Retinal ganglion cell

Neurophth Receives IND Clearance from FDA for AAV-ND1 Gene Therapy of LHON

Retrieved on: 
Monday, December 19, 2022
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Professor Bin Li, Founder, Chairman and CEO of Neurophth, said, "We are thrilled to have received IND clearance for NFS-02, marking our second program to reach clinical development.

Key Points: 
  • Professor Bin Li, Founder, Chairman and CEO of Neurophth, said, "We are thrilled to have received IND clearance for NFS-02, marking our second program to reach clinical development.
  • Based on encouraging preclinical data, we believe that this gene therapy drug candidate holds promise to become a safe and effective treatment for patients."
  • Our most advanced investigational gene therapy drug candidate, NR082 (rAAV2-ND4), in development for the treatment of mtND4-mediated LHON, has been granted orphan drug designation (ODD) by the U.S. FDA and EMA.
  • Recently, our second gene therapy drug candidate NFS-02, has been granted IND clearance from U.S. FDA.

Ray Therapeutics and Forge Biologics Expand Their Viral Vector cGMP Partnership to Encompass Plasmid DNA Manufacturing

Retrieved on: 
Monday, October 10, 2022
Other Manufacturing, Research, Genetics, Clinical Trials, Manufacturing, Biotechnology, Health, Pharmaceutical, Science, Ignition, CA, Retina, Therapy, Retinal ganglion cell, Prevalence, Optogenetics, Retinal degeneration, Field line, Light, CGMP, Person, Biotechnology, Patient, B cell, Research, Program, Multimedia, Â, Severe cognitive impairment, Plasmid, Partnership, Retinitis, Visual acuity, Nyctalopia, Neuron, Ray, AAV, RP, CEO, RGC, DNA, Central retinal artery occlusion, HEK, Retinitis pigmentosa, Pharmaceutical industry, Vaccine, Hearth, Forge

View the full release here: https://www.businesswire.com/news/home/20221010005240/en/

Key Points: 
  • View the full release here: https://www.businesswire.com/news/home/20221010005240/en/
    Forge will provide research-grade and GMP-Pathway plasmid manufacturing services, in addition to adeno-associated viral vector (AAV) process development, scale-up engineering, and cGMP manufacturing services for Ray Therapeutics program, RTx-015.
  • The program will continue to utilize Forges platform manufacturing processes, including its proprietary HEK 293 suspension Ignition Cells and pEMBR adenovirus helper plasmid.
  • All development and cGMP manufacturing activities will occur at the Hearth, Forges 200,000 square foot gene therapy cGMP production facility in Columbus, Ohio.
  • Ray Therapeutics lead candidate RTx-015 uses intravitreal administration, from which the vector diffuses into the retina and transduces primarily the RGCs.

Q BIOMED ANNOUNCES ISSUANCE OF PATENT FOR GROUNDBREAKING GDF15 BIOMARKER

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Tuesday, September 27, 2022
Securities Exchange Act of 1934, Therapy, IOP, Visual Science, Drug Quality and Security Act, Glaucoma, Certificate, USE, Washington University in St. Louis, NEW, European Certification and Qualification Association, Washington University School of Medicine, Securities Act of 1933, Industry, GDF15, Severe cognitive impairment, Forward-looking statement, Survival, Patient, Eye, Retinal ganglion cell, Physician, Regulation, Cell, Neuroprotection, Research, Vascular disease, Intraocular pressure, SEC, Optic nerve, HIV disease progression rates, RGC Resources, Patent, Medical imaging, Pharmaceutical industry, Ophthalmology

NEW YORK , Sept. 27, 2022 /PRNewswire/ -- Q BioMed Inc. (QBIO), a commercial stage biotechnology acceleration development company, is pleased to announce receipt of a European Certificate of Grant for GDF15, a diagnostic marker for determining the severity of glaucoma using the expression levels of Growth Differentiation Factor 15 (GDF15) licensed from the Washington University in St. Louis. Determining the severity of glaucoma using this biomarker will aid in treatment decisions for patients diagnosed with, and being treated for, glaucoma.

Key Points: 
  • Determining the severity of glaucoma using this biomarker will aid in treatment decisions for patients diagnosed with, and being treated for, glaucoma.
  • Q BioMed and its technology partner Mannin Research Inc. are currently developing therapeutics with novel mechanisms of action, to treat Primary Open-Angle Glaucoma.
  • Q BioMed aims to accelerate the monetization of biomedical technologies through rapid innovation and collaborative partnerships with industry leading researchers.
  • Q BioMed believes its assets in oncology, vascular disease, and rare orphan diseases address unmet medical needs and large markets.

InMed Pharmaceuticals Reports Full Year Fiscal 2022 Financial Results and Provides Business Update

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Friday, September 23, 2022
Adult, Department of Biotechnology, Safety, Drug discovery, Data monitoring committee, Research, Compliance, Patient, Clinical trial, Investment, Nasdaq, Appetite, Neuroprotection, Study, Disease, Workforce, Sale, Food, Glaucoma, Company, Acquisition, Wound healing, Person, Molecule, Insurance, THCV, Ageing, Patent, FDA, MD, Review, Adolescence, BBA, Ministry of Health & Wellness, Method, Intraocular pressure, Cannabinol, Neurodegeneration, Epidermolysis bullosa, Department, Cannabinoid, Isaac of Troki, CBN, Calendar, EB, Program, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Retinal ganglion cell, Biotechnology, Control, University, INMED, Pharmaceutical industry, Chemistry

The Companys full financial statements and related MD&A for the fiscal year ended June 30, 2022, will be available at www.inmedpharma.com and at www.sedar.com .

Key Points: 
  • The Companys full financial statements and related MD&A for the fiscal year ended June 30, 2022, will be available at www.inmedpharma.com and at www.sedar.com .
  • InMed is evaluating the safety of INM-755 (cannabinol) cream and its preliminary efficacy in treating symptoms and wound healing over a 28-day treatment period.
  • In the second half of fiscal 2022 and during the subsequent months, InMed and its BayMedica subsidiary implemented significant cost saving measures, including some personnel changes.
  • Also, as part of these expense reduction initiatives, no employee received an annual performance bonus for fiscal year 2022.

Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates

Retrieved on: 
Monday, August 8, 2022
Biotechnology, Pharmaceutical, Health, Clinical Trials, FDA, Dravet syndrome, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Movement, Death, Government budget balance, CSF, Asos, SUDEP, ASO, Retinal ganglion cell, Risk, IVT, Diagnosis, Technology, NHP, Cognition, Targeted molecular therapy for neuroblastoma, FALCON, Sleep, Haploinsufficiency, Research, Caregiver, Cerebrospinal fluid, TANGO, Language, Plasma, Patient, Quality of life, Therapy, Company, Contract, Prognosis, License, Seizure, RGC, Autonomic nervous system, Intellectual disability, RNA, Safety, Lists of diseases, Epilepsy, Collaboration, OLE, ADOA, Protein, Nasdaq, SCN1A, Food, EMA, Pharmacokinetics, Disease, Vaccine, Pharmaceutical industry, Cryptocurrency, Online gambling, Animal, Stoke, OPA1

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today reported financial results for the second quarter of 2022 and provided business updates.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today reported financial results for the second quarter of 2022 and provided business updates.
  • In the second half of 2022, the Company expects to begin enrollment in a prospective natural history study (FALCON) of people living with ADOA.
  • Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines.
  • Stoke is pursuing the development of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.

GenSight Biologics reports 5 years’ data showing sustained efficacy and safety following one-time treatment with LUMEVOQ®

Retrieved on: 
Wednesday, July 20, 2022
Biotechnology, Health, Genetics, Pharmaceutical, Clinical Trials, Sequence, Logarithm, Vision, Incidence, Safety, Review, Mitochondrion, Eye, LHON, Union, Research, EMA, Mitochondrial disease, Systematic review, Visual acuity, Color, AAV, Leber's hereditary optic neuropathy, Natural history, Optogenetics, Severe cognitive impairment, Science Translational Medicine, Quality of life, European Medicines Agency, Pain, Field line, Year, Retina, Ophthalmology, CRR, ND4, Retinal ganglion cell, CHMP, Institute, MTS, Hospital, Medical device, Pharmaceutical industry, Vaccine, Medical imaging, Patient, LogMAR chart, N, Optic neuropathy

Year 5 values were the LogMAR readings nearest to 1,825 days post treatment recorded between 1,825 +/- 28 days post-treatment.

Key Points: 
  • Year 5 values were the LogMAR readings nearest to 1,825 days post treatment recorded between 1,825 +/- 28 days post-treatment.
  • Responder analyses at Year 5 indicate that improved BCVA was a benefit for a substantial proportion of the study participants.
  • Efficacy and safety of intravitreal gene therapy for Leber hereditary optic neuropathy treated within 6 months of disease onset.
  • The primary objective is to assess the long-term safety of intravitreal LUMEVOQ administration up to 5 years post-treatment.

GenSight Biologics Announces Publication of a Study of the Impact of LHON Disease on the Lives of Patients and Relatives in Journal of Neuro-Ophthalmology

Retrieved on: 
Thursday, May 19, 2022
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The study determined that the impact of LHON extends beyond vision-related activity limitations, while addressing its psychosocial impact.

Key Points: 
  • The study determined that the impact of LHON extends beyond vision-related activity limitations, while addressing its psychosocial impact.
  • Additionally, the study determined that despite residing in different countries, LHON patients and their relatives described similar experiences in the four areas of focus in this study.
  • The objective of this study was to comprehensively explore the impact of LHON on the lives of patients and their relatives at the time of diagnosis and now.
  • The Impact of Leber Hereditary Optic Neuropathy on the Quality of Life of Patients and Their Relatives: A Qualitative Study
    1.

InMed Pharmaceuticals Reports Third Quarter Fiscal 2022 Financial Results and Provides Business Update

Retrieved on: 
Friday, May 13, 2022
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BayMedica has also begun commercial scale production of its delta-9 dominant tetrahydrocannabivarin (THCV) in anticipation of commencing B2B sales.

Key Points: 
  • BayMedica has also begun commercial scale production of its delta-9 dominant tetrahydrocannabivarin (THCV) in anticipation of commencing B2B sales.
  • Formulated cannabinoid products in different delivery forms can help to expand product development options for manufacturers of health and wellness products.
  • To support our commercial efforts, in February the Company appointed seasoned business executive, Jerry P. Griffin, as VP of Sales and Marketing.
  • Reported sales were impacted this quarter by a delay in the product launches of two new rare cannabinoids, CBDV and THCV.

Stoke Therapeutics Reports First Quarter Financial Results and Provides Business Updates

Retrieved on: 
Tuesday, May 10, 2022
Research, FDA, Genetics, Clinical Trials, Biotechnology, Pharmaceutical, Health, Optical, Science, Other Science, Epilepsy, NHP, Targeted molecular therapy for neuroblastoma, European Medicines Agency, Safety, Seizure, Caregiver, Collaboration, Quality of life, Dravet syndrome, Language, IVT, Technology, SCN1A, Annual general meeting, Risk, Association for Research in Vision and Ophthalmology, Company, Plasma, Asos, ARVO, Patient, FDA, ASO, Disease, Haploinsufficiency, Research, Therapy, EMA, Pharmacokinetics, RNA, Autonomic nervous system, Medicine, Retinal ganglion cell, Cell, Cerebrospinal fluid, Contract, Cognition, ADOA, Sleep, Gene, Association, FALCON, American Society of Gene and Cell Therapy, The Association, Movement, Protein, Nasdaq, Government budget balance, Death, TANGO, Â, OPA1, Diagnosis, License, OLE, Prognosis, Intellectual disability, Society, SUDEP, Lists of diseases, Vaccine, Pharmaceutical industry, Online gambling, Ophthalmology, Stoke

Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today reported financial results for the first quarter of 2022 and provided business updates.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today reported financial results for the first quarter of 2022 and provided business updates.
  • We believe we are in a strong financial position with sufficient capital to support our current research and clinical development efforts into 2025.
  • The European Medicines Agency (EMA) granted orphan drug designation for STK-001 as a potential new treatment for Dravet syndrome.
  • In the second quarter of 2022, the Company expects to initiate a prospective natural history study (FALCON) of people living with ADOA.

Stoke Therapeutics Presents New In-Vivo Data That Demonstrated Dose-Related Target Engagement and OPA1 Protein Upregulation in Retinal Tissue Following Administration of STK-002

Retrieved on: 
Monday, May 2, 2022
Research, Genetics, Clinical Trials, Other Health, Biotechnology, Pharmaceutical, Health, Optical, Science, Other Science, COVID-19, U.S. Securities and Exchange Commission, Mutation, Private Securities Litigation Reform Act, Twitter, Nasdaq, Asos, Targeted molecular therapy for neuroblastoma, RNA, Company, Retinal ganglion cell, Haploinsufficiency, Stoke, Research, Eye, TANGO, ARVO, ADOA, RGC, Injection, Patient, Security (finance), Association, Incidence, Association for Research in Vision and Ophthalmology, Severe cognitive impairment, Tissue, Significance, Potency (pharmacology), Founder effect, Dravet syndrome, ASO, Disease, NMD, Life, RNA transfection, IVT, Annual general meeting, Central nervous system, Therapy, Vaccine, Pharmaceutical industry, Ophthalmology, OPA1

Stoke Therapeutics, Inc. (Nasdaq: STOK), abiotechnologycompanydedicatedtoaddressingthe underlying causeof severe diseases by upregulating protein expression with RNA-based medicines, todayannounced new preclinical data that demonstrated dose-related target engagement and increases in OPA1 protein levels in retinal tissue of non-human primates (NHPs) following intravitreal (IVT) administration of STK-002.

Key Points: 
  • Stoke Therapeutics, Inc. (Nasdaq: STOK), abiotechnologycompanydedicatedtoaddressingthe underlying causeof severe diseases by upregulating protein expression with RNA-based medicines, todayannounced new preclinical data that demonstrated dose-related target engagement and increases in OPA1 protein levels in retinal tissue of non-human primates (NHPs) following intravitreal (IVT) administration of STK-002.
  • Target engagement and OPA1 protein increases were sustained for at least eight weeks post-injection.
  • A dose-related increase in OPA1 protein was also detected in retinal ganglion cells (RGCs) of NHPs treated with STK-002.
  • An estimated 65% to 90% of cases are caused by mutations in the OPA1 gene, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein expression and disease manifestation.