New data shows FLT201 could yield sustained levels of -glucocerebrosidase (GCase) variant 85 to help treat patients with Gaucher disease.
Freeline Therapeutics Holdings announced the data with 1 oral and 3 e-poster presentations the 17THAnnual WORLDSymposium showing the benefit of FLT201 allowing them to move forward with the first ever in-human dose finding studies of the gene therapy.
The treatment leverages Freelines proprietary high-potency adeno-associated virus capsid (AAVS3).
In addition, GCasevar 85, which is being compared to a wild-type GCase, has shown a greater than 20-fold increase in half-life in lysosomal pH and a 6-10 fold increase in half-life in serum, which produces a 20-fold increase in potency of the vector.
The gene therapy is a combination of AAVS3 capsid and a liver-specific promoter that drives the expression of GCasevar85.
These data demonstrate preclinical proof-of-concept for the potential of the program to provide functional cures in patients with the most common form of Gaucher disease, Type 1. Included in these data are demonstration of GCase expression, cellular uptake, tissue penetration, enzymatic activity, and clearance of disease causing substrate, glucosylsphingosine (lyso-Gb1), Romuald Corbau, PhD, Chief Scientific Officer of Freeline, said in a statement. Considered in totality, these data suggest FLT201 may be able to deliver sustained GCase expression in difficult-to-reach tissues, such as bone marrow and lung, as evidenced by the substrate clearance.
In the first study, involving a wild-type mouse, FLT201 resulted in robust GCase expression in the liver and sustained GCase secretion into the plasma, with enhanced and sustained GCase uptake observed in key tissues involved in Gaucher disease including spleen, bone marrow and lung.
In addition, the researchers used Gba-deficient mice and found the treatment resulted in the restoration of GCase activity in difficult-to-reach tissues, observed by decreased levels of disease causing substrate, lyso-Gb1, increased concentrations of plasma GCase and reduced activated macrophages and inflammation in the lung.
The investigators also found from in vitro studies the treatment allowed GCasevar 85 to be taken up by human peripheral blood mononuclear cells and macrophages at levels comparable to those seen with ERT.
Finally, the gene therapy treatment in rhesus macaques was well-tolerated with rapid and robust increases in plasma GCase levels.
In the second poster, the researchers found variant 85 showed the highest level of GCase activity when transduced using AAVS3 in Huh7 cells, with more than an 80-fold increase in activity compared to wild-type GCase. GCasevar85 also showed increased stability in different physiological media compared with enzyme replacement therapy, without differing in its fundamental enzymatic parameter KM.
In the third poster, the researchers showed a custom in vitro phage display library against the enzyme -galactosidase A (GLA) to screen and develop a unique GLA neutralizing IgG4 antibody.
Freeline is expecting to initiate the first in-human dose finding studies of FLT201 by late 2021.
FLT201 data suggest that our gene therapy candidate for Gaucher disease is capable of delivering -glucocerebrosidase variant85 to tissues not sufficiently addressed by standard-of-care enzyme replacement therapy, said Theresa Heggie, Chief Executive Officer of Freeline, in a statement. In addition, we believe steady delivery of enzyme into target tissues to enable sustained clearance of pathologic substrate has the potential to offer significant improvements in clinical outcomes over existing standard of care.
- At FDA meeting, gene therapy experts wrestle with field's blindspots - BioPharma Dive - September 5th, 2021
- Astellas again hits pause on gene therapy trial - BioPharma Dive - September 5th, 2021
- Joe Rogan falsely says mRNA vaccines are 'gene therapy' - PolitiFact - September 5th, 2021
- Connecticuts Children first to enroll affordable gene therapy - WTNH.com - September 5th, 2021
- Cell And Gene Therapy Manufacturing Market Size Worth $57.4 Billion By 2028: Grand View Research, Inc. - PRNewswire - September 5th, 2021
- Global Gene Therapy Market Top Companies, Size, Growth Analysis, Segmentation, Industry Outlook Analysis , and Forecast 2020 to 2027 UNLV The Rebel... - September 5th, 2021
- Regenerative medicine 2021: report highlights record year for the sector - Clinical Trials Arena - September 5th, 2021
- Science Will Win Podcast: Season 1 - Pfizer - September 5th, 2021
- GenSight Biologics Announces Publication of RESTORE Study Data Demonstrating Sustained Efficacy 3 Years After Unilateral Injection of LUMEVOQ -... - September 5th, 2021
- Novartis, Gates Foundation pursue a simpler gene therapy for sickle cell - STAT - February 17th, 2021
- Affinia Therapeutics Announces Addition of Gene Therapy Scientific and Medical Experts to Leadership Team to Advance Novel Gene Therapy Platform and... - February 17th, 2021
- Avance Biosciences Expanding Houston Campus in Support of Cell and Gene Therapy Drug Development - BioSpace - February 17th, 2021
- Sensorion and Institut Pasteur announce new gene therapy collaboration - BioPharma-Reporter.com - February 17th, 2021
- Gene Therapy GS010 Safe, Well-Tolerated for LHON Patients - MD Magazine - February 17th, 2021
- bluebird bio 'baffled' after NICE rejects beta-thalassaemia gene therapy - - pharmaphorum - February 17th, 2021
- Global Gene Therapy Market Outlook to 2030 - by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of... - February 17th, 2021
- GenSight Biologics' gene therapy proves safe in LHON trial - Clinical Trials Arena - February 17th, 2021
- Sio Gene Therapies to Present at the 10th Annual SVB Leerink Global Healthcare Conference - GlobeNewswire - February 17th, 2021
- Beti-Cel Gene Therapy Frees Patients With Beta-Thalassemia From Red Blood Cell Transfusions - OncLive - February 17th, 2021
- Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe... - February 17th, 2021
- CDMO Vigene plots cell and gene therapy manufacturing expansion, adding 245 new jobs along the way - FiercePharma - February 17th, 2021
- Taysha Gene Therapies Announces Formation of Independent Scientific Advisory Board - Business Wire - February 17th, 2021
- bluebird bio's beta-thalassaemia gene therapy rejected by NICE - PharmaTimes - February 17th, 2021
- Europe Cell and Gene Therapy Market Industry Outlook and Forecast Report 2021-2026 with Data-driven Insights on the Impact of COVID-19 -... - February 17th, 2021
- Europe Cell and Gene Therapy Market Report 2021-2026: Prominent Players are Novartis, Spark Therapeutics, Amgen, Gilead Sciences & Organogenesis -... - February 17th, 2021
- Rentschler Biopharma to build new cell and gene therapy capabilities in the UK - BioSpace - February 17th, 2021
- Gene Therapy Market to Reflect Impressive Growth Rate During 2020-2027 AveXis, Vineti, uniQure NV, Spark Therapeutics KSU | The Sentinel Newspaper -... - February 17th, 2021
- Genethon and WhiteLab Genomics Join Forces to Enhance Gene Therapy Through Artificial Intelligence - BioSpace - February 17th, 2021
- Avrobio Gene Therapy Shows Early Promise in Fabry, Other Rare Lysosomal Diseases - BioSpace - February 11th, 2021
- Global Genes Announces New Multimedia Series Focused on Advances in Gene Therapy and Editing, in Collaboration with the National Institutes of Health... - February 11th, 2021
- The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 20212026 - GlobeNewswire - February 11th, 2021
- NICE Draft Guidance Recommends that Patients be Denied Access to bluebird bio's Gene Therapy for Life-Limiting Rare Blood Diseas - PharmiWeb.com - February 11th, 2021
- Dyno Therapeutics Announces Publication in Nature Biotechnology Demonstrating Use of Artificial Intelligence to Generate Unprecedented Diversity of... - February 11th, 2021
- Breakthrough gene therapy helps Utah boy thrive - Yahoo News - February 11th, 2021
- Taysha Gene Therapies Announces Participation in Upcoming Investor Healthcare Conferences - Yahoo Finance - February 11th, 2021
- Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 - Insight, Epidemiology and Forecasts - ResearchAndMarkets.com - Yahoo... - February 11th, 2021
- Amicus Therapeutics Presents Positive Preclinical Fabry Disease Gene Therapy Data at the 17th Annual WORLDSymposium 2021 - GlobeNewswire - February 11th, 2021
- Paragon Biosciences Expands Cell And Gene Therapy Platform - Contract Pharma - February 11th, 2021
- FDA Clears IND Application for Passage Bio's Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare... - February 11th, 2021
- Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key... - February 11th, 2021
- FDA approves third gene therapy for large B-cell lymphoma - European Pharmaceutical Review - February 11th, 2021
- Europe Cell and Gene Therapy Market Size to Reach Revenues of USD 2.9 Billion by 2026 - Arizton - PRNewswire - February 7th, 2021
- FDA Issues More Guidance on Gene and Cell Therapy Products - JD Supra - February 7th, 2021
- Spark Therapeutics Announces First Participant Dosed in Phase 1/2 Study of Investigational Gene Therapy for Late-Onset Pompe Disease - BioSpace - February 7th, 2021
- The gene therapy market is projected to be worth USD 14.6 billion in 2030, growing at a CAGR of 30%, over the next decade, claims Roots Analysis -... - February 7th, 2021
- AGTC Executives Awarded First Place in the BioProcess International Reader's Choice Awards, Cell & Gene Therapies Category - GlobeNewswire - February 7th, 2021
- Retinal Gene Therapy Market: Advent of High-end Technologies to Support Development of the Market - BioSpace - February 7th, 2021
- Sio Gene Therapies Announces Receipt of $11.6 Million from Closing of the Sale of Arvelle Therapeutics - BioSpace - February 7th, 2021
- Welsh, Carson, Anderson & Stowe Commits $250 Million in a Strategic Partnership with Kiniciti, a Newly-Formed Platform Investing in Cell and Gene... - February 7th, 2021
- Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)... - February 7th, 2021
- Abeona Therapeutics Announces Clinical Investigator Webinar to Review ABO-102 and ABO-101 Clinical Data Presented at the 17th Annual WORLDSymposium -... - February 7th, 2021
- Decibel and Catalent Sign Development and Manufacturing Agreement for Dual-Vector Gene Therapy for the Treatment of Congenital Hearing Loss - Yahoo... - February 7th, 2021
- Meeting the commercialization challenge of a surging gene and cell therapy market - FierceBiotech - February 7th, 2021
- Albumedix enters into collaboration agreement with Cell and Gene Therapy Catapult - PharmiWeb.com - February 7th, 2021
- Adverum Biotechnologies Announces Publication of Preclinical Long-Term Safety Data on ADVM-022 IVT Gene Therapy - BioSpace - February 7th, 2021
- Avacta JV raises $7.3m for cell and gene therapy push | Business Weekly - Business Weekly - February 7th, 2021
- Taysha Gene Therapies Highlights Strategic Priorities and Provides 2021 Business Outlook - Business Wire - February 7th, 2021
- FDA's New Guidance on Cell and Gene Therapy Recommends Assessment of COVID-19 Transmission Risks - Lexology - February 7th, 2021
- After reeling back a gene therapy from Sanofi, a North Carolina upstart bags enough money to do something about it - Endpoints News - December 17th, 2020
- Gene Therapy in One Eye Improves Vision in Both Eyes - The Scientist - December 17th, 2020
- After leaving Wall Street to launch a gene therapy upstart, Rachel McMinn nabs $115M to drive her first candidate to the clinic - Endpoints News - December 17th, 2020
- Global Gene Therapy Market Report 2020-2030 Featuring Novartis, Bluebird Bio, Spark Therapeutics, Audentes Therapeutics, Voyager Therapeutics,... - December 17th, 2020
- Cell and Gene Therapy Global Market Report 2020-30: COVID-19 Growth and Change - GlobeNewswire - December 17th, 2020
- Passage Bio Invests In Gene Therapy Manufacturing R&D Site - Contract Pharma - December 17th, 2020
- Gene Therapy Market Worth USD 35.67 Billion at 33.6% CAGR; Rising Prevalence of Spinal Muscular Atrophy to Augment Growth: Fortune Business Insights -... - December 17th, 2020
- Though Promising, Gene Therapies Face Durability And Reimbursement Headwinds - Forbes - December 17th, 2020
- What is gene therapy? - The Star Online - December 17th, 2020
- They thought their gene therapy failed. Instead, it spawned a medical mystery - Endpoints News - December 17th, 2020
- Health Canada approves Zolgensma, the one-time gene therapy for pediatric patients with spinal muscular atrophy (SMA) - Canada NewsWire - December 17th, 2020
- Gene Therapy Injection in One Eye Improves Vision in Both - Technology Networks - December 17th, 2020
- Locanabio Raises $100 Million to Advance RNA-Targeted Gene Therapies - BioSpace - December 17th, 2020
- Worldwide Gene Therapy Industry to 2025 - Cancer is Expected to Hold Significant Market Share in the Indication Segment - ResearchAndMarkets.com -... - December 17th, 2020
- Taysha Gene Therapies Set to Join Russell 2000 Index on December 21, 2020 - Business Wire - December 17th, 2020
- Rare Disease Gene Therapy Market: Increasing cases of genetic diseases to drive the market - BioSpace - December 17th, 2020
- Single gene therapy injection surprisingly boosts vision in both eyes - New Atlas - December 17th, 2020
- Freeline takes the haemophilia B gene therapy fight to Uniqure - Vantage - December 17th, 2020
- Gene Therapy for Hemophilia B Found Safe and Effective in First Phase III Trial - PRNewswire - December 14th, 2020
- ASH: UniQure/CSL hem B gene therapy curbs bleeding in phase 3even in patients with anti-AAV antibodies - FierceBiotech - December 14th, 2020
- Gene Therapy Market Analysis by Vector Type, Application, Region - Global Market Insights, Covid-19 Impact, Competition and Forecast to 2025 -... - December 14th, 2020
- Retinal Gene Therapy Market to Witness Sales Slump in Near Term Due to COVID-19; Long-term Outlook R - PharmiWeb.com - December 14th, 2020