Advancements in Gene Therapy for Retinal Diseases
Veeral S. Sheth, MD, MBA, Director of Clinical Research at the University of Retina and Macula Associates, discusses advanced therapies for retinal diseases.
Optogenetics Device With Gene Therapy is Safe, Efficacious in Retinitis Pigmentosa
Following GS030 optogenetic therapy, the first treated patient was able to locate and count objects on a table and could identify crosswalks in the street.
Gene Therapy for Diabetic Macular Edema Shows Mixed Efficacy, Safety
Findings from the phase 2 INFINITY trial identified dose-dependent safety outcomes of ADVM-022 in patients with diabetic macular edema.
Pfizer-BioNTech COVID-19 Vaccine Receives FDA Approval
The 2-dose vaccine has been approved for use 9 months after its Emergency Use Authorization (EUA).
Gene Therapy AGTC-501 Hits Phase 1/2 Marks for Rare Pediatric Blindness
A podcast interview with study author Paul Yang, MD, PhD, on the current research and future implementation of the agent.
Sickle Cell Gene Therapy Could Significantly Impact Medicaid Budgeting, Access
A budget impact analysis suggest the burgeoning drug class may not be as available as it could be for sickle cell patients.
AT-GTX-502 Could Provide JNCL Patient Stabilization
Descriptive findings from the phase 1/2 study allude to promise for the gene therapy in the rare pediatric disorder.
LYS-SAF302 Linked to MPS-IIIA Biologic Response in AAVance Trial
New phase 2/3 findings meet investigators' hypotheses on the gene therapy's mixed benefits for heparan sulfate reduction.
Transpher B Findings: ABO-101 Benefits CNS Biomarkers in Patients with MPS-IIB
The AAV-based gene therapy was well-tolerated and showed enough promising effect to warrant further investigation, researchers reported.
Transpher A Findings: ABO-102 Provides Continuous MPS-IIIA Neurocognition Gain
Investigators observed sustained, dose-dependent benefits for young patients at 24 months.
CTX001 Data Suggest Possible Functional Cure for Sickle Cell, β-Thalassemia
The HSPC therapy showed fetal hemoglobin production in transfusion-dependent patients in a matter of months—with sustained results over 1 year.
Fabry Disease Gene Therapy AVR-RD-01 Completely Reduces Toxic Kidney Substrate
New WORLDSymposium findings show a treated patient had 100% reduction in the accumulating fatty substrate associated with the burdensome rare disease after 1 year.
Gene Therapy Reduces Annualized Hemophilia A Bleeding Rate by 84% in Phase 3 Trial
New one-year data makes history as the first to indicate the superiority of a one-injection gene therapy versus Factor VIII prophylaxis.
Elliott Vichinsky, MD: Voxelotor and New HOPE Data for Sickle Cell
The UCSF hematologist discusses the growing portfolio for the sickle cell therapy after ASH 2020.
Largest Hemophilia B Gene Therapy Study to Date Hits Early Benefit, Safety Marks
The historic phase 3 HOPE-B trial shows all but 1 treated patient discontinued prophylaxis after a single dose of etranacogene deaparvovec.
New Gene Therapy Product ARU-1801 Shows Durable, Safe Sickle Cell Effect
The second process of ARU-1801 in a new patient resulted in up to 4 times greater engraftment of transduced hematopoietic stem cells and progenitors at 6 months.
Gene-Editing Therapy CTX001 Reports Durable Effects for TDT, Sickle Cell Patients
The furthest-progressed gene-edited therapy for a genetic disease reported historic efficacy data in ongoing phase 1/2 trials.
Gene Therapy Shows Early Potential in XLRP
New phase 1/2 data shows AAV-RGPR may benefit a previously untreated population of inherited retina disease boys and young men.
Single-Injection Cell Therapy Relieves CMD Symptoms
New findings from ESCaPE-MD show CD34+ cell therapy significantly benefitted patients with coronary microvascular dysfunction over 6 months.
Joshua Hare, MD: The Research Hurdles of Stem Cell Therapy
Despite an embrace of greater patient populations by the FDA, cardiovascular research into stem cell therapy has been slow and burdened.
Joshua Hare, MD: Stem Cell Therapy in Heart Failure
As safety and efficacy programs advance, clinicians consider the investigative therapy's potential in cardiology.
Gene Therapy Valoctocogene Roxaparvovec Nears Hemophilia FDA Submission
BioMarin announced the investigative gene therapy reached pre-specified criteria for Factor VIII levels in adult patients with severe hemophilia A.
Dr. Geoff Bellingan: The Future for Multi-Stem Cell Therapy
Previous analyses into MultiStem Cell Therapy have shown benefits and safety in stroke patients. What will it take for the therapy to reach a phase 3 trial for ARDS?
Dr. Geoff Bellingan: MultiStem Cell Therapy in ARDS
Early-stage study results from the multi-center MUST-ARDS trial show the ex-vivo adult progenitor cell-expanding therapy is capable of improving 28-day mortality, care burden, and overall safety in patients with ARDS.
FDA Clears Gene Therapy for Huntington Disease Assessment
An Investigational New Drug application was approved to clinically assess AMT-130 as the first potential drug marketed for the neurodegenerative disease.
Retina Stem Cell Therapy Restores Vision in Animal Models
In an effort to reprogram retina cell regeneration, investigators activated dormant stem cells then aided other stem cells in developing into rod photoreceptor cells—the most abundant cell type in the retina which first aid the retina in sensing light.
Investigative Gene Therapy RGX-314 Reduces Anti-VEGF Dosing in AMD Patients
The REGENXBIO drug's promising phase 1 results has led to the addition of a fourth patient dosing cohort and a phase 2 trial initiation.
Stephen R. Russell, MD: The Significance of New Voretigene Neparvovec-ryzl Data for IRD
How the three-year results of a gene therapy for inherited retinal disease may redefine its potential in ophthalmology.
Voretigene Neparvovec-rzyl Gene Therapy Continues to Improve IRD Patient Marks in Year 3
A three-year update of VN for patients with biallelic RPE65 mutation-associated inherited retinal disease improved on the common standard-of-care for retina disease.
Characterizing Dangerous, Unregulated Stem Cell Therapy for Ocular Disease
Though cell therapies have gained FDA approval to treat any ocular disease, companies have marketed predominately to patients with AMD, with procedures that could lead to blindness.
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