An assessment of gene therapy ADVM-022 for the treatment of diabetic macular edema (DME) identified dose-dependent safety factors that may influence direction of investigation into the promising therapy in patients with retina diseases.
Findings from the phase 2 INFINITY trial, presented at the American Academy of Ophthalmology (AAO) 2021 Meeting this week by David S. Boyer, MD, Adjunct Clinical Professor of Ophthalmology at USC Keck School of Medicine, contribute to the growing portfolio of ADVM-022 in diabetic ophthalmic disease specifically.
It has already been said that ADVM-022 is a novel biofactory approach to gene therapy, Boyer said. There is a strong, ubiquitous promoter that is designed for robust protein expression, liberating aflibercept from ourselves.
The prior OPTIC study has shown that patients with nAMD previously required frequent injection therapy to maintain vision. Whats more, investigators observed a 97% reduction in mean annualized number of anti-VEGF injections among the 15 patients with nAMD administered 6E11 vg/eye ADVM-022while mean best corrected visual acuity (BCVA) and central subfield thickness (CST) levels were maintained.
OPTIC also showed no patients treated with ADVM-022 reported treatment-related non-ocular adverse events. All treatment-related ocular adverse events were either mild (83%) or moderate (17%) in severity. Ocular inflammation at the 2x1011 vg/eye dose regimen was minimal, and generally resolved with steroid eye drops.
The robust findings of this trial led to the phase 2 INFINITY, in which Boyer and colleagues sought the durability, safety and efficacy of intravitreal ADVM-022 injection in 34 patients with DME. Investigators compared high dose (6x1011 vg/eye), low dose (2x1011), and control for time to worsening of DME disease in the study eye at 24 weeks.
Patients were screened and randomized, then received a loading dose injection of either sham or aflibercept 2 mg. Investigational gene therapy injections were given at day 8.
Over 24 weeks, just 3 (25%) and 5 (39%) patients on high-dose and low-dose ADVM-022 required a supplemental aflibercept injection to address worsening DME at week 24, versus 8 (89%) patients administered aflibercept.
Whats more, nearly half of patients (46%) in each ADVM-022 treatment arm reported a 2 step improvement in diabetic retinopathy severity scale (DRSS) scores at week 12. Another 18% and 36%, respectively, achieved a 3 step improvement by week 24.
That said, more gene therapy-treated patients in the phase 2 trial reported intraocular inflammation: 83% and 92% of the high- and low-dose group, respectively, reported any inflammation, versus just 33% of aflibercept patients. ADVM-022-related adverse events were 57% mild, 41% moderate, and 2% severe.
Across OPTIC and INFINITY, investigators observed dose- and disease state-dependent factors on ADVM-022 efficacy and safety in patients with either nAMD or DME. Boyer stated future development plans for the gene therapy will focus on the treatment of nAMD, and a lower doses.
The difference between the safety seen in OPTIC and INFITIY is being studied, but is unknown, Boyer concluded.
The study, Results From a Phase 2 Study of ADVM-022 Intravitreal Gene Therapy for Diabetic Macula Edema: The INFINITY Trial, was presented at AAO 2021.
Here is the original post:
Gene Therapy ADVM-022 Provides Mixed Efficacy, Safety in Treating DME - MD Magazine
- Overview: Gene Structure - Holland-Frei Cancer Medicine ... - December 23rd, 2021
- Bluebird, Editas: Gene Editing Stocks Had A Tough Year. Will 2022 Be Better? - Forbes - December 23rd, 2021
- Triangle gene editing firms CEO: There is a revolution occurring in medicine - WRAL TechWire - December 23rd, 2021
- Integra Therapeutics Q&A: building next-generation gene editing tools - pharmaceutical-technology.com - December 23rd, 2021
- Global Regenerative Medicine Market is Expected to Reach USD 57.08 Billion by 2027, Growing at a CAGR of 11.27% Over the Forecast Period. -... - December 23rd, 2021
- Gene Mutation Found that is Linked to the Development of Kidney Disease - Clinical OMICs News - December 23rd, 2021
- Vaccines are just the beginning for RNA. The technology is being tested on heart and liver diseases. - The Philadelphia Inquirer - December 23rd, 2021
- A look at prospects for the US gene therapy industry - BioPharma-Reporter.com - December 23rd, 2021
- Novartis to acquire Gyroscope Therapeutics, adding a one-time gene therapy that could transform care for geographic atrophy, a leading cause of... - December 23rd, 2021
- Betagenon co-founder publishes that AMPK activator O304 prevents gene expression changes and remobilisation of histone marks in islets of diet-induced... - December 23rd, 2021
- CUIMC Year in Review: Health News - Columbia University Irving Medical Center - December 23rd, 2021
- EP. 6B: Phenotypic Theranostics in the Future of Precision Medicine - Targeted Oncology - December 23rd, 2021
- Emendo Biotherapeutics and Seattle Children's Research Institute Announce Collaboration to Develop CRISPR-based Therapeutic Strategy for Severe... - December 23rd, 2021
- Open letter to employees, technologists, professionals and physicians of the Optilab laboratory medicine clusters in Quebec - McGill University Health... - December 23rd, 2021
- First variant of concern evolved to evade immune system - EurekAlert - December 23rd, 2021
- Double vaccinated and still infected with Omicron? Heres how long you should isolate - The Indian Express - December 23rd, 2021
- Genetic Tests Prompt Therapy Adjustments in Epilepsy - Medscape - December 22nd, 2021
- How to Know What Strain of COVID-19 You Have - Do Doctors Know What Variant You Have? - Prevention.com - December 22nd, 2021
- Low expression of PLAT in Breast cancer | IJGM - Dove Medical Press - December 22nd, 2021
- Outlook on the Advanced Therapy Medicinal Products CDMO Global Market to 2028 - Rising Number of Clinical Trials for ATMP is Driving Growth -... - December 22nd, 2021
- MiNA Therapeutics Appoints Two Independent Directors With Extensive Industry Experience to Support Next Phase of Growth - BioSpace - December 22nd, 2021
- New gene therapy could provide cure for sickle cell disease, according to UAB study - The Mix - December 19th, 2021
- Durham gene therapy plant to help Pfizer develop treatments for rare diseases - WRAL Tech Wire - December 19th, 2021
- Researchers zero in on therapeutic target for aggressive uterine cancer - Michigan Medicine - December 19th, 2021
- Generation Bio shares halved as hemophilia gene therapy hunt goes back to square one - FierceBiotech - December 19th, 2021
- URMC & RIT faculty awarded patent for gene transfer technology that could transform cancer therapies - URMC - December 19th, 2021
- Very important pharmacogene variants in the Blang population | PGPM - Dove Medical Press - December 19th, 2021
- Gene Sequencing Market Research, Analysis and Global Study |Roche, Johnson & Johnson, Illumina, Thermo Fisher Scientific - Digital Journal - December 19th, 2021
- Global Gene Editing Market Research Report 2021 Featuring CRISPR, GenScript, Horizon Discovery Group, Integrated DNA Technologies and New England... - December 19th, 2021
- Association between the anti-aging protein klotho with sleep | IJGM - Dove Medical Press - December 19th, 2021
- Covid-19: 7 Nigeria returnees admitted to Chennais Kings Institute of Preventive Medicine for suspected - Free Press Journal - December 19th, 2021
- Tessera Therapeutics Adds New Executives to its Leadership Team as the Company Continues to Pioneer Gene Writing Technology as New Category in Genetic... - December 13th, 2021
- Rare gene mutation in some Black Americans may allow earlier screening of heart failure - National Institutes of Health - December 13th, 2021
- Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Bleeding Control in Highest Dose Cohort Through Two Years Following Hemophilia... - December 13th, 2021
- On the hunt for new ALS drugs, researchers see progress, and a long road ahead - BioPharma Dive - December 13th, 2021
- Europe Cell and Gene Therapy Market Size to Reach Revenues of USD 2.9 Billion by 2026 - Arizton - PRNewswire - December 13th, 2021
- Researchers Dig Up Genes and Cells Related to Skull Formation in Mice - Genetic Engineering & Biotechnology News - December 13th, 2021
- Syrian refugee is thriving five years after last-gasp gene therapy - STAT - STAT - December 13th, 2021
- The perils of flawed research and the ivermectin debacle - Medical News Today - December 13th, 2021
- Roche launches the AVENIO Edge System to simplify and automate next-generation sequencing sample preparation, reduce human error and advance precision... - December 13th, 2021
- Why Does The COVID-19 Virus 'Escape' From Our Immune Systems? - Texas A&M University Today - December 13th, 2021
- Hydra DNA Reveals Theres More Than One Way to Regrow a Head - The New York Times - December 13th, 2021
- Graphite Bio Presents Overview of Phase 1/2 CEDAR Trial Evaluating Investigational Gene Editing Therapy GPH101 in Sickle Cell Disease at 63rd ASH... - December 13th, 2021
- Use of Race in Clinical Diagnosis and Decision Making: Overview and Implications - Kaiser Family Foundation - December 13th, 2021
- Identification of Significantly Expressed Gene Mutations for Automated Classification of Benign and Malignant Prostate Cancer - DocWire News - December 13th, 2021
- VUMC: Gene Discoveries Give New Hope To People Who Stutter - Cannon Courier - December 13th, 2021
- Relationship among Homocysteine, Inflammation in AIS | NDT - Dove Medical Press - December 13th, 2021
- Groundbreaking Experimental Compound Displays Effectiveness in Treating Symptoms of Autism and Alzheimers Disease - SciTechDaily - December 13th, 2021
- '90 Day Fianc: Before the 90 Days': Everything to Know About Alina's Rare Medical Condition Diastrophic Dysplasia - Showbiz Cheat Sheet - December 13th, 2021
- Cincinnati Children's teams up with CTI on cell and gene therapies - The Lane Report - December 7th, 2021
- Is 'personalised medicine' the future of healthcare? - Tatler - December 7th, 2021
- Francis Collins on medical advances, vaccine hesitancy and Americans' ill health : Shots - Health News - NPR - December 7th, 2021
- Gene Expression Analysis Market: Rise in the need for personalized medicine is likely to influence the global market - BioSpace - December 7th, 2021
- Atamyo Therapeutics Obtains First Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-100, its Gene Therapy to Treat Limb-Girdle... - December 7th, 2021
- Gyroscope Therapeutics Announces Appointment of Tony Adamis to Board of Directors - Business Wire - December 7th, 2021
- Tune Therapeutics Launches with Pioneering Epigenomic Control Platform to Master Gene Networks, Treat Broad Range of Diseases - PRNewswire - December 7th, 2021
- Rhythm Pharmaceuticals and RareStone Ltd. Announce Exclusive Licensing Agreement for the Development and Commercialization of IMCIVREE (setmelanotide)... - December 7th, 2021
- The top 10 health and medicine breakthroughs of 2021 - Popular Science - December 7th, 2021
- SNPs rs251124, rs2287926, and rs173686 with IA | NDT - Dove Medical Press - December 7th, 2021
- Potential New Therapeutic Option May Combat Drug Resistance in HER2+ Breast Cancer and Ovarian Cancer - Cancer Network - December 7th, 2021
- Penn's Medical Emergency Response Team, 'prepared for anything' | Penn Today - Penn Today - December 7th, 2021
- Gene Therapy May Reverse Hurler Syndrome, a Rare and Severe Illness in Kids - HealthDay - November 21st, 2021
- Immunosuppressants linked to severe reactions in people with common genetic profile - Stanford Medical Center Report - November 21st, 2021
- A video that originated on InfoWars is filled with falsehoods about COVID-19 vaccines - PolitiFact - November 21st, 2021
- Gene signature may give an upper hand in metastatic breast cancer - Baylor College of Medicine News - November 21st, 2021
- Lung cancer management in the era of precision medicine - Express Healthcare - November 21st, 2021
- Powerful gene-based testing by integrating long-range chromatin interactions and knockoff genotypes - pnas.org - November 21st, 2021
- Stoke Therapeutics Announces Presentations from the Company's Dravet Syndrome Program at the American Epilepsy Society 2021 Annual Meeting - Business... - November 21st, 2021
- European Commission approves Roche's Gavreto (pralsetinib) for the treatment of adults with RET fusion-positive advanced non-small cell lung cancer |... - November 21st, 2021
- Maternal cannabis use is associated with suppression of immune gene networks in placenta and increased anxiety phenotypes in offspring - pnas.org - November 21st, 2021
- Vertex Announces Reimbursement Agreement in Spain for KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat People With... - November 21st, 2021
- Efficacy of PARP Inhibitors as Maintenance Therapy for Metastatic Castration-Resistant Prostate Cancer: A Meta-Analysis of Randomized Controlled... - November 21st, 2021
- Gene Therapy and Genetic Engineering - MU School of Medicine - November 17th, 2021
- Gene editing: Great for medicine but ethical issues arise - November 17th, 2021
- Gene on, gene off: Chroma Medicine turns lights on with $125M to control gene expression with ex-Editas, Regeneron leaders - FierceBiotech - November 17th, 2021
- gene therapy | Description, Uses, Examples, & Safety ... - November 17th, 2021
- Sarepta Therapeutics Names Louise Rodino-Klapac, Ph.D., Head of Research and Development - Yahoo Finance - November 17th, 2021
- CRISPR Therapeutics and ViaCyte, Inc. to Start Clinical Trial of the First Gene-Edited Cell Replacement Therapy for Treatment of Type 1 Diabetes -... - November 17th, 2021
- 8 medical advances you may have missed during COVID-19 - AAMC - November 17th, 2021
- PNAS Papers on Woodrat Microbiome, Maternal Cannabis Use, Gene-Based Testing - GenomeWeb - November 17th, 2021