Manufacturing remains big challenge for cell and gene therapy industry
Science Advisory Board spoke with Eric Blair, chief commercial officer for Andelyn Biosciences, at last week’s 2022 Cell & Gene Meeting on the Mesa about the challenges of manufacturing to meet growing demand in the cell and gene therapy industry. Read More
Gene editing advances continue a pace, but progress is still early: panel
With the first-ever approval of a CRISPR gene editing therapy expected in 2023, companies at last week’s Cell & Gene Meeting on the Mesa say they are looking forward to other near- and longer-term breakthrough technologies on the horizon. Read More
Roadblocks continue to hamper CGT companies as they look to commercialize their products
The cell and gene therapy (CGT) sector will continue to face investment and regulator challenges in the near term. That’s the consensus of companies in the space who participated in a workshop at this week’s Cell & Gene Meeting on the Mesa in Carlsbad, CA. Read More
FDA biologics chief outlines ways to remove barriers to cell and gene therapies
At this year's Meeting on the Mesa, the U.S. Food and Drug Administration’s top biologics regulator said the use of a “cookbook” for developing cell and gene therapy products and global regulatory convergence could help to facilitate their development and take them to the next level. Read More
CRISPR-Cas9 screen uncovers chance to repurpose drugs to treat sickle cell disease
A CRISPR-Cas9 screen has generated insights into the switch from fetal to adult hemoglobin that suggest a new approach for treating beta hemoglobinopathies such as sickle cell disease. Read More
New ARM CEO provides vision for cell and gene therapy sector, need to address stakeholder concerns
Timothy Hunt, the new CEO of the Alliance for Regenerative Medicine (ARM), kicked off the 2022 Cell & Gene Meeting on the Mesa, laying out his vision for the industry and emphasizing the need to engage all major stakeholders. Read More
CRISPR-Cas9 variant enables DNA cutting at ‘practically any sequence’
Massachusetts General Hospital researchers have engineered a CRISPR-Cas9 variant that overcomes a previous restriction on the locations that DNA can be cut in the laboratory, suggesting molecular cloning approaches can be simplified. Read More
Panel to focus on pluripotent stem cells at Cell & Gene Meeting on the Mesa
A panel on October 13 at the Cell & Gene Meeting on the Mesa in Carlsbad, CA, will focus on new pluripotent stem cell (PSC) developments and advancements as well as the challenges of scaling up PSC-derived cell therapies. Read More
Gene therapy one step closer to delivery across blood-brain barrier: study
Brigham and Women’s Hospital researchers say the rational design of adeno-associated viruses has generated gene therapy delivery vectors that are better at crossing the blood-brain barrier to treat diseases of the central nervous system. Read More
Mechanistic link between mutation, brain cancer opens up R&D opportunities
Researchers have identified the mechanisms that mean people with a single nucleotide polymorphism have an increased risk of a subtype of brain tumors. Read More
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