Platform enables precise, efficient delivery of genetic medicines in vivo
Biotech company Homology Medicines is leveraging 15 human hematopoietic stem cell-derived adeno-associated virus vectors to precisely and efficiently deliver genetic medicines in vivo via gene therapy or nuclease-free gene editing modality. CEO Albert Seymour spoke with Science Advisory Board about their platform. Read More
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
Healing properties of senescent cells raise questions about anti-aging research
University of California, San Francisco researchers have found some senescent cells help to heal damaged tissues, raising questions about the merits of killing them to address age-related diseases. 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
Benchtop single-molecule protein sequencing unlocks new way to study proteome
Life sciences company Quantum-Si has developed a benchtop instrument that performs single-molecule protein sequencing, setting the stage for a new approach to studying the proteome. 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
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