Stargardt disease

Nanoscope Therapeutics Announces Presentations at the ARVO Annual Meeting

Retrieved on: 
Thursday, May 2, 2024
Macular degeneration, Patient, Stargardt disease

Abstract Title: Longitudinal BCVA Analysis of Patients with Stargardt Disease and Macular Degeneration Treated With MCO-010, a Mutation-Agnostic Optogenetic Therapy: 48-Week Results From a Phase 2a Clinical Trial (STARLIGHT)

Key Points: 

Abstract Title: Longitudinal BCVA Analysis of Patients with Stargardt Disease and Macular Degeneration Treated With MCO-010, a Mutation-Agnostic Optogenetic Therapy: 48-Week Results From a Phase 2a Clinical Trial (STARLIGHT)

Ocugen to Present on Modifier Gene Therapy Platform at Association for Research in Vision and Ophthalmology 2024 Annual Meeting

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Friday, April 26, 2024
Stargardt disease, The Association, Safety, Retinitis pigmentosa, Armada, NR2E3, Gardian, Research, Doctor of Philosophy, Vaccine, Retina, Research and development, Patient, Geographic atrophy, Clinical trial, Annual general meeting, ARVO, Pharmaceutical industry

MALVERN, Pa., April 26, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, and vaccines, today announced that the Company will present on its innovative modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa (Phase 3 LiMeliGhT clinical trial), OCU410 for the treatment of geographic atrophy (Phase 1/2 ArMaDa clinical trial), and OCU410ST for the treatment of Stargardt disease (Phase 1/2 GARDian clinical trial) at The Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting in Seattle, WA from May 5-9, 2024.

Key Points: 
  • MALVERN, Pa., April 26, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, and vaccines, today announced that the Company will present on its innovative modifier gene therapy platform, including OCU400 for the treatment of retinitis pigmentosa (Phase 3 LiMeliGhT clinical trial), OCU410 for the treatment of geographic atrophy (Phase 1/2 ArMaDa clinical trial), and OCU410ST for the treatment of Stargardt disease (Phase 1/2 GARDian clinical trial) at The Association for Research in Vision and Ophthalmology (ARVO) 2024 Annual Meeting in Seattle, WA from May 5-9, 2024.
  • “With three gene therapies to treat blindness diseases currently in the clinic, now is an exciting time for Ocugen and the patients who can potentially benefit from our first-in-class modifier gene therapy platform,” said Dr. Shankar Musunuri, Chairman, CEO and Co-founder of Radius.
  • “We look forward to sharing more about the scientific foundation of our programs and providing clinical updates with industry leaders during ARVO.”
    Ocugen’s presence at ARVO 2024 includes:
    Title: OCU400 Nuclear Hormone Receptor-Based Gene Modifier Therapy: Safety and Efficacy from Phase 1/2 Clinical Trial for Retinitis Pigmentosa Associated with NR2E3 and RHO Mutations
    Presenter: Arun Upadhyay, PhD, Chief Scientific Officer, Head of Research & Development, Ocugen
    Ocugen is committed to bringing game-changing therapies to treat inherited retinal diseases as well as blindness diseases affecting millions to market and working even harder to provide access to patients globally.

AAVantgarde announces updated NHP data from its Stargardt disease program in a poster presentation at the ARVO 2024 annual meeting

Retrieved on: 
Wednesday, April 24, 2024
Ophthalmology, Safety, Stargardt, Research, AAV, CSO, NHP, ABCA4, Stargardt disease, ARVO, Vaccine

MILAN, Italy, April 24, 2024 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage, Italian-based international biotechnology company with two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery, today announces the publication of an abstract submitted to The Association for Research in Vision and Ophthalmology Annual Meeting (ARVO), to be held May 5-9 in Seattle.

Key Points: 
  • MILAN, Italy, April 24, 2024 (GLOBE NEWSWIRE) -- AAVantgarde Bio (AAVantgarde), a clinical-stage, Italian-based international biotechnology company with two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery, today announces the publication of an abstract submitted to The Association for Research in Vision and Ophthalmology Annual Meeting (ARVO), to be held May 5-9 in Seattle.
  • “We are delighted to be presenting positive NHP safety and efficacy data from our Stargardt program demonstrating that AAV.ABCA4.intein vectors can be administered safely to NHPs under an immunosuppressive protocol designed for use in man, and at doses showing robust expression matching the pattern of endogenous ABCA4.
  • It is encouraging to see that our AAV.ABCA4.intein product shows potential to treat retinal dystrophies, such as Stargardt Disease,” said Prof. Alberto Auricchio, CSO of AAVantgarde.
  • Dr. Natalia Misciattelli, CEO of AAVantgarde explained that “This positive data in NHP is very encouraging as it shows the potential of our Stargardt program, and it provides hope to the underserved Stargardt patients that currently don’t have many therapeutic options available.”
    Presentation Number / Posterboard Number: 6097 - B0943

AAVantgarde presents positive preclinical data in large animal models from its Stargardt disease program in an oral presentation at the ASGCT 2024 annual meeting

Retrieved on: 
Tuesday, April 23, 2024
Stargardt disease, Safety, Stargardt, Cell, AAV, Messenger, Lipofuscin, Gene, Society, CSO, NHP, Vaccine

The data to be presented confirms that AAV intein-mediated retinal gene therapy for Stargardt disease is effective and safe in large animal models (pig and NHP).

Key Points: 
  • The data to be presented confirms that AAV intein-mediated retinal gene therapy for Stargardt disease is effective and safe in large animal models (pig and NHP).
  • In pigs, AAVantgarde demonstrate that lipofuscin accumulation in the retinal pigmented epithelium was reduced upon subretinal delivery of AAV-ABCA4 intein vectors.
  • Similarly, in NHP, BaseScope analysis showed nearly total photoreceptor co-expression of mRNAs encoding both ABCA4-intein halves across an extended NHP retinal region.
  • Prof. Alberto Auricchio, CSO of AAVantgarde stated “We are very excited to be presenting positive Pig and NHP safety and efficacy data from our Stargardt program as an oral presentation, supporting both the efficacy and safety of AAV-ABCA4-intein vectors in relevant large animal models, and providing important insights towards the clinical translation of this platform for gene therapy of STGD1.”

Nanoscope Therapeutics Enhances Mutation-Independent Retinal Gene Therapy Programs with Appointment of Allen C. Ho, MD, as Chief Medical Advisor

Retrieved on: 
Thursday, April 25, 2024
Stargardt disease, RP, State, Stargardt, Research, Diabetic retinopathy, Communication, FACS, Thomas Jefferson University, Patient, Geographic atrophy, American Academy of Ophthalmology, Clinical trial, Therapy, AAO, Medical device

DALLAS, April 25, 2024 /PRNewswire/ -- Nanoscope Therapeutics, Inc. , a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, announced today the appointment of Allen C. Ho, MD, FACS, FASRS, as Chief Medical Advisor, where he will help define the strategy of developing the gene mutation-agnostic therapies.

Key Points: 
  • DALLAS, April 25, 2024 /PRNewswire/ -- Nanoscope Therapeutics, Inc. , a clinical-stage biotechnology company developing gene therapies for retinal degenerative diseases, announced today the appointment of Allen C. Ho, MD, FACS, FASRS, as Chief Medical Advisor, where he will help define the strategy of developing the gene mutation-agnostic therapies.
  • Dr. Ho is Attending Surgeon and Director of Retina Research at Wills Eye Hospital and Co-Director of the Wills Eye Hospital Retina Service.
  • "We are excited to have Dr. Allen C. Ho join Nanoscope as Chief Medical Advisor," said Sulagna Bhattacharya, Nanoscope co-founder and CEO.
  • "As a world-renowned retina specialist and leader in innovative treatments for retinal diseases, his insight will be invaluable to Nanoscope.

Prevent Blindness Declares May as First-ever Inherited Retinal Disease (IRD) Genetic Testing Awareness Month

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Wednesday, April 24, 2024
Education, Paratriathlon, University, Blood, Birth defect, Retina, RP, Social media, Diagnosis, Stargardt disease, Stargardt, Marketing, Strabismus, Genetic, Johnson & Johnson, Gene, Spark Therapeutics, Choroid, Research, Visual impairment, Genetic testing, Pediatric ophthalmology, Disease, Visual Science, Patient, Eye, Cone dystrophy, MBA, Vision, Student, Therapy, Organization, IRD, Pharmaceutical industry

CHICAGO, April 24, 2024 /PRNewswire-PRWeb/ -- Prevent Blindness, the nation's leading nonprofit eye health and safety organization, has designated May as "Inherited Retinal Disease (IRD) Genetic Testing Awareness Month," expanding its previous IRD and Genetic Testing Awareness Week initiative. The group is providing a variety of tools to promote awareness and education for IRDs and the importance of genetic testing, including a free webinar, expert and patient videos, shareable social media graphics, and fact sheets in English and Spanish. IRD Genetic Testing Awareness Month is supported by funding from Johnson & Johnson, and Spark® Therapeutics.

Key Points: 
  • Prevent Blindness expands IRD and genetic testing awareness initiative to educate patients on various forms of IRDs, and the importance of genetic testing to confirm diagnosis and possible treatments.
  • CHICAGO, April 24, 2024 /PRNewswire-PRWeb/ -- Prevent Blindness , the nation's leading nonprofit eye health and safety organization, has designated May as "Inherited Retinal Disease (IRD) Genetic Testing Awareness Month," expanding its previous IRD and Genetic Testing Awareness Week initiative.
  • ET, the National Center for Children's Vision and Eye Health at Prevent Blindness will be hosting the free webinar, " Pathway to Diagnosis: Genetic Testing for Inherited Retinal Diseases ."
  • To register for the "Pathway to Diagnosis: Genetic Testing for Inherited Retinal Diseases" webinar, visit PreventBlindness.org/IRD-webinar-2024.

SalioGen Therapeutics Announces Data Presentations at 2024 Annual Meetings for the Association for Research in Vision and Ophthalmology (ARVO) and American Society of Gene & Cell Therapy (ASGCT)

Retrieved on: 
Monday, April 22, 2024
Stargardt disease, Safety, Abstract, Research, Cystic fibrosis, CFTR, Cell, DNA, Coding, Gene, Society, Paratriathlon, Genome, Therapy, Biotechnology, ARVO

LEXINGTON, Mass., April 22, 2024 /PRNewswire/ -- SalioGen Therapeutics, a biotechnology company developing next-generation genetic medicines based on its novel Gene Coding™ technology, today announced that four abstracts, including one oral presentation, will be presented at the upcoming annual meetings of the Association for Research in Vision and Ophthalmology (ARVO) and the American Society of Gene & Cell Therapy (ASGCT).

Key Points: 
  • The presented findings will highlight preclinical data supporting the tolerability and in vivo efficacy of SGT-1001, a development candidate for the one-time treatment of Stargardt disease, a genetic condition that causes progressive vision loss.
  • A separate program under development for cystic fibrosis will show success in targeting integration of the CFTR gene into the native CFTR intron 1, a major step forward in targeted delivery of a large genetic cargo.
  • SalioGen will also share genomic profiling data on its novel Gene Coding technology, which uses transposition to integrate large DNA constructs (up to 100kb) into the genome and promises to overcome key safety risks and limitations of other approaches to genetic medicine.
  • ARVO will take place in Seattle, Washington, from May 5 to 9, 2024, and ASGCT will take place in Baltimore, Maryland, from May 7 to 11, 2024.

Ocugen Provides Business Update with Certain Financials for the Year Ending 2023

Retrieved on: 
Tuesday, April 2, 2024
Stargardt disease, Disease, Safety, Webcast, Inflammation, Patient, CGMP, Data, Inhalation, Cohort, RP, Life, Bank statement, Investment, Clinical trial, NIAID, Injection, AMD, Vaccine, Nextgen, Geographic atrophy, RHO, FDA, Lipid metabolism, Government, Oxidative stress, Pharmaceutical industry

Completed Cohort 1 dosing for OCU410 and OCU410ST gene therapy clinical studies for geographic atrophy (GA) and Stargardt disease, respectively

Key Points: 
  • Completed Cohort 1 dosing for OCU410 and OCU410ST gene therapy clinical studies for geographic atrophy (GA) and Stargardt disease, respectively
    MALVERN, Pa., April 02, 2024 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today provided a general business update with certain financials for the year ending 2023.
  • During the fourth quarter of 2023, the Company announced its mucosal vaccine candidate, OCU500, was chosen for the multi-billion-dollar NIAID Project NextGen initiative.
  • The first patient was dosed in the Phase 1/2 trials to assess the safety and efficacy of OCU410ST for Stargardt disease in November 2023.
  • Ocugen’s cash, cash equivalents, and investments totaled $39.5 million as of December 31, 2023, compared to $90.9 million as of December 31, 2022.

Ocugen Announces Positive Data and Safety Monitoring Board Review and Initiation of Enrollment in Medium Dose for OCU410ST—a Modifier Gene Therapy—in GARDian Study for Stargardt Disease

Retrieved on: 
Monday, April 1, 2024
Stargardt disease, Gardian, Patient, Cohort, Șaeș, Clinical trial, MPH, Safety, DSMB, FACS, Vaccine, FDA, Pharmaceutical industry

Three patients with Stargardt disease were dosed in the Phase 1/2 clinical trial to date.

Key Points: 
  • Three patients with Stargardt disease were dosed in the Phase 1/2 clinical trial to date.
  • An additional three patients will be dosed with the medium dose (Cohort 2) and three patients with the high dose (Cohort 3) of OCU410ST in the dose-escalation phase.
  • “The DSMB has recommended moving forward to medium dose for dosing subjects with Stargardt disease,” said Dr. Peter Chang, MD, FACS, DSMB Chair for the OCU410ST clinical trial.
  • Phase 1 is a multicenter, open-label, dose ranging study consisting of three dose levels [low dose (3.75×1010 vg/mL), medium dose (7.5×1010 vg/mL), and high dose (2.25×1011 vg/mL)].

KALA BIO Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Corporate Update

Retrieved on: 
Friday, March 29, 2024
Retina, Chair, Three Months, Biologics license application, Secretome, Disease, Infection, Incidence, Paratriathlon, Inflammation, Patient, Sale, Limbal stem cell, Research, CIRM, Acquisition, Commercial, Eye, Safety, Retinitis pigmentosa, Food, FDA, BLA, Stargardt disease, Physician, Contingency, Protease, Cryptocurrency

ARLINGTON, Mass., March 29, 2024 (GLOBE NEWSWIRE) -- KALA BIO, Inc. (NASDAQ:KALA), a clinical-stage biopharmaceutical company dedicated to the research, development and commercialization of innovative therapies for rare and severe diseases of the eye, today reported financial results for the fourth quarter and full year ended December 31, 2023 and provided a corporate update.

Key Points: 
  • “In 2023, we focused on clinical execution, advancing our Phase 2b CHASE trial of KPI-012 for the treatment of PCED.
  • In December 2023 and March 2024, KALA announced private placement financings with an institutional investor, priced at-the-market under Nasdaq rules.
  • Cash Position: As of December 31, 2023, KALA had cash and cash equivalents of $50.9 million, compared to $56.1 million as of September 30, 2023.
  • Operating Loss: For the quarter ended December 31, 2023, loss from operations was $9.6 million, compared to $10.3 million for the same period in 2022.