Rhodopsin

Prime Medicine Reports Third Quarter 2023 Financial Results and Provides Business Updates

Retrieved on: 
Friday, November 3, 2023
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CAMBRIDGE, Mass., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today reported financial results and provided business updates for the third quarter ended September 30, 2023.

Key Points: 
  • 11,795,452 --
    CAMBRIDGE, Mass., Nov. 03, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, today reported financial results and provided business updates for the third quarter ended September 30, 2023.
  • Together, these encouraging results mark meaningful progress toward the clinic and support our strategy of simultaneously advancing multiple programs forward.
  • In August 2023, Prime Medicine received Rare Pediatric Drug designation (RPDD) from the U.S. Food and Drug Administration (FDA) for PM359.
  • Year-to-date, Prime Medicine continues to make significant progress, executing against key initiatives to drive its Prime Editing platform forward.

Prime Medicine Presents First In Vivo Proof-of-Concept Prime Editing Data Demonstrating Ability of Prime Editors to Treat Ophthalmological Diseases

Retrieved on: 
Tuesday, October 24, 2023
Well, International Symposium on Endovascular Therapy, AAV, Rhodopsin, RNA, Congress, Photoreceptor, PCR, Research, Retinal, RHO, Disease, Glial fibrillary acidic protein, Retina, GFAP, Eye, Paratriathlon, Patient, Gene expression, Vaccine

CAMBRIDGE, Mass., Oct. 24, 2023 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated, one-time curative genetic therapies, today reported new preclinical data demonstrating the ability of Prime Editors to efficiently and precisely correct the predominant mutations that cause rhodopsin associated autosomal dominant retinitis pigmentosa (RHO adRP). The data were presented today at the International Symposium on Retinal Degeneration 2023 Congress (RD2023) in Costa del Sol, Spain.

Key Points: 
  • The data were presented today at the International Symposium on Retinal Degeneration 2023 Congress (RD2023) in Costa del Sol, Spain.
  • “The data presented today are the first proof-of-concept data for Prime Editing’s application in treating ophthalmological indications, and highlight the ability of Prime’s novel, dual AAV delivery platform to efficiently deliver Prime Editors to the eye,” said Jeremy Duffield, M.D., Ph.D., Chief Scientific Officer of Prime Medicine.
  • In today’s presentation at RD2023, Prime Medicine shared findings from in vivo studies in humanized mice with its Prime Editors targeting these prevalent RHO mutations.
  • Efficient delivery of Prime Editors by dual AAV to human (retinal explants) and murine (in vivo) photoreceptors.

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
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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 Announces Positive Preliminary Safety and Efficacy Results from the Phase 1/2 Trial of OCU400, a Modifier Gene Therapy Product Candidate, for the Treatment of Retinitis Pigmentosa and Leber Congenital Amaurosis

Retrieved on: 
Friday, April 14, 2023
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“It is very gratifying to see these positive preliminary results from our novel modifier gene therapy approach,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen.

Key Points: 
  • “It is very gratifying to see these positive preliminary results from our novel modifier gene therapy approach,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen.
  • A total of 18 RP subjects have been enrolled in this study—10 subjects in the dose escalation and 8 subjects in the expansion phase, respectively.
  • We further expanded this Phase1/2 trial to enroll LCA patients with CEP290 gene mutation and pediatric patients with NR2E3, RHO and CEP290 mutations.
  • CanSinoBIO, Ocugen’s strategic partner, provided all CMC development and clinical supplies for the Phase 1/2 trial of OCU400.

Ocugen Announces Completion of Dosing in OCU400 Phase 1/2 Cohort 2

Retrieved on: 
Wednesday, October 12, 2022
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MALVERN, Pa., Oct. 12, 2022 (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 Independent Data and Safety Monitoring Board (DSMB) for the OCU400 Phase 1/2 clinical trial completed a review of safety data for subjects enrolled in Cohort 2 and recommends proceeding to enroll subjects in Cohort 3. The Company expects to complete Cohort 3 enrollment in Q4 2022 as planned. 

Key Points: 
  • The Company expects to complete Cohort 3 enrollment in Q4 2022 as planned.
  • Im very pleased with the progress of the clinical trial, said David Birch, PhD, Director, Retina Foundation of the Southwest, primary investigator of the study.
  • OCU400 clinical trial is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated with NR2E3 (Nuclear Receptor Subfamily 2 Group E Member 3) and RHO (Rhodopsin) Mutations.
  • OCU400 is part of Ocugens Modifier Gene Therapy Platformtargeting inherited retinal disease and dry age-related macular degeneration.

Ocugen, Inc. Announces Positive DSMB Recommendation for OCU400-101 Clinical Trial

Retrieved on: 
Monday, April 25, 2022
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The DSMB recommended that the Company continue enrolling the remaining study subjects in this current cohort at the target dose level.

Key Points: 
  • The DSMB recommended that the Company continue enrolling the remaining study subjects in this current cohort at the target dose level.
  • 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 information involves risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements.
  • Any forward-looking statements that we make in this press release speak only as of the date of this press release.

Publication in Nature by OMass Founder, Professor Dame Carol Robinson, Shows Power of Native Mass Spectrometry in Drug Discovery by Capturing GPCR Signalling in Native Membrane Environment

Retrieved on: 
Wednesday, April 6, 2022
Medicine, Therapy, Lipid, Degenerative disease, National academy, University, Royal, GPCR, Royal Society, University of Oxford, European Chemical Society, Research, Rhodopsin, Louis-Jeantet Prize for Medicine, Kavli Foundation (United States), Drug discovery, Science, Commander (order), Industry, Technology, Membrane protein, Regeneration, Ecosystem, Kavli Institute of Nanoscience, Mass spectrometry, Female, G protein-coupled receptor, Protein, Pharmaceutical industry, Fine chemical, Nature, Chemistry

The paper, for the first time, shows the power of native mass spectrometry in capturing a G protein-coupled receptor (GPCR) signalling cascade in a native membrane environment.

Key Points: 
  • The paper, for the first time, shows the power of native mass spectrometry in capturing a G protein-coupled receptor (GPCR) signalling cascade in a native membrane environment.
  • Native mass spectrometry is a core element of OMass proprietary technology platform, OdyssION, which also integrates novel biochemistry techniques and custom chemistry.
  • Professor Robinson has pioneered the field of mass spectrometry and established it as a method to analyse proteins in their native state.
  • This latest research proposes a new use of native mass spectrometry through the characterization of GPCR targets utilizing native membrane environments as opposed to purified proteins.

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.

Ocugen, Inc. Announces U.S. FDA Acceptance of Investigational New Drug Application to Initiate a Phase 1/2 Clinical Trial for Gene Therapy Candidate OCU400 to Treat Inherited Retinal Degeneration

Retrieved on: 
Thursday, December 9, 2021
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Were collaborating with leading centers in eye care and have been vital partners to getting our trial launched and receive patients.

Key Points: 
  • Were collaborating with leading centers in eye care and have been vital partners to getting our trial launched and receive patients.
  • With this final decision by the FDA, we are embarking on a new pathway of care through this innovative gene therapy.
  • Traditional gene therapy, which transfers a functional version of a non-functional gene into target cells, targets only one individual gene mutation at a time.
  • OCU400 was granted four orphan drug disease designations from the FDA for treating four different gene mutation-associated retinal degenerative diseases between 2019 and 2020.

Lineage Cell Therapeutics Presents Encouraging Data on Vision Restoration Program at Society for Neuroscience’s 49th Annual Scientific Meeting

Retrieved on: 
Wednesday, October 23, 2019
Biotechnology, Health, Stem cells, Optical, Clinical trials, Branches of biology, Biology, Emerging technologies, RTT, Eye, Blindness, Stem cells, Retina, Macular degeneration, Rod cell, Rhodopsin, Draft:Adeno associated virus and gene therapy of the human retina

Rhodopsin is a biological pigment found in the rods of the retina and is extremely sensitive to light, thus enabling vision in low-light conditions.

Key Points: 
  • Rhodopsin is a biological pigment found in the rods of the retina and is extremely sensitive to light, thus enabling vision in low-light conditions.
  • The Vision Restoration Program is distinct from OpRegen , the Companys clinical-stage cell therapy program which features the sub-retinal delivery of retinal pigment epithelium cells for the treatment of dry age-related macular degeneration (dry-AMD).
  • Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs.
  • With this platform Lineage develops and manufactures specialized, terminally-differentiated human cells from its pluripotent and progenitor cell starting materials.