Leber congenital amaurosis

Editas Medicine Announces Second Quarter 2022 Results and Business Updates

Retrieved on: 
Wednesday, August 3, 2022
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This enables Editas Medicine to include patients efficacy data in a marketing application for EDIT-301 in the future.

Key Points: 
  • This enables Editas Medicine to include patients efficacy data in a marketing application for EDIT-301 in the future.
  • Editas Medicine remains on track to provide a clinical update on the BRILLIANCE trial in the second half of 2022.
  • The increase was primarily attributable to the additional program licensed by Bristol Myers Squibb in the second quarter of 2022.
  • ET to provide and discuss a corporate update and financial results for the second quarter of 2022.

McKinsey expands network of Digital Capability Centers in partnership with New Jersey Innovation Institute (NJII) with advanced biopharma manufacturing learning facility in Newark

Retrieved on: 
Saturday, July 16, 2022
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NEWARK, N.J., July 15, 2022 /PRNewswire/ -- McKinsey & Company launched a new high-tech learning and innovation facility today in the Garden State to support the advancement of cell and gene therapy (CGT) manufacturing, expanding its global network of Digital Capability Centers.

Key Points: 
  • In partnership with the New Jersey Innovation Institute (NJII) and operated by BioCentriq, McKinsey's Newark-based facility specializes in innovative manufacturing processes in life sciences.
  • This facility will help biopharma companies sustain higher levels of innovation and accessibility by helping to solve their operational challenges."
  • In addition to the 250 McKinsey experts involved in this global network, the Digital Capability Center network also has partnerships with 150+ tech companies and startups.
  • The Digital Capability Center New Jersey will be located on the Newark campus of the New Jersey Institute of Technology.

Opus Genetics to Present at OIS Retina Innovation Summit 2022

Retrieved on: 
Monday, July 11, 2022
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RESEARCH TRIANGLE PARK, N.C., July 11, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced that Ben Yerxa, Ph.D., president and CEO of Opus, will present at the Ophthalmology Innovation Source (OIS) Retina Innovation Summit 2022 on Wednesday, July 13, 2022, in New York.

Key Points: 
  • RESEARCH TRIANGLE PARK, N.C., July 11, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-first gene therapy company developing treatments for inherited retinal diseases, today announced that Ben Yerxa, Ph.D., president and CEO of Opus, will present at the Ophthalmology Innovation Source (OIS) Retina Innovation Summit 2022 on Wednesday, July 13, 2022, in New York.
  • Were honored to be selected to participate in the OIS Innovation Showcase and highlight Opus patient-first, science-driven approach and pipeline of AAV-based gene therapy programs, said Dr. Yerxa.
  • The OIS Retina Innovation Summit brings together entrepreneurs, ophthalmic start-up companies, clinical thought leaders, industry executives and investment professionals for a day-long summit showcasing novel therapies in development for ophthalmic diseases and vision disorders.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.

Prevent Blindness Declares Third Annual Inherited Retinal Disease (IRD) Genetic Testing Week as May 16-22

Retrieved on: 
Wednesday, May 11, 2022
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CHICAGO, May 11, 2022 /PRNewswire-PRWeb/ -- Prevent Blindness, the nation's leading nonprofit eye health and safety organization, has declared May 16-22, 2022 as the third annual "Inherited Retinal Disease (IRD) Genetic Testing Week." Educational materials, such as a fact sheet, social media graphics, and a dedicated webpage, preventblindness.org/inherited-retinal-diseases/, are available.

Key Points: 
  • CHICAGO, May 11, 2022 /PRNewswire-PRWeb/ -- Prevent Blindness , the nation's leading nonprofit eye health and safety organization, has declared May 16-22, 2022 as the third annual "Inherited Retinal Disease (IRD) Genetic Testing Week."
  • For the third consecutive year, IRD Genetic Testing Week at Prevent Blindness is supported by Spark Therapeutics , a fully integrated company and member of the Roche group dedicated to challenging the inevitability of genetic disease.
  • Inherited Retinal Diseases, also referred to as inherited retinal disorders, are a group of diseases that can cause severe vision loss or even blindness.
  • "Through early detection, specifically through genetic testing, and treatment, severe vision loss and blindness from Inherited Retinal Diseases can be avoided in many cases," said Jeff Todd, president and CEO of Prevent Blindness.

Opus Genetics Appoints Jennifer Hunt Chief Development Officer

Retrieved on: 
Wednesday, May 11, 2022
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RESEARCH TRIANGLE PARK, N.C., May 11, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced the appointment of Jennifer Hunt as Chief Development Officer (CDO).

Key Points: 
  • RESEARCH TRIANGLE PARK, N.C., May 11, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced the appointment of Jennifer Hunt as Chief Development Officer (CDO).
  • Hunt brings over 25 years of drug development experience to Opus, with specific expertise in global clinical operations, product development and program management in biologics, small molecules, gene editing and gene therapy.
  • Prior to joining Opus, she held key clinical and regulatory positions at several biopharmaceutical companies, including Genzyme, Voyager Therapeutics and Editas Medicine.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.

Opus Genetics Announces Promising New Data Highlighting Potential of AAV-based Gene Therapies for the Treatment of Rare Inherited Retinal Diseases

Retrieved on: 
Wednesday, May 4, 2022
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RESEARCH TRIANGLE PARK, N.C., May 04, 2022 (GLOBE NEWSWIRE) -- Opus Genetics, a patient-focused gene therapy company developing treatments for inherited retinal diseases, today announced promising new preclinical data from studies evaluating the potential of its gene therapies OPGx-001 and OPGx-002 to address forms of Leber congenital amaurosis (LCA), a group of rare inherited retinal diseases characterized by photoreceptor degeneration, due to mutations of LCA5 or RDH12 genes, respectively.

Key Points: 
  • In preparation for IND-enabling trials of OPGx-001 and OPGx-002, studies were conducted to determine eligibility, therapeutic window, and possible outcome measures for gene therapy for LCA5 and RDH12 inherited retinal diseases.
  • In addition, safety evaluations for the subretinal delivery of an AAV8 vector containing LCA5 or RDH12 were performed in non-human primates (NHP).
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.
  • Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies.

New Data on Potential of Opus AAV-based Gene Therapies for Rare Inherited Retinal Diseases to be Presented at Association for Research in Vision and Ophthalmology Annual Meeting 2022

Retrieved on: 
Thursday, April 28, 2022
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The annual summit, held prior to the ARVO Annual Meeting, features presentations by leading retinal disease experts on potential gene and stem-cell therapies and how best to deliver them to patients.

Key Points: 
  • The annual summit, held prior to the ARVO Annual Meeting, features presentations by leading retinal disease experts on potential gene and stem-cell therapies and how best to deliver them to patients.
  • Opus Genetics is a groundbreaking gene therapy company for inherited retinal diseases with a unique model and purpose.
  • Backed by Foundation Fighting Blindnesss venture arm, the RD Fund, Opus combines unparalleled insight and commitment to patient need with wholly owned programs in numerous retinal diseases.
  • Its AAV-based gene therapy portfolio tackles some of the most neglected forms of inherited blindness while creating novel manufacturing scale and efficiencies.

ProQR to Present at the Retinal Cell and Gene Therapy Innovation Summit and the Association for Research in Vision and Ophthalmology (ARVO) 2022

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Tuesday, April 26, 2022
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I look forward to continuing to work with ProQR on this investigational treatment.

Key Points: 
  • I look forward to continuing to work with ProQR on this investigational treatment.
  • Leber congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children.
  • LCA10 is caused by mutations in the CEP290 gene, of which the c.2991+1655A>G (p.Cys998X) mutation has the highest prevalence.
  • LCA10 leads to early loss of vision causing most people to lose their sight in the first few years of life.

Editas Medicine Announces Dosing of First Pediatric Patient in the BRILLIANCE Clinical Trial of EDIT-101 for LCA10

Retrieved on: 
Monday, April 11, 2022
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CAMBRIDGE, Mass., April 11, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced the administration of EDIT-101, an experimental CRISPR gene editing medicine, to the first pediatric patient enrolled in the BRILLIANCE clinical trial, which is designed to test the safety of EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder. This marks the world’s first in vivo, or inside the body, dosing of a pediatric patient with a CRISPR gene editing experimental medicine.

Key Points: 
  • This marks the worlds first in vivo, or inside the body, dosing of a pediatric patient with a CRISPR gene editing experimental medicine.
  • Currently, there are no approved treatments for LCA10, and we look forward to sharing future updates from the BRILLIANCE trial, including sharing additional clinical data, later this year.
  • Enrolling this first pediatric patient in the BRILLIANCE trial is an important step toward bringing potentially life-changing treatments to children with genetic retinal diseases.
  • Previously, Editas Medicine completed dosing of all adult cohorts in its BRILLIANCE study and announced preliminary EDIT-101 clinical results demonstrated a favorable safety profile and encouraging signals of clinical benefit.

Ocugen, Inc. Announces First Patient Dosed in Phase 1/2 Clinical Trial for Gene Therapy Candidate OCU400 to Treat Inherited Retinal Degeneration

Retrieved on: 
Friday, April 1, 2022
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This first patient dosing marks the beginning of the dose-escalating, observer-blind, Phase 1/2 safety and efficacy study.

Key Points: 
  • This first patient dosing marks the beginning of the dose-escalating, observer-blind, Phase 1/2 safety and efficacy study.
  • The first phase of the study is a safety evaluation of the product, eventually progressing into an efficacy study in patients.
  • Traditional gene therapy, which transfers a functional version of a non-functional gene into target cells, addresses only one individual gene mutation at a time.
  • This Phase 1/2 clinical trial targets people who have RP resulting from mutations in the NR2E3 and RHO genes.