Gene therapy of the human retina

Clearside Biomedical Positive OASIS and Extension Study Data Presented at The Retina Society 56th Annual Scientific Meeting

Retrieved on: 
Monday, October 16, 2023
CLS, CST, Eye, Inflammation, MD, Aes, Toxicity, Axitinib, SCS, Disease, Gene therapy of the human retina, NAMD, AMD, Diabetic retinopathy, Clinical trial, Persistent, Pharming, Patient, Retina, Visual acuity, Injection, Observation, Șaeș, TKI, Pharmaceutical industry

ALPHARETTA, Ga., Oct. 16, 2023 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), announced today that key data from the Phase 1/2a OASIS clinical trial were presented at The Retina Society 56th Annual Scientific Meeting. The results of the trial demonstrate the key benefits of Clearside’s patented suprachoroidal delivery platform and its lead drug candidate, CLS-AX (axitinib injectable suspension) being developed for the treatment of neovascular age-related macular degeneration (wet AMD or nAMD).

Key Points: 
  • ALPHARETTA, Ga., Oct. 16, 2023 (GLOBE NEWSWIRE) -- Clearside Biomedical, Inc. (Nasdaq: CLSD), a biopharmaceutical company revolutionizing the delivery of therapies to the back of the eye through the suprachoroidal space (SCS®), announced today that key data from the Phase 1/2a OASIS clinical trial were presented at The Retina Society 56th Annual Scientific Meeting.
  • Based on the data from OASIS, CLS-AX is currently being investigated in a Phase 2b clinical trial entitled ODYSSEY.
  • “Our trial is enrolling as planned and we expect data from ODYSSEY in the third quarter of 2024.
  • In the OASIS and Extension Study, participants with wet AMD who were sub-responders with active disease at screening were followed for up to 6 months after CLS-AX treatment.

Nanoscope Therapeutics Announces Presentations at The Retina Society 56th Annual Scientific Meeting

Retrieved on: 
Monday, October 9, 2023
Paratriathlon, Thomas Jefferson University, University, Stargardt disease, Clinical trial, Gene therapy of the human retina, Affiliation, Therapy, Randomness, Research, Patient, Retina, Optogenetics, Retinitis pigmentosa, Pharmaceutical industry, Medical device, Ophthalmology, Electrophysiology

DALLAS, Oct. 9, 2023 /PRNewswire/ -- Nanoscope Therapeutics Inc. , a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases and macular degenerations, today announced presentations at The Retina Society 56th Annual Scientific Meeting taking place in New York, New York from October 11-14, 2023.

Key Points: 
  • DALLAS, Oct. 9, 2023 /PRNewswire/ -- Nanoscope Therapeutics Inc. , a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases and macular degenerations, today announced presentations at The Retina Society 56th Annual Scientific Meeting taking place in New York, New York from October 11-14, 2023.
  • In her presentation, Dr. Kay will discuss clinical data and analysis from Nanoscope's Phase 2b RESTORE trial in patients with advanced retinitis pigmentosa.
  • Title: MCO-010 Optogenetic Therapy for Profound Vision Loss in Stargardt Disease: 12-Month Outcomes from the Phase 2 STARLIGHT Trial
    Presenter: Allen Ho, M.D., Professor of Ophthalmology at Thomas Jefferson University and Attending Surgeon, Director of Retina Research and Co-Director of the Retina Service at Wills Eye Hospital.
  • In this late-breaking presentation, Dr. Ho will discuss recent 1 year data from Nanoscope's Phase 2 STARLIGHT trial of MCO-010 therapy in patients with advanced Stargardt macular degeneration.

Adverum Biotechnologies Reports Third Quarter 2022 Financial Results

Retrieved on: 
Thursday, November 10, 2022
Quality of life, GLOBE, Private Securities Litigation Reform Act, NAMD, Prevalence, Investment, Gene therapy of the human retina, Annual general meeting, Food, Nasdaq, Research, Society, Incidence, Injection, Technology, IRF, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Neovascularization, Severe cognitive impairment, SEC, NASDAQ, American Academy, Patient, AMD, Macular degeneration, Safety, Compliance, U.S. Securities and Exchange Commission, Caregiver, OPTIC, VEGF, Home After Three Months Away, Retina, Standard of care, European Medicines Agency, Drug discovery, SRF, IVT, Pharmaceutical industry, Birth control, Vaccine, Adverum Biotechnologies

REDWOOD CITY, Calif., Nov. 10, 2022 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today announced financial results for the third quarter ended September 30, 2022.

Key Points: 
  • In July 2022, Adverum completed an Investigational New Drug amendment with the U.S. Food and Drug Administration to advance a Phase 2 trial of Ixo-vec in wet AMD.
  • Stock-based compensation expense included in research and development expenses was $1.4 million for the third quarter of 2022.
  • Stock-based compensation expense included in general and administrative expenses was $3.1 million for the third quarter of 2022.
  • Additional risks and uncertainties facing Adverum are set forth under the caption Risk Factors and elsewhere in Adverums Securities and Exchange Commission (SEC) filings and reports, including Adverums Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 filed with the SEC on August 11, 2022.

Rezolute Reports First Quarter Fiscal 2023 Results and Highlights Company Progress

Retrieved on: 
Wednesday, November 9, 2022
GLOBE, Private Securities Litigation Reform Act, Forward-looking statement, Congenital hyperinsulinism, Risk, Gene therapy of the human retina, RIZE, ESPE, Nasdaq, BGM, Diabetic retinopathy, DME, Hypoglycemia, G&A, Securities Act of 1933, MAD, R, SEC, Founder, Safety, Securities Exchange Act of 1934, Clinical trial, Research, Patient, CGM, Pharmaceutical industry, Rezolution Pictures

REDWOOD CITY, Calif., Nov. 09, 2022 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT), a clinical-stage biopharmaceutical company dedicated to developing transformative therapies with the potential to shift the treatment paradigms of devastating metabolic diseases, today announced its financial results for the first quarter of fiscal 2023 ended September 30, 2022.

Key Points: 
  • Research and development (R&D) expenses were $7.7 million for the first quarter of fiscal 2023, compared to $5.8 million for the same period in fiscal 2022.
  • General and administrative (G&A) expenses were $2.5 million for the first quarter of fiscal 2023, compared to $1.9 million for the same period in fiscal 2022.
  • Net loss was $9.8 million for the first quarter of fiscal 2023, compared to $7.8 million for the same period in fiscal 2022.
  • Rezolute is also developing RZ402, an orally available plasma kallikrein inhibitor, for the treatment of diabetic macular edema.

Adverum Biotechnologies Presents Positive End of Study Results from the OPTIC Trial in Wet AMD Including Two-Year Outcomes Following a Single Intravitreal Injection of Ixo-vec (ADVM-022)

Retrieved on: 
Friday, November 4, 2022
Patient, Severe cognitive impairment, Gene therapy of the human retina, VEGF, AMD, Private Securities Litigation Reform Act, Research, Compliance, Maintenance, CST, IVT, GLOBE, Steroid, Safety, Incidence, OPTIC, Retina, NAMD, Macular degeneration, Nasdaq, Standard of care, Physician, U.S. Securities and Exchange Commission, NASDAQ, Prevalence, Injection, Inflammation, Wills Eye Hospital, Technology, SEC, Neovascularization, Birth control, Pharmaceutical industry, Vaccine, Adverum Biotechnologies

REDWOOD CITY, Calif., Nov. 04, 2022 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today announced long-term follow-up data from the OPTIC study treating wet age-related macular degeneration (wet AMD) during The Retina Society Annual Meeting in Pasadena, California. New data presented include final two-year safety and efficacy assessment of Ixo-vec (ixoberogene soroparvovec, formerly referred to as ADVM-022), including reduction in annualized anti-VEGF injections, clinically relevant aflibercept protein levels and maintenance to improvement of mean best-corrected visual acuity (BCVA) and central subfield thickness (CST). OPTIC study participants could enroll in an extension study for an additional three years for a total of five years of follow-up.

Key Points: 
  • OPTIC study participants could enroll in an extension study for an additional three years for a total of five years of follow-up.
  • We are pleased to present our final two-year analysis from our OPTIC trial of Ixo-vec for the treatment of wet AMD.
  • All 2E11 participants were inflammation free and did not require steroids to treat inflammation at the end of the study.
  • Wet AMD, also known as neovascular AMD or nAMD, is an advanced form of AMD, affecting approximately 10% of patients living with AMD.

Rezolute Announces Presentation at The Retina Society 55th Annual Scientific Meeting

Retrieved on: 
Thursday, October 20, 2022
CA, DME, SEC, MAD, Lists of diseases, Gene therapy of the human retina, Congenital hyperinsulinism, Patient, Twitter, Securities Act of 1933, Nasdaq, Private Securities Litigation Reform Act, Diabetic retinopathy, Securities Exchange Act of 1934, GLOBE, Forward-looking statement, Risk, Pharmaceutical industry, Rezolution Pictures

Rezolute strives to disrupt current treatment paradigms by developing transformative therapies for devastating rare and chronic metabolic diseases.

Key Points: 
  • Rezolute strives to disrupt current treatment paradigms by developing transformative therapies for devastating rare and chronic metabolic diseases.
  • Patient, clinician, and advocate voices are integrated in the Companys drug development process, enabling Rezolute to boldly address a range of severe conditions.
  • Rezolute is steadfast in its mission to create profound, positive, and lasting impact on patients lives.
  • Rezolute is also developing RZ402, an orally available plasma kallikrein inhibitor, for the treatment of diabetic macular edema.

2021 Report on Retinal Degeneration Drugs in Development - ResearchAndMarkets.com

Retrieved on: 
Thursday, July 22, 2021
Pharmaceutical, Health, Optical, Ophthalmology, Vision, Retinal degeneration, Retina, Anatomy, Vision Institute, Gene therapy of the human retina

The "Retinal Degeneration (Ophthalmology) - Drugs in Development, 2021" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Retinal Degeneration (Ophthalmology) - Drugs in Development, 2021" report has been added to ResearchAndMarkets.com's offering.
  • The report provides comprehensive information on the therapeutics under development for Retinal Degeneration, complete with analysis by Stage of Development, Drug Target, Mechanism of Action (MoA), Route of Administration (RoA) and Molecule Type.
  • Additionally, the report provides an overview of key players involved in therapeutic development for Retinal Degeneration and features dormant and discontinued projects.
  • The report reviews key players involved in the development of Retinal Degeneration (Ophthalmology) therapeutics and enlists all their major and minor projects.

OpRegen® Clinical Data Continues to Demonstrate Functional and Anatomical Improvements in Patients With Dry AMD With Geographic Atrophy

Retrieved on: 
Tuesday, July 20, 2021
Biotechnology, Optical, Health, Clinical trials, Ophthalmology, Organ systems, Vision, Geographic atrophy, Macular degeneration, Blindness, Retinal pigment epithelium, Retina, Emerging technologies, Diabetic retinopathy, Retinopathy, Gene therapy of the human retina, OpRegen, Age-Related Macular Degeneration, Lineage Cell Therapeutics, Inc., OPREGEN, AGE-RELATED MACULAR DEGENERATION, LINEAGE CELL THERAPEUTICS, INC.

OpRegen is an investigational cell therapy consisting of allogeneic retinal pigment epithelium (RPE) cells, administered one time to the subretinal space, for the treatment of dry age-related macular degeneration (AMD) with geographic atrophy (GA).

Key Points: 
  • OpRegen is an investigational cell therapy consisting of allogeneic retinal pigment epithelium (RPE) cells, administered one time to the subretinal space, for the treatment of dry age-related macular degeneration (AMD) with geographic atrophy (GA).
  • We look forward to continuing to follow all study patients and reporting our efficacy and safety observations over time.
  • Improvements in best corrected visual acuity (BCVA) for Cohort 4 patients reached up to +19 letters on the Early Treatment Diabetic Retinopathy Study (ETDRS) chart.
  • There are two forms of AMD: dry (atrophic) AMD and wet (neovascular) AMD.

LogicBio Therapeutics to Participate in William Blair Biotech Focus Conference 2021

Retrieved on: 
Thursday, July 8, 2021
Branches of biology, Biology, Applied genetics, Adeno-associated virus, Parvovirinae, Parvoviruses, Emerging technologies, Jean Bennett, Gene therapy of the human retina

LEXINGTON, Mass., July 8, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood, today announced that Frederic Chereau, chief executive officer of LogicBio, will participate in a virtual panel discussion entitled "Delivery and Durability of Genetic Medicines" at the William Blair Biotech Focus Conference 2021 on Thursday, July 15, 2021 at 10:00 a.m.

Key Points: 
  • LEXINGTON, Mass., July 8, 2021 /PRNewswire/ --LogicBio Therapeutics, Inc.(Nasdaq:LOGC), a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood, today announced that Frederic Chereau, chief executive officer of LogicBio, will participate in a virtual panel discussion entitled "Delivery and Durability of Genetic Medicines" at the William Blair Biotech Focus Conference 2021 on Thursday, July 15, 2021 at 10:00 a.m.
  • LogicBio Therapeutics is a clinical-stage genetic medicine company pioneering gene editing and gene delivery platforms to address rare and serious diseases from infancy through adulthood.
  • The Company's second platform, sAAVy, is an adeno-associated virus (AAV) capsid engineering platform designed to optimize gene delivery for treatments in a broad range of indications and tissues.
  • For more information, visit www.logicbio.com , which does not form a part of this release.

Neurogene Announces EMA Grants Orphan Drug Designation to CLN5 Batten Disease Gene Therapy 

Retrieved on: 
Tuesday, June 29, 2021
Biotechnology, FDA, Health, Genetics, Pharmaceutical, Branches of biology, Life sciences, Biology, Applied genetics, Gene delivery, Gene therapy, Emerging technologies, Medical genetics, Adeno-associated virus, Batten disease, Therapy, Gene therapy of the human retina, Orphan Drug Designation, CLN5, a subtype of Batten disease, Genetic Testing, Neurogene Inc., ORPHAN DRUG DESIGNATION, CLN5, A SUBTYPE OF BATTEN DISEASE, GENETIC TESTING, NEUROGENE INC.

Neurogene is collaborating with Batten disease experts, regulatory authorities, and caregivers to provide a safe and effective gene therapy for this disease as quickly as possible.

Key Points: 
  • Neurogene is collaborating with Batten disease experts, regulatory authorities, and caregivers to provide a safe and effective gene therapy for this disease as quickly as possible.
  • Applications for orphan designation are examined by the EMAs Committee for Orphan Medicinal Products (COMP), using the Committees network of experts.
  • Our lead programs use adeno-associated virus (AAV) vector-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene.
  • Neurogene is also developing novel gene therapy technologies to advance treatments for complex neurological diseases that conventional gene therapy cannot successfully address.