70 Results
Sort By:
Published on July 13, 2021
The World Health Organization (WHO) has released findings from its two-year independent global consultation on how human genome editing tools such as CRISPR should be used in an ethical and non-harmful way to benefit human health, without holding back scientific progress. The 18-member consultation group was established after it was…
Published on April 20, 2021
Nearly six years after announcing a potentially $2.5 billion therapeutic development collaboration followed by an additional up to $1.175 billion agreement in 2019, Vertex Pharmaceuticals has, once again, thrown more money into the pot for CRISPR Therapeutics to lead the development, manufacturing, and commercialization of CTX001, the CRISPR-Cas9 gene-edited therapy…
Published on August 3, 2020
Biomarkers hold out the promise of significant time and money savings for drug discovery and development, but finding the right ones is notoriously tough. Many putative biomarkers fail validation after years of study, leaving developers poorer but not much wiser—a fact that highlights the complexity of biomarker development. But a…
Published on February 11, 2020
2019 saw a new record set for a list price of a prescription therapy—$2.1 million for the gene therapy Zolgensma launched by AxeVis, a Novartis Company—and resulting renewed concerns over the price of treatments and whether politicos will ever be able to address the issue as the nation’s presidential election…
Published on January 30, 2020
A new survey conducted by Zogby Research for the American Society of Human Genetics shows that 84% of Americans agree that more human genetics research is needed and 74% support increased federal funding for human genetics research. The survey also confirms the importance placed on confidentiality and security of research…
Published on January 9, 2020
For her whole life, Victoria Gray has lived with the debilitating pain of sickle cell disease, a genetic condition that affects millions worldwide. She’s endured transfusions and hospitals stays, and lived with the fear that deadly complications of the disease could leave her children motherless. In July, she was the…
Published on July 17, 2019
CRISPR-Cas9 is best known for its powerful ability to make double-stranded breaks in DNA, allowing scientists to delete and edit genes with relative ease. But switch out Cas9 for another protein, and CRISPR becomes a programmable tool for detecting the presence of certain nucleic acid sequences. This feature has startups…
Published on April 24, 2019
On the eve of DNA Day, leaders in the gene therapy community have co-signed a letter to Alex Azar, the Secretary of the Department of Health and Human Services (DHHS), urging a “binding global moratorium” on germline editing. The letter is signed by more than 60 luminaries in the fields…
Published on September 6, 2018
After scientists at the Wellcome Sanger Institute last month published findings that called the precision of CRISPR-Cas9 gene editing into question, three public companies focused on the technology saw their share prices fall to their 2018 lows before starting to climb back. Between March 9 and August 20: CRISPR Therapeutics…
Published on March 31, 2016
Determining the precise molecular structure of DNA was not only the launching point of what would become the field of modern molecular biology, but it also led scientists to avant-garde thinking about how to detect and ultimately treat disease. These novel concepts would quickly develop into a clinical reality that…
Published on January 21, 2016
The potential of genome editing has ignited a firestorm of novel ideas for the clinical application of technology for treating genetic diseases. However, the efficiency of the CRISPR-Cas9 system can often be low with respect to replacing the newly excised DNA strand with a nascent, unmutated copy. This has led…
Published on August 14, 2024
Artificial intelligence (AI)-based tools that rely on large language models (LLMs) struggle to correctly identify genetic conditions based on patient descriptions of their own health, show results from a study led by the National Institutes of Health in Bethesda. Lead investigator Benjamin Solomon, MD, clinical director at the NIH’s National…
Published on June 5, 2024
Just as 23andMe, once direct-to-consumer (DTC) testing’s darling, faced a huge drop in share value and once hot startup LunaDNA was shuttering, Panacea was promoting its new DTC genetic testing service. This might seem counter-intuitive, but consumer genetics is far from dead, even the DTC version. Panacea, for example, says…
Published on May 31, 2024
Researchers have identified a compact version of a gene-editing protein that is small enough to fit into adeno-associated viruses (AAVs) and can be modified to deliver highly efficient, targeted therapy to cells. The relatively small version of the CRISPR-associated protein Cas12a occurs naturally in the Erysipelotrichia class of bacteria and is…
Published on January 3, 2024
Gene editing firm Tome Biosciences has acquired startup Replace Therapeutics less than a month after Tome emerged from stealth with $213M in funding. This development adds fuel to an already hot field. In early December, Vertex and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel [exa-cel]) for sickle cell became the first ever…