RPGR

Beacon Therapeutics Announces Positive 12-Month Data from Phase 2 SKYLINE Trial of AGTC-501 in Patients with X-Linked Retinitis Pigmentosa

Retrieved on: 
Thursday, February 8, 2024

Response rates in study eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 were similar to the untreated fellow eyes in the high dose cohort (0% for both).

Key Points: 
  • Response rates in study eyes treated with a low dose (7.5 E+10 vg/eye) of AGTC-501 were similar to the untreated fellow eyes in the high dose cohort (0% for both).
  • In addition, patients in the high dose cohort also demonstrated a robust improvement in visual function, including mean retinal sensitivity.
  • XLRP is an orphan disease predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene.
  • We look forward to announcing the initiation of our Phase 2/3 VISTA trial in the first half of 2024."

Beacon Therapeutics launches with £96 million ($120 million) to develop a new generation of gene therapies for retinal diseases resulting in blindness

Retrieved on: 
Monday, June 12, 2023

XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene.

Key Points: 
  • XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene.
  • Beacon Therapeutics’s first pre-clinical asset is an intravitreally (IVT) delivered novel AAV based program for dry Age-related Macular Degeneration (dry AMD).
  • Beacon Therapeutics will be led by David Fellows, the former Chief Executive of Nightstar Therapeutics with over 40 years’ experience in the ophthalmology field.
  • Beacon Therapeutics is quite unique in being a company that at launch is already underpinned by excellent clinical trial data.

MeiraGTx Reports First Quarter 2023 Financial and Operational Results

Retrieved on: 
Thursday, May 11, 2023

On track for BLA submission of botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) in 2024

Key Points: 
  • On track for BLA submission of botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) for the treatment of X-linked retinitis pigmentosa (XLRP) in 2024
    LONDON and NEW YORK, May 11, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced financial and operational results for the first quarter ended March 31, 2023, and provided a corporate update.
  • In May 2023, MeiraGTx closed the previously announced private investment in public equity (PIPE) financing, raising approximately $60 million in aggregate gross proceeds.
  • Cash and cash equivalents were $68.8 million as of March 31, 2023, compared to $115.5 million as of December 31, 2022.
  • License revenue was $3.3 million for the quarter ended March 31, 2023, compared to $5.6 million for the quarter ended March 31, 2022.

MeiraGTx Announces Upcoming Presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting

Retrieved on: 
Friday, April 21, 2023

LONDON and NEW YORK, April 21, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced two abstract presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting .

Key Points: 
  • LONDON and NEW YORK, April 21, 2023 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced two abstract presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting .
  • The second abstract will present data from MeiraGTx’s proprietary promoter platform involving the use of novel, AI-assisted engineered promoters to improve gene expression in rod photoreceptors.
  • “We are pleased to present recent data from the Phase 1/2 clinical study of our investigational gene therapy bota-vec at this year’s ARVO meeting.
  • The abstracts can be found on the ARVO Annual Meeting website .

Janssen to Highlight Innovation in Retinal Pipeline at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting

Retrieved on: 
Friday, April 21, 2023

RARITAN, N.J., April 21, 2023 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that five company-sponsored presentations will be featured during the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting in New Orleans from April 23-27, 2023. Janssen presentations will include updates from the Phase 1/2 MGT009 trial for investigational gene therapy botaretigene sparoparvovec (bota-vec, formerly AAV-RPGR) in patients with the inherited retinal disease (IRD) X-linked retinitis pigmentosa (XLRP) associated with the retinitis pigmentosa GTPase regulator (RPGR) gene, and pooled safety analysis data from two Phase 1 trials of the investigational gene therapy JNJ-81201887 (JNJ-1887); one trial in patients with geographic atrophy (GA), a late-stage and severe form of age-related macular degeneration (AMD) and one trial in wet AMD (Abstracts #5446 and #732-C0314).1,2

Key Points: 
  • Important new data on Janssen's investigational gene therapy portfolio, including botaretigene sparoparvovec (bota-vec) and JNJ-1887, will be presented
    RARITAN, N.J., April 21, 2023 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that five company-sponsored presentations will be featured during the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting in New Orleans from April 23-27, 2023.
  • In this new era of innovation, we're taking a cutting-edge approach to saving sight through groundbreaking technologies such as gene therapy, and we're thrilled to showcase some of this research at this year's ARVO annual meeting," said James List, M.D., Ph.D., Global Therapeutic Area Head, whose team oversees a portfolio of programs including Retina at Janssen Research & Development, LLC.
  • "We are on a bold mission to restore and preserve vision for people living with retinal diseases, and these important data bring us one step closer to building a brighter future for the patients we serve."
  • Abstracts can also be found on the ARVO Annual Meeting website.

AAV Vectors in Gene Therapy - Pipeline Insight, 2022 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, November 22, 2022

The "AAV vectors in gene therapy - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "AAV vectors in gene therapy - Pipeline Insight, 2022" drug pipelines has been added to ResearchAndMarkets.com's offering.
  • AAV vectors are the leading viral vectors for gene delivery to treat a variety of human diseases.
  • AAV vectors in gene therapy: Therapeutic Assessment
    This segment of the report provides insights about the different AAV vectors in gene therapy drugs segregated based on following parameters that define the scope of the report, such as:
    There are approx.
  • The companies which have their AAV vectors in gene therapy drug candidates in the most advanced stage, i.e.

Foundation Launching its Largest Natural History Study to Date for 1,500 People with Inherited Retinal Diseases Caused by Rare Mutated Genes

Retrieved on: 
Thursday, November 10, 2022

COLUMBIA, Md., Nov. 10, 2022 /PRNewswire/ -- The Foundation Fighting Blindness, the world's leading organization committed to finding treatments and cures for blinding retinal diseases, is committing at least $8.6 million for its Uni-Rare Study, a new natural history study for approximately 1,500 people with one of more than 300 rare genes associated with inherited retinal diseases (IRDs) including: retinitis pigmentosa, Leber congenital amaurosis, Usher syndrome, and a broad range of other conditions.

Key Points: 
  • "A majority of the 300 genes linked to inherited retinal diseases have not been well characterized in the clinic.
  • "We are excited to launch a highly inclusive study to benefit a large segment of the population affected by IRDs, which are so genetically diverse."
  • Established in 1971, the Foundation Fighting Blindness is the world's leading private funding source for retinal degenerative disease research.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.

MeiraGTx Reports Second Quarter 2022 Financial and Operational Results

Retrieved on: 
Thursday, August 11, 2022

LONDON and NEW YORK, Aug. 11, 2022 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical-stage gene therapy company, today announced financial results for the second quarter ended June 30, 2022, and provided an update on recent progress.

Key Points: 
  • LONDONandNEW YORK, Aug. 11, 2022 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc(Nasdaq: MGTX), a vertically integrated, clinical-stage gene therapy company, today announced financial results for the second quarter ended June 30, 2022, and provided an update on recent progress.
  • Full MGT009 data will be presented at the AAO annual meeting, being held September 30 October 3, 2022, in Chicago, IL.
  • The Company will present new data from its gene regulation platforms at medical meetings in the second half of 2022.
  • The Company believes it will have sufficient capital to fund operating expenses and capital expenditure requirements into the fourth quarter of 2024.

AGTC to Present at Upcoming American Society of Retina Specialists Annual Meeting

Retrieved on: 
Thursday, July 7, 2022

Each presentation will be shared during the American Society of Retina Specialists (ASRS) Annual Meeting, taking place July 13-16 in New York City.

Key Points: 
  • Each presentation will be shared during the American Society of Retina Specialists (ASRS) Annual Meeting, taking place July 13-16 in New York City.
  • We are honored to join our colleagues in the ophthalmology community, during this important retina meeting, to present data from the exciting gene therapies we are developing at AGTC as potential treatments for both ACHM and XLRP, said Dr. Susan Schneider, Chief Medical Officer of AGTC.
  • We look forward to sharing future updates as we continue to progress these assets through their respective clinical development paths.
  • Also, forward-looking statements represent managements plans, estimates, assumptions and beliefs only as of the date of this release.

AGTC to Present Three-Month Interim Results from the SKYLINE Clinical Trial of AGTC-501 for the Treatment of X-Linked Retinitis Pigmentosa at the 45th Macula Society Annual Meeting

Retrieved on: 
Monday, June 6, 2022

We look forward to sharing future updates as we continue to progress AGTC-501 through the Vista Phase 2/3 clinical trial and towards a potential BLA submission.

Key Points: 
  • We look forward to sharing future updates as we continue to progress AGTC-501 through the Vista Phase 2/3 clinical trial and towards a potential BLA submission.
  • Presentation details are as follows:
    X-linked Retinitis Pigmentosa (XLRP) is an inherited condition that causes progressive vision loss in boys and young men.
  • Characteristics of the disease include night blindness in early childhood and progressive constriction of the visual field.
  • AGTCs most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with inherited retinal diseases.