RGNX REGENXBIO Inc.

30.96
-2.25  -7%
Previous Close 33.21
Open 32.77
52 Week Low 27.0192
52 Week High 50.26
Market Cap $1,324,134,680
Shares 42,769,208
Float 33,937,065
Enterprise Value $1,165,714,397
Volume 263,459
Av. Daily Volume 414,676
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Upcoming Catalysts

Drug Stage Catalyst Date
RGX-111
Mucopolysaccharidosis Type I (MPS I)
Phase 1/2
Phase 1/2
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RGX-121
MPS II (Hunter Syndrome)
Phase 1/2
Phase 1/2
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Drug Pipeline

Drug Stage Notes
RGX-202
Duchenne
Phase 1
Phase 1
IND filing expected end of 2021.
RGX-314 - ALTITUDE
Diabetic retinopathy
Phase 2
Phase 2
Phase 2 initial data update from Cohort 1 at 3-months after one-time treatment, 33% of patients demonstrated a ≥2 step improvement from baseline on the ETDRS-DRSS compared to 0% of patients in observational control. Suprachoroidal delivery dose was well tolerated in 15 patients in Cohort 1 with no drug-related serious adverse events, no intraocular inflammation observed. Cohorts 2 and 3 continue to enroll patients at a dose level of 5x1011 GC/eye - noted November 16, 2021.
RGX-314
Neovascular Age-related macular degeneration (AMD)
Phase 1/2
Phase 1/2
Phase 1/2a two year results reported dose-dependent protein levels across all 5 cohorts and 20 SAEs were reported in 13 patients, with one possibly drug-related SAE reported in a patient in Cohort 5 causing significant decrease in vision, noted November 12, 2021.
RGX-314 (AAVIATE)
Wet AMD using suprachoroidal delivery
Phase 2
Phase 2
Phase 2 interim results reported that 15 patients in Cohort 2 had a mean BCVA change of -0.1 letters when measured from Day 1 and +0.2 letters when measured from Week 1. These patients also demonstrated stable central retinal thickness (CRT), noted November 12, 2021. Phase 2 safety results reported at Retina Society 54th Annual Scientific Meeting showed that it was well tolerated by 50 patients in the first 3 cohorts - noted October 4, 2021.
RGX-314 (ATMOSPHERE)
Wet AMD
Phase 2/3
Phase 2/3
Phase 2/3 trial is enrolling. Second pivotal trial to be initiated 4Q 2021.
RGX-501
Homozygous familial hypercholesterolemia (HoFH)
Phase 1/2
Phase 1/2
Development to be discontinued - noted August 6, 2020.

Latest News

  1. ROCKVILLE, Md., Nov. 22, 2021 /PRNewswire/ --

    • Potential one-time gene therapy for the treatment of Duchenne, includes a novel, optimized microdystrophin transgene and REGENXBIO's proprietary NAV® AAV8 vector
    • Commercial-scale cGMP material to be used in clinical development
    • Company on track to submit IND by end of 2021

    REGENXBIO Inc. (NASDAQ:RGNX) today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for RGX-202, a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne). RGX-202 is designed to deliver a novel, optimized microdystrophin transgene with a unique C-terminal domain and a muscle specific promoter to support targeted therapy for improved resistance to muscle damage…

    ROCKVILLE, Md., Nov. 22, 2021 /PRNewswire/ --

    • Potential one-time gene therapy for the treatment of Duchenne, includes a novel, optimized microdystrophin transgene and REGENXBIO's proprietary NAV® AAV8 vector
    • Commercial-scale cGMP material to be used in clinical development
    • Company on track to submit IND by end of 2021

    REGENXBIO Inc. (NASDAQ:RGNX) today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for RGX-202, a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne). RGX-202 is designed to deliver a novel, optimized microdystrophin transgene with a unique C-terminal domain and a muscle specific promoter to support targeted therapy for improved resistance to muscle damage associated with Duchenne. RGX-202 uses REGENXBIO's proprietary NAV® AAV8 vector. 

    "This important designation is a milestone in the development of RGX-202 and highlights the need for potential new treatment options for patients with Duchenne," said Olivier Danos, Ph.D., Chief Scientific Officer of REGENXBIO. "The novel microdystrophin transgene in RGX-202 includes coding regions that retain essential functional elements of naturally occurring dystrophin to potentially improve muscle strength and resistance in patients with Duchenne. We look forward to advancing this one-time gene therapy into the clinic." 

    REGENXBIO expects to submit an Investigational New Drug (IND) application to the FDA for RGX-202 by the end of 2021. Commercial-scale cGMP material has already been produced at 1,000 liter capacity using REGENXBIO's suspension cell culture manufacturing process, and the Company's internal cGMP facility is expected to allow for production up to 2,000 liters for the clinical development of RGX-202.

    FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.

    About RGX-202

    RGX-202 is designed to deliver a novel microdystrophin transgene which retains key elements of the dystrophin protein, including an extended coding region of the C-Terminal (CT) domain found in naturally-occurring dystrophin, as well as other fundamental improvements to the transgene. Presence of the CT domain has been shown to recruit several key proteins to the muscle cell membrane, leading to improved muscle resistance to contraction-induced muscle damage in dystrophic mice. Additional design features, including codon optimization and reduction of CpG content, may potentially improve gene expression, increase translational efficiency and reduce immunogenicity. RGX-202 is designed to support the delivery and targeted expression of genes throughout skeletal and heart muscle using the NAV AAV8 vector, a vector used in numerous clinical trials, and a well-characterized muscle targeting promoter (Spc5-12).

    About REGENXBIO Inc.

    REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

    Forward-Looking Statements

    This 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 timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development 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, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, 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, 2020 and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at www.sec.gov. 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.

    Contacts:

    Tricia Truehart

    Investor Relations and Corporate Communications

    347-926-7709

    Investors:

    Brendan Burns, 212-600-1902

    Media:

    David Rosen, 212-600-1902

    (PRNewsfoto/REGENXBIO Inc.)

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-announces-orphan-drug-designation-granted-to-rgx-202-a-novel-gene-therapy-candidate-for-the-treatment-of-duchenne-muscular-dystrophy-301429654.html

    SOURCE REGENXBIO Inc.

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  2. ROCKVILLE, Md., Nov. 12, 2021 /PRNewswire/ --

    • Suprachoroidal delivery of RGX-314 in Phase II AAVIATE® trial for the treatment of wet AMD continues to be well tolerated in 50 patients from Cohorts 1-3 with no drug-related serious adverse events
    • Positive initial data from Cohort 2 in AAVIATE trial at six months after one-time treatment of RGX-314
      • Treatment effect observed with stable visual acuity and retinal thickness
      • Demonstrated meaningful reduction (>70%) in anti-VEGF treatment burden; 40% of patients in Cohort 2 were anti-VEGF injection-free
    • Additional data from Cohort 1 in Phase II ALTITUDE™ trial for the treatment of DR demonstrated stable visual acuity at three months after one-time treatment of RGX-314

    REGENXBIO Inc. (NASDAQ:RGNX) today…

    ROCKVILLE, Md., Nov. 12, 2021 /PRNewswire/ --

    • Suprachoroidal delivery of RGX-314 in Phase II AAVIATE® trial for the treatment of wet AMD continues to be well tolerated in 50 patients from Cohorts 1-3 with no drug-related serious adverse events
    • Positive initial data from Cohort 2 in AAVIATE trial at six months after one-time treatment of RGX-314
      • Treatment effect observed with stable visual acuity and retinal thickness
      • Demonstrated meaningful reduction (>70%) in anti-VEGF treatment burden; 40% of patients in Cohort 2 were anti-VEGF injection-free
    • Additional data from Cohort 1 in Phase II ALTITUDE™ trial for the treatment of DR demonstrated stable visual acuity at three months after one-time treatment of RGX-314

    REGENXBIO Inc. (NASDAQ:RGNX) today announced additional positive interim data from the ongoing Phase II AAVIATE® trial and the ongoing Phase II ALTITUDE™ trial of RGX-314 using in-office suprachoroidal delivery for the treatment of wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR) without center-involved diabetic macular edema (CI-DME), respectively. The results were presented at the American Academy of Ophthalmology (AAO) 2021 Annual Meeting by Robert L. Avery, M.D., Founder of California Retina Consultants and Research Foundation. 

    "We are pleased to share this initial data from Cohort 2 of the AAVIATE trial which provides encouraging evidence of the emerging clinical profile of RGX-314 for the treatment of wet AMD using suprachoroidal delivery. In the data reported today, RGX-314 was observed to be well tolerated in Cohort 2, with stable visual acuity and retinal thickness as well as a meaningful reduction in anti-VEGF treatment burden at six months," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "We look forward to providing additional updates from the program."

    "These initial results from patients in Cohort 2 of the AAVIATE trial at six months after suprachoroidal administration of RGX-314 reinforce the potential impact that RGX-314 could have on the overall clinical management of patients with wet AMD," said Dr. Avery. "I am encouraged by the six-month data in Cohort 2 and look forward to reviewing further data from Cohorts 1-3 and from the higher dose level in Cohorts 4 and 5."

    Study Design and Safety Update in Phase II AAVIATE Trial of RGX-314 for the Treatment of Wet AMD Using Suprachoroidal Delivery

    AAVIATE is a multi-center, open-label, randomized, active-controlled, dose-escalation trial that will evaluate the efficacy, safety and tolerability of suprachoroidal delivery of RGX-314 using the SCS Microinjector®. Twenty patients in Cohort 1 were randomized to receive RGX-314 at a dose level of 2.5x1011 genomic copies per eye (GC/eye) versus monthly 0.5 mg ranibizumab intravitreal injection at a 3:1 ratio. Twenty patients in Cohort 2 were randomized to receive RGX-314 at a dose level of 5x1011 GC/eye through two injections versus monthly 0.5 mg ranibizumab intravitreal injection at a 3:1 ratio. Cohort 3 is evaluating RGX-314 at the same dose level as Cohort 2 in 20 patients who are neutralizing antibody (NAb) positive. Enrollment is ongoing in two additional cohorts (Cohorts 4 and 5) to evaluate RGX-314 at a third dose level of 1x1012 GC/eye. Cohort 4 will enroll 15 patients who will be dosed with RGX-314 and Cohort 5 will evaluate the same dose level of RGX-314 in 20 patients who are NAb positive. Patients do not receive prophylactic immune suppressive corticosteroid therapy before or after administration of RGX-314.    

    As of November 4, 2021, RGX-314 was reported to be well tolerated across 50 patients dosed in Cohorts 1-3. Four serious adverse events (SAEs) were reported in four patients, all of which were considered not related to RGX-314.1  For the total group of Cohorts 1 and 2, all common treatment emergent adverse events (TEAEs) through 6 months in the study eye were mild, and included conjunctival hemorrhage, worsening of wet AMD, dry eye, episcleritis, and conjunctival hyperemia. Mild intraocular inflammation observed on slit-lamp examination was reported at similar incidence across both dose levels in Cohorts 1 and 2, with four out of 15 patients in Cohort 1 and three out of 15 patients in Cohort 2. All cases of inflammation in both cohorts were resolved within days to weeks on topical corticosteroids.

    Summary of Data for Cohort 2 in Phase II AAVIATE Trial of RGX-314 for the Treatment of Wet AMD at Six Months

    In Cohort 2, patients dosed with RGX-314 demonstrated stable Best Corrected Visual Acuity (BCVA) and central retinal thickness (CRT) at 6 months. Fifteen patients in Cohort 2 dosed with RGX-314 had a mean BCVA change of -0.1 letters (95% Confidence Interval: -3.8, 3.6) when measured from Day 1 (at Screening) and +0.2 letters (-2.7, 3.1) when measured from Week 1 (prior to Randomization). These patients also demonstrated stable central retinal thickness (CRT), with a mean change of -33 µm (-71, 5) at six months from Day 1. Ten control patients receiving monthly injections of ranibizumab in Cohorts 1 and 2 had a mean BCVA change at six months of +4.0 letters (-0.5, 8.5) when measured from Day 1 and +1.3 letters (-2.2, 4.8) when measured from Week 1. Patients receiving monthly injections of ranibizumab had a mean change of CRT of -12 µm (-33, 8) at six months from Day 1.

    There was a meaningful reduction in anti-vascular endothelial growth factor (anti-VEGF) treatment burden in patients following administration of RGX-314 compared to the mean annualized injection rate during the 12 months prior to administration. Patients in Cohort 2 received a mean of 1.3 injections over six months following administration of RGX-314, which represents a 71.8% reduction in anti-VEGF treatment burden. Six out of 15 patients (40%) in Cohort 2 received no anti-VEGF injections over six months following RGX-314 administration. In these patients, visual acuity and CRT was observed to be stable from Day 1 over six months, with a mean change of BCVA of +1.0 letters (-3.8, 5.8), and a mean change of CRT of +8 µm (-9.2, 24.2). 

    Summary of Visual Acuity Data for Cohort 1 in Phase II ALTITUDE Trial of RGX-314 for the Treatment of Diabetic Retinopathy at Three Months

    REGENXBIO shared additional data from Cohort 1 of the ongoing ALTITUDE trial for the treatment of DR without CI-DME using in-office suprachoroidal delivery, supporting the positive initial data previously reported at the American Society of Retina Specialists (ASRS) Annual Meeting in October 2021. At three months, 15 patients dosed with 2.5x1011 GC/eye of RGX-314 demonstrated stable BCVA of +2.6 letters, while five patients in the observational control arm demonstrated stable BCVA of -0.4 letters.

    Data presented today are available on the "Presentations and Publications" section of the REGENXBIO website at www.regenxbio.com.

    About RGX-314

    RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

    REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

    About Wet AMD

    Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the United States, Europe and Japan, with up to 2 million people living with wet AMD in these geographies alone. Current anti-VEGF therapies have significantly changed the landscape for treatment of wet AMD, becoming the standard of care due to their ability to prevent progression of vision loss in the majority of patients. These therapies, however, require life-long repeated intraocular injections to maintain efficacy. Due to the burden of treatment, patients often experience a decline in vision with reduced frequency of treatment over time.

    About Diabetic Retinopathy

    Diabetic retinopathy (DR) is the leading cause of vision loss in adults between 24 and 75 years of age worldwide. DR affects approximately eight million people in the United States alone. The spectrum of DR severity ranges from non-proliferative diabetic retinopathy (NPDR) to proliferative diabetic retinopathy (PDR) and as DR progresses, a large proportion of patients develop vision threatening complications, including diabetic macular edema (DME) and neovascularization that can lead to blindness.  Current treatment options for patients with DR include "watchful waiting", anti-VEGF treatment, retinal laser or surgical treatment.

    About REGENXBIO Inc.

    REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

    Forward-Looking Statements

    This 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 timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development 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, the impact of the COVID-19 pandemic or similar public health crises on REGENXBIO's business, 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, 2020 and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at www.sec.gov. 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.

    1 One patient in Cohort 1 discontinued the study after Week 12 as a result of death, which was assessed to be unrelated to RGX-314. At the time of the death, the subject was free of anti-VEGF injections.

    SCS Microinjector® is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

    Contacts:

    Tricia Truehart

    Investor Relations and Corporate Communications

    347-926-7709

    Investors:

    Brendan Burns, 212-600-1902

    Media:

    David Rosen, 212-600-1902

    (PRNewsfoto/REGENXBIO Inc.)

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-presents-additional-positive-interim-data-from-trials-of-rgx-314-in-wet-amd-and-diabetic-retinopathy-using-suprachoroidal-delivery-at-aao-2021-301423126.html

    SOURCE REGENXBIO Inc.

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  3. ROCKVILLE, Md., Nov. 9, 2021 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced the closing of its Collaboration and License Agreement with AbbVie to develop and commercialize RGX-314, a potential one-time gene therapy for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other chronic retinal diseases. As previously announced on September 13, 2021, under the terms of the agreement, REGENXBIO will receive an upfront payment from AbbVie of $370 million with the potential for REGENXBIO to receive up to $1.38 billion in additional development, regulatory and commercial milestones.

    Under the collaboration, REGENXBIO will be responsible for completion of the ongoing trials of RGX-314. AbbVie…

    ROCKVILLE, Md., Nov. 9, 2021 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced the closing of its Collaboration and License Agreement with AbbVie to develop and commercialize RGX-314, a potential one-time gene therapy for the treatment of wet age-related macular degeneration (wet AMD), diabetic retinopathy (DR) and other chronic retinal diseases. As previously announced on September 13, 2021, under the terms of the agreement, REGENXBIO will receive an upfront payment from AbbVie of $370 million with the potential for REGENXBIO to receive up to $1.38 billion in additional development, regulatory and commercial milestones.

    Under the collaboration, REGENXBIO will be responsible for completion of the ongoing trials of RGX-314. AbbVie and REGENXBIO will collaborate and share costs on additional trials of RGX-314, including the planned second pivotal trial evaluating subretinal delivery for the treatment of wet AMD and future trials. AbbVie will lead the clinical development and commercialization of RGX-314 globally. REGENXBIO will participate in U.S. commercialization efforts as provided under a mutually agreed upon commercialization plan.

    REGENXBIO and AbbVie will share equally in profits from net sales of RGX-314 in the U.S. and AbbVie will pay REGENXBIO tiered royalties on net sales of RGX-314 outside the U.S. In addition, REGENXBIO will lead the manufacturing of RGX-314 for clinical development and U.S. commercial supply, and AbbVie will lead manufacturing of RGX-314 for commercial supply outside the U.S.

    About RGX-314

    RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

    REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

    About REGENXBIO Inc.

    REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

    SCS Microinjector® is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

    Forward-Looking Statements

    This 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 collaboration with AbbVie and 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 outcome of REGENXBIO's collaboration with AbbVie 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, 2020 and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at www.sec.gov. 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.

    Contacts:

    Tricia Truehart

    Investor Relations and Corporate Communications

    347-926-7709

    Investors:

    Brendan Burns, 212-600-1902

    Media:

    David Rosen, 212-600-1902

     

    (PRNewsfoto/REGENXBIO Inc.)

     

    Cision View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-announces-closing-of-eye-care-collaboration-agreement-with-abbvie-301420381.html

    SOURCE REGENXBIO Inc.

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  4. ROCKVILLE, Md., Nov. 8, 2021 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced it will participate in the following investor conferences, which will each be held in a virtual meeting format:

    Barclays Gene Editing & Gene Therapy Summit
    Presentation: Monday, November 15, 2021, at 1:30 p.m. ET

    Piper Sandler 33rd Annual Virtual Healthcare Conference
    Date: December 2, 2021
    Recorded Fireside Chat will be available: Monday, November 22, 2021, at 10:00 a.m. ET

    A live webcast of the Barclays presentation and the recording of the Piper Sandler fireside chat can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com for approximately 30 days following the events.

    About REGENXBIO Inc.

    REGENXBIO is a leading clinical-stage…

    ROCKVILLE, Md., Nov. 8, 2021 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced it will participate in the following investor conferences, which will each be held in a virtual meeting format:

    Barclays Gene Editing & Gene Therapy Summit

    Presentation: Monday, November 15, 2021, at 1:30 p.m. ET

    Piper Sandler 33rd Annual Virtual Healthcare Conference

    Date: December 2, 2021

    Recorded Fireside Chat will be available: Monday, November 22, 2021, at 10:00 a.m. ET

    A live webcast of the Barclays presentation and the recording of the Piper Sandler fireside chat can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com for approximately 30 days following the events.

    About REGENXBIO Inc.

    REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

    Contacts:

    Tricia Truehart

    Investor Relations and Corporate Communications

    347-926-7709

    Investors:

    Brendan Burns, 212-600-1902

    Media:

    David Rosen, 212-600-1902

    (PRNewsfoto/REGENXBIO Inc.)

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  5. ROCKVILLE, Md., Nov. 5, 2021 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced that data from the RGX-314 clinical trials will be presented in an oral presentation at the American Academy of Ophthalmology 2021 Annual Meeting taking place in New Orleans, LA from November 12 to 15, 2021. RGX-314 is a potential one-time gene therapy in clinical development for the treatment of wet age-related macular degeneration (wet AMD) and diabetic retinopathy.

    The American Academy of Ophthalmology 2021 Annual Meeting presentation is as follows:

    Title: Two Year Results from the Subretinal RGX-314 Gene Therapy Phase 1/2a Study for the Treatment of nAMD, and an Update on Suprachoroidal Trials
    Presenter: Robert L. Avery, M.D., Founder of California…

    ROCKVILLE, Md., Nov. 5, 2021 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX) today announced that data from the RGX-314 clinical trials will be presented in an oral presentation at the American Academy of Ophthalmology 2021 Annual Meeting taking place in New Orleans, LA from November 12 to 15, 2021. RGX-314 is a potential one-time gene therapy in clinical development for the treatment of wet age-related macular degeneration (wet AMD) and diabetic retinopathy.

    The American Academy of Ophthalmology 2021 Annual Meeting presentation is as follows:

    Title: Two Year Results from the Subretinal RGX-314 Gene Therapy Phase 1/2a Study for the Treatment of nAMD, and an Update on Suprachoroidal Trials

    Presenter: Robert L. Avery, M.D., Founder of California Retina Consultants and Research Foundation

    Session Title: RET10 - Section VIII: First-time Results of Clinical Trials

    Date/Time: Friday, November 12, 2021, from 3:32 to 3:38 p.m. CT

    Type: Oral presentation                                          

    About RGX-314

    RGX-314 is being investigated as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.

    REGENXBIO is advancing research in two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.

    About REGENXBIO Inc.

    REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV® Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

    SCS Microinjector® is a trademark of Clearside Biomedical, Inc. All other trademarks referenced herein are registered trademarks of REGENXBIO.

    Contacts:

    Tricia Truehart

    Investor Relations and Corporate Communications

    347-926-7709

    Investors:

    Brendan Burns, 212-600-1902

    Media:

    David Rosen, 212-600-1902

    (PRNewsfoto/REGENXBIO Inc.)

     

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