Self-complementary adeno-associated virus

4BIO Capital’s review of AAV gene therapy clinical trials published in Nature Reviews Drug Discovery

Retrieved on: 
Monday, January 25, 2021
Branches of biology, Biology, Genetics, Applied genetics, Gene delivery, Genetic engineering, Parvovirinae, Adeno-associated virus, AAV, Gene therapy, Oncolytic AAV, Self-complementary adeno-associated virus

LONDON & BOSTON 4BIO Capital (4BIO or the Group), an international venture capital firm focused solely on the advanced therapies sector, announces the publication of a systematic review paper entitled The Clinical Landscape for AAV Gene Therapies in Nature Reviews Drug Discovery.

Key Points: 
  • LONDON & BOSTON 4BIO Capital (4BIO or the Group), an international venture capital firm focused solely on the advanced therapies sector, announces the publication of a systematic review paper entitled The Clinical Landscape for AAV Gene Therapies in Nature Reviews Drug Discovery.
  • The review, led by Dr. Dima Kuzmin in collaboration with academics from the University of Oxford and Childrens Hospital of Philadelphia as well as several members of 4BIOs management and advisory teams, analyses 149 unique adeno-associated virus (AAV) gene therapy clinical trials, determining current key trends and depicting the impact of clinical trials on the gene therapy field.
  • This is the first systematic review and meta-analysis that establishes transition probabilities, cumulative safety, and efficacy data for AAV gene therapies.
  • The authors show that AAV gene therapy is generally safe and well tolerated, with no clinical trials failing to reach their primary safety endpoints prior to the cut-off date of the review.

Applied DNA Positions LinearDNA™ Platform as the Next Generation Manufacturing Platform for Adeno-Associated Virus-based Gene Therapies Through New Patent Filing

Retrieved on: 
Monday, August 17, 2020
Infectious diseases, Hospitals, Genetics, Clinical trials, Stem cells, Biotechnology, Health, Pharmaceutical, Oncology, Branches of biology, Biology, Life sciences, Genetic engineering, Biotechnology, Applied genetics, Molecular biology, Gene therapy, Gene delivery, Plasmid, Adeno-associated virus, Self-complementary adeno-associated virus

The Patent claims priority to a previously filed provisional patent application filed with the USPTO in August of 2019.

Key Points: 
  • The Patent claims priority to a previously filed provisional patent application filed with the USPTO in August of 2019.
  • AAV is generally accepted as the preferred vector for gene therapy, and so, has an enormous breadth as a therapeutic gene delivery system.
  • At Applied DNA, we believe we have solved the linear DNA production challenge and can make the DNA sequences at scale for gene therapies.
  • Utilizing our LinearDNA platform to manufacture amplicons, we can deliver gene therapy constructs that potentially lower the risks that come with the use of plasmid-based AAV manufacture.