs. 37.5 % in... Read More

ROCKVILLE, Md. , Nov. 1, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Wednesday, November 8, 2023 , at 4:30 p.m. EST to discuss its financial results for the quarter ended September 30, 2023 , and recent operational highlights. Listeners can register for the webcast via this link . Analysts wishing to participate in the question and answer session should use this... Read More

ROCKVILLE, Md. , Oct. 30, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the American Academy of Ophthalmology (AAO) 2023 Annual Meeting being held November 3-6, 2023 in San Francisco, CA. The presentations will highlight new data for ABBV-RGX-314, an investigational one-time AAV Therapeutic being developed in collaboration with AbbVie for the treatment of wet age-related macular... Read More

ROCKVILLE, Md. , Oct. 5, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the Jefferies CNS/Neuro Summit on Thursday, October 12, 2023 . Jefferies CNS/Neuro Summit Presentation Date: Thursday, October 12, 2023 Presentation Time: 2:00 p.m. ET Location: New York, NY A live webcast of the presentation can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com.... Read More

RGX-202, a potential one-time AAV Therapeutic for the treatment of Duchenne that includes an optimized transgene for a novel microdystrophin, continues to be well-tolerated in three patients from dose level 1 (1x10 14 GC/kg) Initial biomarker data in two patients who completed three-month assessment demonstrate robust microdystrophin expression with localization to the muscle cell membrane Patient aged 4.4 years old had... Read More

ROCKVILLE, Md. , Sept. 26, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the 28 th Annual Congress of the World Muscle Society, taking place in Charleston, SC from October 3-7, 2023 . Presentations include interim data from the Phase I/II AFFINITY DUCHENNE™ trial, including longer-term safety and initial microdystrophin protein expression levels in muscle at three months. The... Read More

ROCKVILLE, Md. , Sept. 5, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following upcoming investor conferences: Morgan Stanley 21st Annual Global Healthcare Conference Fireside Chat Date: Tuesday, September 12, 2023 Fireside Chat Time: 10:10 a.m. ET Location: New York, NY 2023 Baird Global Healthcare Conference Fireside Chat Date: Wednesday, September 13, 2023 Fireside Chat... Read More

Six-month data from single-patient, investigator-initiated trial showed that one-time administration of RGX-181 was well tolerated, achieved sustained gene expression and demonstrated clinically meaningful improvements across multiple measures including reduced (86%) seizure frequency. Investigators observed encouraging neurodevelopmental skill acquisition at 6 months ROCKVILLE, Md. , Aug. 30, 2023 /PRNewswire/ -- REGENXBIO... Read More

ROCKVILLE, Md. , Aug. 23, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023 in Jerusalem, Israel ( August 29 – September 1 ). Investigators will deliver encore presentations of interim clinical trial data from REGENXBIO's pipeline of one-time investigational AAV Therapeutics for progressive, neurodegenerative... Read More

Continued progress on '5x'25' strategy to advance five AAV Therapeutics from REGENXBIO's internal pipeline and licensed programs into pivotal-stage or commercial products by 2025 ABBV-RGX-314 program for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie Interim results from Phase II AAVIATE ® and ALTITUDE ® trials of dose level 3 cohorts with short-course prophylactic steroid eye... Read More

ROCKVILLE, Md. , Aug. 1, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host one-on-one investor meetings at the 2023 Wedbush PacGrow Healthcare Conference on Tuesday, June 8, 2023 . 2023 Wedbush PacGrow Healthcare Conference Date: Tuesday, August 8, 2023 About REGENXBIO Inc. REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of... Read More

ROCKVILLE, Md. , July 26, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Wednesday, August 2, 2023 , at 4:30 p.m. ET to discuss its financial results for the quarter ended June 30, 2023 , and recent operational highlights. Listeners can register for the webcast via this link . Analysts wishing to participate in the question and answer session should use this link . A... Read More

ROCKVILLE, Md. , July 24, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced three presentations at the American Society of Retina Specialists (ASRS) Annual Scientific Meeting, taking place in Seattle, Washington from July 28 through August 1, 2023 . The presentations will highlight data for ABBV-RGX-314, an investigational one-time AAV therapeutic being developed in collaboration with AbbVie for the... Read More

Interim results from Phase II AAVIATE and ALTITUDE trials of dose level 3 cohorts with short-course prophylactic steroid eye drops resulted in zero cases of intraocular inflammation Initial safety results from Cohort 1 of the AFFINITY DUCHENNE study support well-tolerated profile to date; initial efficacy data to be presented at World Muscle Society meeting in October A new program in Duchenne with preclinical data using... Read More

REGENXBIO AAV Therapeutics pipeline to be discussed by management and external experts Focus on programs for Wet AMD, Diabetic Retinopathy, and Duchenne Muscular Dystrophy ROCKVILLE, Md. , June 27, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a virtual investor day on July 11, 2023 . During the event, members of REGENXBIO's management team and external experts will provide an update to... Read More

ROCKVILLE, Md. , June 20, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it has filed a second complaint for patent infringement against Sarepta Therapeutics, Inc. (Sarepta) arising from Sarepta's manufacture, use and imminent commercial launch of SRP-9001 for the treatment of Duchenne muscular dystrophy. The complaint asserted U.S. Patent No. 11,680,274, which covers Sarepta's AAVrh74-based gene... Read More

ROCKVILLE, Md. , June 1, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present and host one-on-one investor meetings at the Jefferies Global Healthcare Conference on Thursday, June 8, 2023 . Jefferies Global Healthcare Conference Date: Thursday, June 8, 2023 Presentation: 1:30 p.m. ET A live webcast of the presentation can be accessed in the Investors section of REGENXBIO's website at... Read More

RMAT recognizes that the preliminary clinical evidence from RGX-121, a potential one-time AAV Therapeutic, indicates the potential to address unmet medical needs for MPS II RMAT designation is for gene therapies intended to treat or cure serious condition in order to expedite the drug development and review processes CAMPSIITE™ trial is enrolling MPS II patients as part of a pivotal program that incorporates material from... Read More

ROCKVILLE, Md. , May 22, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present in a fireside chat and panel as well as host one-on-one investor meetings at the Barclays Gene Editing & Therapy Summit 2023 on Wednesday, May 24, 2023 . Barclays Gene Editing & Therapy Summit 2023 Date: Wednesday, May 24, 2023 Fireside Chat: 9:15 a.m. ET Panel: Genomic Medicines – Are We Ready for... Read More

Research in MPS IVA led by REGENXBIO scientists moving into clinic Donation of NAV ® Technology licenses will support development of gene therapies for rare diseases ROCKVILLE, Md. , May 16, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that preclinical research in Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio syndrome, was selected for inclusion in the Foundation for the National... Read More

ROCKVILLE, Md. , May 8, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following upcoming investor conferences: BofA Securities 2023 Health Care Conference Fireside Chat Date: Wednesday, May 10, 2023 Fireside Chat Time: 5:00 p.m. PT Location: Las Vegas, NV RBC 2023 Global Healthcare Conference Fireside Chat Date: Tuesday, May 16, 2023 Fireside Chat Time: 3:35 p.m. ET Location:... Read More

Continued progress on '5x'25' strategy to advance five AAV Therapeutics from REGENXBIO's internal pipeline and licensed programs into pivotal-stage or commercial products by 2025 Announced Updates to Eye Care Collaboration with AbbVie Investigational gene therapy renamed ABBV-RGX-314 IND sponsorship recently transferred to AbbVie for all ongoing clinical trials Expanding sites and enrollment in ATMOSPHERE ® and ASCENT™... Read More

ROCKVILLE, Md. , May 2, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced presentations at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, taking place in Los Angeles, California from May 16 through 20, 2023 . The presentations highlight the Company's end-to-end capabilities across clinical development and research and early development. The presentations will be presented as... Read More

ROCKVILLE, Md. , April 26, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Wednesday, May 3, 2023 , at 4:30 p.m. ET to discuss its financial results for the quarter ended March 31, 2023 , and recent operational highlights. Listeners can register for the webcast via this link . Analysts wishing to participate in the question and answer session should use this link . A... Read More

ROCKVILLE, Md. , April 18, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will present in a fireside chat at Chardan's 7th Annual Genetic Medicines and Cell Therapy Manufacturing Summit on Tuesday, April 25, 2023 at 9:30 a.m. ET . The conference will be held in a virtual meeting format. A live webcast of the fireside chat can be accessed in the Investors section of REGENXBIO's website at... Read More

RGX-202 is a potential one-time AAV Therapeutic for the treatment of Duchenne and includes an optimized transgene for a novel microdystrophin and REGENXBIO's proprietary NAV ® AAV8 vector Commercial-scale cGMP material from the REGENXBIO Manufacturing Innovation Center to be used in the clinical trial AFFINITY DUCHENNE™ Phase I/II trial of RGX-202 is active and recruiting patients; anticipated to report initial trial data in... Read More

ROCKVILLE, Md. , March 8, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following upcoming investor conferences: Barclays Global Healthcare Conference Fireside Chat Date: Wednesday, March 15, 2023 Fireside Chat Time: 5:00 p.m. ET Location: Miami, FL BMO Biopharma Spotlight Series: Novel Technologies for the Delivery of Genetic Medicines Panel: Innovative AAVs Targeting Diverse... Read More

Continued progress on '5x'25' strategy to advance five AAV Therapeutics from REGENXBIO's internal pipeline and licensed programs into pivotal-stage or commercial products by 2025 RGX-314 program for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie Enrollment ongoing in the pivotal ATMOSPHERE ® and ASCENT™ clinical trials using subretinal delivery, expected to support BLA filing... Read More

RGX-111 is an investigational AAV Therapeutic for the treatment of severe MPS I that is part of REGENXBIO's clinical-stage pipeline of neurodegenerative disease programs RGX-111, a potential one-time gene therapy for MPS I, continues to be well-tolerated across two dose levels, with no drug-related serious adverse events New biomarker and neurodevelopmental data continue to indicate encouraging CNS profile in patients dosed... Read More

RGX-121, a potential one-time gene therapy for the treatment of MPS II, continues to be well-tolerated with no drug-related SAEs across three dose levels Additional data from patients in Cohort 3 using pivotal program dose level continue to demonstrate largest reductions in CSF GAGs, continuing to approach normal levels at 48 weeks New, longer-term clinical measures demonstrated continued improvement in neurodevelopmental... Read More

ROCKVILLE, Md. , Feb. 21, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Tuesday, February 28, 2023 , at 4:30 p.m. ET to discuss its financial results for the quarter and full year ended December 31, 2022 , and recent operational highlights. Listeners can register for the webcast via this link . Analysts wishing to participate in the question and answer session... Read More

New interim data from Phase I/I/III trial of RGX-121 for the treatment of MPS II and Phase I/II trial of RGX-111 for the treatment of MPS I to be presented ROCKVILLE, Md. , Feb. 17, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that three oral and eight poster presentations will be presented at the 19 th Annual WORLD Symposium ™ 2023, taking place in Orlando, Florida from February 22 through 26, 2023.... Read More

RGX-314 produced by the NAVXpress platform process has been well-tolerated and demonstrated a similar clinical profile to the initial adherent cell culture process NAVXpress platform process is incorporated in the two ongoing pivotal trials and is expected to be used for future commercialization of RGX-314; the two pivotal trials are expected to support BLA submission in 2024 Company to host live webcast with wet AMD Key... Read More

ROCKVILLE, Md. , Feb. 8, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present at the SVB Securities Global Biopharma Conference on Thursday, February 16, 2023 . SVB Securities Global Biopharma Conference Date: Thursday, February 16, 2023 Presentation: 12:00 p.m. ET A live webcast of the presentation can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com . An... Read More

ROCKVILLE, Md. , Feb. 6, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that three presentations on RGX-314 will be presented at the Angiogenesis, Exudation, and Degeneration 2023 Conference taking place virtually from February 10-11, 2023 . New data will be presented from a Phase II bridging study evaluating the pharmacodynamics, safety and efficacy of RGX-314 delivered subretinally using cGMP material... Read More

Company has initiated Phase I/II AFFINITY DUCHENNE ™ trial of RGX-202 Company also enrolling newly active observational screening study, AFFINITY BEYOND, evaluating AAV8 antibody prevalence in boys with Duchenne Commercial-scale cGMP material from the REGENXBIO Manufacturing Innovation Center to be used in the clinical trial RGX-202 is a potential one-time AAV Therapeutic for the treatment of Duchenne and includes an... Read More

Curran Simpson has been appointed Chief Operating Officer Shiva Fritsch assumes the role of Chief Communications Officer ROCKVILLE, Md. , Jan. 9, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the promotion of Curran Simpson from Chief Technology and Operations Officer to Chief Operating Officer. In addition, Shiva Fritsch has been elevated to Chief Communications Officer and will continue in her role as... Read More

ROCKVILLE, Md. , Jan. 3, 2023 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present at the 41st Annual J.P. Morgan Healthcare Conference on Tuesday, January 10, 2023 . 41st Annual J.P. Morgan Healthcare Conference Date: Tuesday, January 10 , 2023 Fireside chat: 11:15 a.m. PT Location: Westin St. Francis, San Francisco, CA A live webcast of the fireside chat can be accessed in the Investors... Read More

RGX-181 and RGX-381 are potential one-time AAV Therapeutics for the treatment of the CNS and ocular manifestations of CLN2 disease, the most common form of Batten disease Patient dosed under a single-patient investigator-initiated study of RGX-181 Company announces approval of CTA for RGX-381 from the UK Health Authority and plans to initiate a Phase I/II clinical trial in the first half of 2023 ROCKVILLE, Md. , Dec. 21,... Read More

RGX-111 is an investigational AAV Therapeutic for the treatment of severe MPS I that is part of REGENXBIO's clinical-stage pipeline of neurodegenerative disease programs. Expanded Cohort 2 enrollment is complete; eight patients have received RGX-111 in the trial Company intends to manufacture commercial-scale cGMP material to support the continued development of RGX-111 Company expects additional interim updates from the... Read More

ROCKVILLE, Md. , Nov. 22, 2022 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following upcoming investor conferences: Piper Sandler 34th Annual Healthcare Conference Presentation Date: Thursday, December 1, 2022 Presentation Time: 9:10 a.m. ET Location: Lotte New York Palace, New York, NY BofA Securities 2022 Virtual Biotech SMID Cap Conference Fireside Chat Date: Thursday, December... Read More