REGENXBIO Inc., a clinical-stage biotechnology company, provides gene therapy product candidates to deliver genes to cells to address genetic defects or to enable cells in the body to produce therapeutic proteins or antibodies that are intended to impact disease. Its gene therapy product candidates are based on NAV Technology Platform, a proprietary adeno-associated virus gene delivery platform. The company's lead product candidate is RGX-314, which is in Phase I/IIa clinical trial for the treatment of wet age-related macular degeneration. It is also developing RGX-121 that is in Phase I/II clinical trial to treat mucopolysaccharidosis type II; RGX-111, which is in Phase I clinical trial for treating mucopolysaccharidosis type I; RGX-181 for the treatment of late-infantile neuronal ceroid lipofuscinosis type II disease; and RGX-501, which is in Phase I/II clinical trials to treat homozygous familial hypercholesterolemia. REGENXBIO Inc. also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies; and has a collaboration and license agreement with Neurimmune AG to develop vectorized antibodies for the treatment of neurodegenerative diseases. The company was formerly known as ReGenX Biosciences, LLC and changed its name to REGENXBIO Inc. in September 2014. REGENXBIO Inc. was founded in 2008 and is headquartered in Rockville, Maryland.
IPO Year: 2015
Exchange: NASDAQ
Website: regenxbio.com
| Date | Price Target | Rating | Analyst |
|---|---|---|---|
| 2/11/2025 | $38.00 → $14.00 | Buy → Neutral | Goldman |
| 2/7/2025 | $27.00 | Outperform | Raymond James |
| 11/15/2024 | $22.00 | Overweight | Morgan Stanley |
| 10/10/2024 | $18.00 | Outperform | Raymond James |
| 6/7/2024 | $38.00 | Buy | Goldman |
| 3/11/2024 | $36.00 | Buy | H.C. Wainwright |
| 3/8/2024 | $20.00 → $35.00 | Sector Perform → Outperform | RBC Capital Mkts |
| 3/6/2024 | $37.00 | Market Perform → Outperform | Leerink Partners |
| 2/21/2024 | $45.00 | Outperform | Raymond James |
| 11/1/2023 | $35.00 | Buy | Stifel |
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-202ROCKVILLE, 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 we
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 measuresBiomarker 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 controlFavorable safety profile continues with no serious adverse events or adverse events of special interest observed Webcast to be held at 8:00 a.m. todayROCKVILLE, Md., June 5, 2025 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced new positive interim data from the Phase I/II AFFI
Event will feature Aravindhan Veerapandiyan, M.D., principal investigator of the AFFINITY DUCHENNE® trialROCKVILLE, 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
$150 million secured at closing extends cash runway into early 2027REGENXBIO 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 Royalty (HCRx). This agreement monetizes select anticipated royalties and milestones, and provides both immediate and expected future, non-dilutive capital. REGENXBIO received $150 million at closing, extending the Company's expected cash runway into early 2027. "This strate
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, (REGENXBIO; Headquarters: Rockville, Maryland, USA; CEO: Curran M. Simpson, NASDAQ:RGNX) for RGX-121 (clemidsogene lanparvovec), a potential first-in-class, investigational gene therapy for the treatment of Mucopolysaccharidosis II (MPS II). The FDA granted REGENXBIO's BLA Priority Review with a Prescription Drug User Fee Act ("PDUFA") t
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 IIPartner Nippon Shinyaku to lead commercialization upon potential approval REGENXBIO to lead commercial manufacturing and supply chainROCKVILLE, Md., May 13, 2025 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) seeking accelerated approval for clemidsogene lanparvovec (RGX-121) for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome. The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act
RGX-202 in Duchenne muscular dystrophy on track for BLA submission mid-2026Pivotal 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 2025FDA acceptance of Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121, partnered with Nippon Shinyaku) expected in May 2025 Pivotal data evaluating the safety and efficacy of the subretinal delivery of surabgene lomparvovec (ABBV-RGX-314, partnered with AbbVie) in patients with wet age-related macular degeneration are expected in 2026 and planning of diabetic retinopathy pivotal study continuesConference call today at 4
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 include encore data from late-stage clinical trials of RGX-121 (clemidsogene lanparvovec) for the treatment of MPS II and RGX-202 for the treatment of Duchenne muscular dystrophy, as well as preclinical research supporting the novel construct of RGX-202 with the C-Terminal dom
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 2025Presentation: Tuesday, May 13, 2025 at 5:00 p.m. PTLocation: Las Vegas, NV RBC Global Healthcare ConferenceFireside Chat: Tuesday, May 20, 2025 at 11:30 a.m. ETLocation: New York, NY H.C. Wainwright BioConnectFireside Chat: Tuesday, May 20, 2025 at 4:00 p.m. ETLocation: New York, NY Stifel Virtual Ophthalmology Forum Fireside Chat: Tuesday, May 27, 2025 at 9:30 a.m. ETLocation: Virtual UBS Spring Biotech Conference1x1 Investor Meetings: Tuesday, June 24, 2025Location: New York, NY Live webcasts of select
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 replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time. ABOUT REGENXBIO Inc.REGENXBIO is a biotechnology company on a mi
Goldman downgraded REGENXBIO from Buy to Neutral and set a new price target of $14.00 from $38.00 previously
Raymond James resumed coverage of REGENXBIO with a rating of Outperform and set a new price target of $27.00
Morgan Stanley resumed coverage of REGENXBIO with a rating of Overweight and set a new price target of $22.00
Raymond James resumed coverage of REGENXBIO with a rating of Outperform and set a new price target of $18.00
Goldman initiated coverage of REGENXBIO with a rating of Buy and set a new price target of $38.00
H.C. Wainwright initiated coverage of REGENXBIO with a rating of Buy and set a new price target of $36.00
RBC Capital Mkts upgraded REGENXBIO from Sector Perform to Outperform and set a new price target of $35.00 from $20.00 previously
Leerink Partners upgraded REGENXBIO from Market Perform to Outperform and set a new price target of $37.00
Raymond James resumed coverage of REGENXBIO with a rating of Outperform and set a new price target of $45.00
Stifel initiated coverage of REGENXBIO with a rating of Buy and set a new price target of $35.00
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RGX-202 in Duchenne muscular dystrophy on track for BLA submission mid-2026Pivotal 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 2025FDA acceptance of Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121, partnered with Nippon Shinyaku) expected in May 2025 Pivotal data evaluating the safety and efficacy of the subretinal delivery of surabgene lomparvovec (ABBV-RGX-314, partnered with AbbVie) in patients with wet age-related macular degeneration are expected in 2026 and planning of diabetic retinopathy pivotal study continuesConference call today at 4
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 replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time. ABOUT REGENXBIO Inc.REGENXBIO is a biotechnology company on a mi
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 use this link. A replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time. ABOUT REGENXBIO Inc.REGENXBIO is a leading
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 link. A replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time. ABOUT REGENXBIO Inc. REGENXBIO is a leading clinical-s
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 a live or recorded webcast of the call, please visit the Investors section of the REGENXBIO website at www.regenxbio.com. The recorded webcast will be available for approximately 30 days following the call. Those who plan on participating are advised to join 15 minutes prior
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. A replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time. ABOUT REGENXBIO Inc. REGENXBIO is a leading clinical-stage
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 controlEarly evidence of strength and motor function improvement observedOn track to initiate pivotal trial in second half of 2024 Webcast this morning, Tuesday, March 5, 8:30 a.m. ET, with principal investigatorROCKVILLE, Md., March 5, 2024 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today reported additional interim safety and efficacy data in the Phase I/II AFFINITY DUCHENNE® trial of RGX-202 in patients with Duchenne muscular dystrophy (Duchenne) ages 4 to11 years old, including RGX-202 microdystrophin expression from
Focus on clinical stage AAV Therapeutic product candidates addressing large commercial opportunities and value generationPrioritized pipeline is expected to further progress to pivotal stage and first BLA filing in 2024New 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 operational runway into the second half of 2025Conference call Tuesday, February 27, at 4:30 p.m. ETROCKVILLE, Md., Feb. 27, 2024 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced financial results for the fourth quarter and full year ended December 31, 2023, and r
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 session should use this link. A replay of the webcast will be available via the company's investor website approximately two hours after the call's conclusion. Those who plan on participating are advised to join 15 minutes prior to the start time. About REGENXBIO Inc. REGENXBIO is
Results support BLA submission in 2024 using the accelerated approval pathwayPrimary 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 therapyCompany plans to discuss these results as part of a full rare disease program update on its conference call today, Wednesday, February 7, 4:30 p.m. ETROCKVILLE, Md., Feb. 7, 2024 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced topline results from the Phase I/II/III CAMPSIITE® trial of RGX-121 for the
NEW YORK, Dec. 6, 2024 /PRNewswire/ -- S&P Dow Jones Indices ("S&P DJI") will make the following changes to the S&P 500, S&P MidCap 400, and S&P SmallCap 600 indices effective prior to the open of trading on Monday, December 23, to coincide with the quarterly rebalance. The changes ensure each index is more representative of its market capitalization range. All companies being added to the S&P 500 are more representative of the large-cap market space, all companies being added to the S&P MidCap 400 are more representative of the mid-cap market space, and all companies being added to the S&P SmallCap 600 are more representative of the small-cap market space. The companies being removed from t
Life sciences veteran Mitchell Chan joins the company ahead of multiple catalysts across late-stage gene therapy programsVit Vasista steps down following more than 15 years at the CompanyROCKVILLE, 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 operating partner in the biotechnology industry, Mr. Chan has held key roles in raising capital, business development transactions and operations for nearly 20 years. "We are excited to welcome Mitchell at this exciting time for REGENXBIO," said Curran Simpson, President and
Curran Simpson, Chief Operating Officer, Appointed as President and CEOCo-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. Co-Founder Kenneth T. Mills will step down as President and Chief Executive Officer following 15 years of leadership at the Company, and he is taking on an expanded role on the Board as Chairman. In connection with these changes, Board member Daniel Tassé has been appointed as RE
NEW YORK, July 07, 2022 (GLOBE NEWSWIRE) -- Eyenovia, Inc. (NASDAQ:EYEN), an ophthalmic pharmaceutical technology company developing a pipeline of late-stage microdose array print (MAP™) therapeutics, today announced that it has appointed Dr. Ellen Strahlman and Dr. Ram Palanki as new and independent members of its Board of Directors. With these appointments, the Eyenovia Board will expand to eight seats, from six currently. "I could not be more pleased to welcome Drs. Strahlman and Palanki to our Board and look forward to their immediate contributions," stated Dr. Sean Ianchulev, chief executive officer and chief medical officer of Eyenovia. "Each brings decades of medical technology, cl
ROCKVILLE, Md., June 7, 2022 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced the appointment of Jennifer Zachary to its Board of Directors, effective June 3, 2022. Ms. Zachary will serve as a member of REGENXBIO's Audit Committee and brings 20 years of legal and regulatory experience working in the pharmaceutical and medical device industries. "We are pleased to welcome Jennifer to our Board of Directors," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "Jennifer is an accomplished pharmaceutical executive with a wealth of knowledge and
ROCKVILLE, Md., Sept. 7, 2021 /PRNewswire/ -- Renowned gene therapy pioneer and biotechnology executive bring extensive R&D leadership and financial and operational expertise to the BoardREGENXBIO Inc. (NASDAQ:RGNX) today announced the appointment of Jean Bennett, M.D., Ph.D., and George Migausky to its Board of Directors, effective September 3, 2021. Dr. Bennett, a leading molecular genetics researcher, is the F.M. Kirby Emeritus Professor of Ophthalmology at the Perelman School of Medicine and previously served as director of the Center for Advanced Retinal and Ocular Therapeutics (CAROT) at the University of Pennsylvania. She will serve as a member of REGENXBIO's Nominating and Corporate
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