Genomics
Optogenetic gene therapy provides hope for retinitis pigmentosa patients
Optogenetic therapy bypasses traditional methods of repairing defective photoreceptors in the eye by targeting other retinal cells. Recently, three examples of optogenetic therapeutics under development highlight many of the challenges this type of gene therapy faces for the treatment of a devastating genetic eye disease. Read More
EMBL partners with DeepMind to predict 3D structure of human proteins
Google Health's DeepMind announced a partnership with the European Molecular Biology Laboratory (EMBL) to offer the scientific community open access to the most comprehensive database developed to date of predicted 3D protein structures. Read More
Cellular programming spurs next-generation cell therapies
Cellular reprogramming of ex vivo cells may offer improvements on first-generation adoptive cell therapies, which have experienced limited clinical success due to complex manufacturing, potential patient rejection, and availability of clinical cell types. Further, in vivo reprogramming may expand on this concept by eliminating the need for manufacture and the risk of immune rejection. Read More
Thermo Fisher opens cGMP plasmid DNA manufacturing facility in Calif.
Thermo Fisher Scientific announced it has officially opened its new current good manufacturing practice (cGMP) plasmid DNA manufacturing facility in Carlsbad, CA, a step the company hopes will help it meet the rapidly growing demand for plasmid DNA-based therapies and vital mRNA-based vaccines. Read More
Gene therapy tackles 2 rare childhood diseases 2 different ways
Two new studies point to the efficient use of gene therapy to treat deadly childhood genetic diseases. The first study used direct gene therapy infusion to treat a rare neurodevelopmental disorder, while the second study used prenatal base editing in mouse models as proof of concept for the treatment of genetic diseases. These vastly different approaches show the promise of gene therapy for a variety of devastating diseases. Read More
Strateos launches hybrid SmartLab software platform
Strateos has launched SmartLab, a modular cloud-based software platform designed to assist life science research organizations in connecting onsite research and development labs with remote-controlled facilities. Read More
Twist acquires iGenomX for $35M
Twist Bioscience has acquired next-generation sequencing library preparation developer iGenomX for $35 million. The deal gives Twist a leg up in the shift from genotyping with static microarray platforms to using sequencing workflows. Read More
Intellia, Regeneron highlight clinical results for CRISPR-Cas9 gene therapy
Intellia Therapeutics and Regeneron Pharmaceuticals have announced positive interim data from an ongoing phase I study of their in vivo candidate, NTLA-2001, as a single-dose treatment for transthyretin amyloidosis. The interim results support the clinical efficacy and safety of in vivo CRISPR genome editing therapy in humans. Read More
Blackstone, Intellia, Cellex to launch company addressing limitations of cell therapies
Blackstone Life Sciences has committed $250 million toward the launch of a new autologous and allogeneic universal chimeric antigen receptor T-cell therapy company. Also taking part in the launch are Intellia Therapeutics and Cellex Cell Professionals, the parent company of GEMoaB, a clinical-stage cell therapy company. Read More
Danaher to acquire Aldevron in deal valued at $9.6B
Danaher announced it has signed a deal to buy DNA and messenger RNA manufacturer Aldevron for approximately $9.6 billion. Aldevron will operate as a standalone company in Danaher's Life Sciences business segment and is expected to boost Danaher's activities in genomic medicine. Read More
Conferences
CPhI North America
August 10-12
Philadelphia, Pennsylvania United States
Bioprocessing Summit
August 16-19
Boston, Massachusetts United States
6th Annual CAR-TCR Summit
August 30 - September 2
Online
European Congress of Immunology (ECI) 2021
September 1-4
Belgrade Serbia
Medical Affairs Strategic Summit (MASS)
September 13-15
New Brunswick, New Jersey United States
Science Briefs
Connect
Science Advisory Board on LinkedIn
Science Advisory Board on Facebook
Science Advisory Board on Twitter