Leber congenital amaurosis

Opus Genetics Announces Completion of Dosing in First Cohort of Phase 1/2 Trial of Gene Therapy OPGx-LCA5 in Patients with Rare Inherited Retinal Disease LCA5

Retrieved on: 
Tuesday, March 26, 2024
Opus, Retina, Leber congenital amaurosis, University of Pennsylvania, VR, Genetics, LCA5, University, Patient, Ophthalmology, LCA, Penn Presbyterian Medical Center, Pharmaceutical industry

RESEARCH TRIANGLE PARK, N.C., March 26, 2024 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first, clinical-stage gene therapy company developing treatments for inherited retinal diseases, today announced that the first cohort has completed dosing in its open-label, dose-escalation Phase 1/2 clinical trial evaluating the subretinal delivery of OPGx-LCA5, an adeno-associated virus 8 (AAV8) vector designed to precisely deliver a functional LCA5 gene to the outer retina in patients with Leber congenital amaurosis (LCA) resulting from biallelic mutations in the LCA5 gene (LCA5).

Key Points: 
  • Based on positive safety and efficacy data from the first cohort of three adult patients, the Company will advance OPGx-LCA5 into the next highest dose.
  • Opus anticipates initiating the next cohort mid-2024.
  • There are also future plans to expand the study population to include subjects 13 years or older.
  • “Based on these early clinical data, we’re excited for the potential of OPGx-LCA5 to transform the lives of patients affected by LCA5,” said Ben Yerxa, Ph.D., chief executive officer of Opus.

BlueRock Therapeutics and Foundation Fighting Blindness announce collaboration to expand the Uni-Rare natural history study of patients living with inherited retinal diseases

Retrieved on: 
Monday, March 25, 2024
Retina, Research, Gene, Development, IRD, Leber congenital amaurosis, Retinitis pigmentosa, Paratriathlon, Patient, Bayer, Foundation Fighting Blindness, Visual impairment, University, Wilfrid Sellars, Senior, IND, Therapy, IPSC, Usher syndrome, B cell, Pharmaceutical industry

The new cohort will include patients living with IRDs caused by mutations in multiple genes.

Key Points: 
  • The new cohort will include patients living with IRDs caused by mutations in multiple genes.
  • “BlueRock is developing a pipeline of cell therapies that we believe has great potential for restoring vision in people living with blindness caused by retinal disease,” said Ahmed Enayetallah, Senior Vice President and Head of Development for BlueRock Therapeutics.
  • Primary photoreceptor diseases are a subgroup of inherited retinal diseases that includes retinitis pigmentosa and cone- rod dystrophies.
  • OpCT-001 aims to restore vision loss caused by these diseases by replacing degenerated tissue in the retina with functional cells.

Foundation Fighting Blindness Hosting Webinar for Eye Care Professionals: Best Clinical Practices for Patients with Inherited Retinal Diseases

Retrieved on: 
Tuesday, February 13, 2024
Therapy, Retinitis pigmentosa, Leber congenital amaurosis, Caregiver, Doctor of Philosophy, Ear, SCCO, University of Iowa, MD, Optometry, Brain damage, Genetic testing, University, Foundation Fighting Blindness, Visual impairment, Stargardt disease, Genetics, OD, Patient, IRD, Rehabilitation, Harvard Medical School, Marshall B. Ketchum University, Science, Usher syndrome, Research, Acquired brain injury, Nursing

COLUMBIA, Md., Feb. 13, 2024 /PRNewswire/ -- The Foundation Fighting Blindness, the driving force in the global development of treatments and cures for blinding diseases, will host a webinar for eye care professionals on the best practices for managing their patients with inherited retinal diseases (IRDs), including retinitis pigmentosa, Usher syndrome, Stargardt disease, and Leber congenital amaurosis. This webinar will be a part of the March Envisioning a Path to Hope initiative, raising awareness among eye care professionals for the valuable resources the Foundation provides IRD patients and caregivers.

Key Points: 
  • This webinar will be a part of the March Envisioning a Path to Hope initiative, raising awareness among eye care professionals for the valuable resources the Foundation provides IRD patients and caregivers.
  • "With nearly 50 clinical trials underway for emerging IRD therapies and no-cost genetic testing available for patients, eye care professionals can do so much to help IRD patients on their challenging journeys," says Michelle Glaze, director of professional outreach, Foundation Fighting Blindness.
  • "We are delighted to host this informative webinar to empower eye doctors to communicate hope and a path forward for their IRD patients and families."
  • She treats patients in the clinical departments of Acquired Brain Injury and Low Vision Rehabilitation, where she also conducts genetic testing for inherited eye conditions.

Atsena Therapeutics Receives Rare Pediatric Disease Designation from FDA for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)

Retrieved on: 
Tuesday, January 16, 2024
Patient, Biologics license application, Therapy, Leber congenital amaurosis, RPD, Regenerative Medicine Advanced Therapy, MBA, Food, Priority review, Paratriathlon, FDA, Pharmaceutical industry

The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1.

Key Points: 
  • The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1.
  • RPD designation is granted by the FDA for serious or life-threatening diseases which affect fewer than 200,000 people in the United States and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age.
  • “Rare Pediatric Disease designation is a significant milestone for our LCA1 program as we explore options to advance ATSN-101 into a pivotal clinical trial,” said Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics.
  • ATSN-101 has demonstrated clinically meaningful improvements in vision at the highest dose and is well-tolerated 12 months post-treatment.

Ocugen Announces OCU400 Receives Regenerative Medicine Advanced Therapy (RMAT) Designation for Treatment of Retinitis Pigmentosa Associated with RHO Mutations

Retrieved on: 
Tuesday, December 19, 2023
Marketing, Metabolism, Homeostasis, FDA, Vaccine, Retinitis pigmentosa, Regenerative medicine, Trial of the century, RP, Doctor of Philosophy, Mutation, RHO, LCA, Reproduction, Patient, Safety, Paratriathlon, Leber congenital amaurosis, Pharmaceutical industry

“FDA’s decision also reinforces the sense of urgency to bring a therapeutic option to these patients.”

Key Points: 
  • “FDA’s decision also reinforces the sense of urgency to bring a therapeutic option to these patients.”
    RMAT designation is part of the 21st Century Cures Act.
  • The program was created to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse, or cure a serious condition.
  • Receiving RMAT designation offers sponsor companies all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with the FDA.
  • RHO mutations affect more than 10,000 of the 110,000 people in the United States diagnosed with RP.

Atsena Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1)

Retrieved on: 
Tuesday, November 14, 2023
Leber congenital amaurosis, Visual impairment, Food, Hope, Therapy, Paratriathlon, BLA, 21st Century Cures Act, Disease, Patient, FDA, MBA, Drug development, Pharmaceutical industry

DURHAM, N.C., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101, the company’s lead investigational gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). RMAT designation was granted based on positive 6-month efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101.

Key Points: 
  • DURHAM, N.C., Nov. 14, 2023 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101, the company’s lead investigational gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1).
  • RMAT designation was granted based on positive 6-month efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101.
  • "RMAT designation is encouraging recognition of the potential of ATSN-101 to be an important treatment and provides hope to children and adults affected by LCA1."
  • Atsena has also received orphan drug designation from the FDA for ATSN-101 for the treatment of LCA1.

New Preliminary Clinical Data on Potential of Opus AAV-based Gene Therapy for Rare Inherited Retinal Disease to be Presented at the American Academy of Ophthalmology Annual Conference 2023

Retrieved on: 
Friday, November 3, 2023
Opus, Retinal degeneration (rhodopsin mutation), Genetics, MD, Leber congenital amaurosis, Stem cell, LCA5, American Academy, Gene, Retina, American Academy of Ophthalmology, LCA, Pharmaceutical industry, Ophthalmology

RALEIGH, N.C., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced that preliminary data from a Phase 1/2 clinical trial evaluating the potential of its gene therapy to address Leber congenital amaurosis (LCA) due to mutations in LCA5 will be presented in a symposium at the American Academy of Ophthalmology annual conference held November 3-6 in San Francisco.

Key Points: 
  • RALEIGH, N.C., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced that preliminary data from a Phase 1/2 clinical trial evaluating the potential of its gene therapy to address Leber congenital amaurosis (LCA) due to mutations in LCA5 will be presented in a symposium at the American Academy of Ophthalmology annual conference held November 3-6 in San Francisco.
  • The data will be presented as part of a broader presentation by Tomas S. Aleman, MD, the principal investigator of the trial, focused on gene augmentation therapies for the treatment of inherited retinal degenerations.
  • Details of the presentation are as follows:
    Session: The Future of Retinal Disease Pharmacological, Stem Cell and Gene Therapy Treatments

Ocugen to Present at the 2023 Cell & Gene Meeting on the Mesa

Retrieved on: 
Wednesday, October 4, 2023
Vaccine, LCA, Policy, Retinitis pigmentosa, RP, Mesa, Cell, News, Leber congenital amaurosis, Pharmaceutical industry

MALVERN, Pa., Oct. 04, 2023 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen will present at the 2023 Cell & Gene Meeting on the Mesa being held October 10-12, 2023 in Carlsbad, CA at the Park Hyatt Aviara Resort.

Key Points: 
  • MALVERN, Pa., Oct. 04, 2023 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen will present at the 2023 Cell & Gene Meeting on the Mesa being held October 10-12, 2023 in Carlsbad, CA at the Park Hyatt Aviara Resort.
  • Dr. Musunuri will provide an overview of Ocugen’s clinical development programs, including the business strategy across its unique gene and cell therapy platforms, anticipated milestones, and more detail about the Company’s recent positive Phase 1/2 OCU400 data results.
  • Details of the presentation are as follows:
    “The Cell & Gene Meeting on the Mesa convenes leading industry professionals confronting major scientific, logistic, policy, and economic questions in cell and gene therapy,” said Dr. Musunuri.
  • Please visit https://meetingonthemesa.com for full information including registration.

Ocugen Announces Positive Clinical Study Update from the Phase 1/2 Trial of OCU400, a Modifier Gene Therapy Product Candidate, for the Treatment of Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA)

Retrieved on: 
Wednesday, September 13, 2023
Vaccine, Retina, IRD, Schepens Eye Research Institute, CEP290, LCA, Cohort, Biotechnology, Eye, Clinical trial, Duke University School of Medicine, Quality of life, Hope, Mutation, Research, Paratriathlon, DSM-IV codes, Retinitis pigmentosa, RP, Associate, Trial of the century, Șaeș, NR2E3, Development, Patient, Webcast, Married to Medicine: Los Angeles, Ageing, RHO, Principal, Leber congenital amaurosis, Safety, Pharmaceutical industry, Medical device, Television, Rhodopsin, Medicine, Ophthalmology

This clinical study update is an extension of results provided by Ocugen on April 14, 2023, and includes additional subjects from the high dose group.

Key Points: 
  • This clinical study update is an extension of results provided by Ocugen on April 14, 2023, and includes additional subjects from the high dose group.
  • Ocugen further expanded this Phase1/2 trial to enroll LCA patients with CEP290 gene mutation and pediatric patients with NR2E3, RHO and CEP290 mutations.
  • Our team's unwavering dedication to advancing modifier gene therapy research demonstrated positive preliminary clinical results that offer renewed hope to patients and their families.
  • “In my view, the clinical study update supports the gene-agnostic mechanism of action of OCU400 in RHO patients.

Ocugen to Host Virtual Investor & Analyst Event on September 13, 2023

Retrieved on: 
Wednesday, September 13, 2023
Vaccine, Medical Affairs Bureau, Schepens Eye Research Institute, LCA, Duke University School of Medicine, Retinitis pigmentosa, Research, Doctor of Philosophy, RP, Associate, Webcast, MPH, Leber congenital amaurosis, Pharmaceutical industry, Ophthalmology, MD, Medicine

MALVERN, Pa., Sept. 12, 2023 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that it will host an Investor and Analyst Event on September 13, 2023 at 8:30 a.m.

Key Points: 
  • MALVERN, Pa., Sept. 12, 2023 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that it will host an Investor and Analyst Event on September 13, 2023 at 8:30 a.m.
  • ET.
  • During the webcast and conference call, members of the Ocugen leadership team and key opinion leaders will discuss updated results from the Phase 1/2 trial of OCU400 for the treatment of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA).
  • Huma Qamar, MD, MPH, Head of Clinical Development and Medical Affairs, Ocugen
    David Birch, PhD, Scientific Director, Retina Foundation of the Southwest, Principal investigator of the study
    Lejla Vajzovic, MD, FASRS, Associate Professor of Ophthalmology with Tenure, Director of Duke Vitreoretinal Fellowship Program at Duke Eye Center and Duke University School of Medicine and leader in gene-therapy research