With a landmark U.K. approval in hand for Casgevy (exagamglogene autotemcel [exa-cel]) to treat sickle cell disease and transfusion-dependent beta thalassemia, Crispr Therapeutics AG and partner Vertex Therapeutics Inc. are turning their attention to the PDUFA dates set by the U.S. FDA for the treatment in both conditions.
A new gene editing method uses the CRISPR technique to modify the cells of an organ in vivo, creating a mosaic used to identify the effects of each altered gene. Scientists from the Swiss Federal Institute of Technology (ETH) in Zürich developed this technology called AAV-Perturb-seq, based on adeno-associated virus (AAV) to target, edit and analyze single-cell genetic perturbations.
Alterations in chromosome number can play a role in cancer progression. An analysis of recurrent aneuploidies, such as the duplication of the long arm of chromosome 1, revealed that it was required for the proliferation of cancer cells carrying this alteration, an effect that was similar to so-called oncogene addiction. These findings have therapeutic implications that could benefit cancer patients depending on the genetic singularity of their tumor cells.
Promising early data continue to roll out for Intellia Therapeutics Inc.’s hereditary angioedema (HAE) candidate, NTLA-2002, with one of the earliest treated patients in the phase I study remaining attack-free for more than a year. But it was the systemic CRISPR candidate’s potential as a one-time treatment that generated the most discussion on the company’s call as investors tried to assess its potential advantage in a crowded HAE market.
The discovery of DNA was a milestone in the history of science that led to a breakthrough in biomedical research. By associating disease and genetics, genome correction techniques were ultimately developed that are supposed to work in the same way that antibiotics and antivirals block pathogenic microorganisms: by directly attacking the causes of disease.
The discovery of DNA was a milestone in the history of science that led to a breakthrough in biomedical research. By associating disease and genetics, genome correction techniques were ultimately developed that are supposed to work in the same way that antibiotics and antivirals block pathogenic microorganisms: by directly attacking the causes of disease.
Scribe Therapeutics Inc. is selling exclusive rights to its CRISPR-based technology to Prevail Therapeutics Inc. for a figure that could top $1.6 billion as the firms team up to develop genetic therapies for serious neurological and neuromuscular diseases.
A research initiative led by Jennifer Doudna and Jill Banfield of the Innovative Genomics Institute at the University of California, Berkeley, has landed $70 million in funding from the Audacious Project to bring the power and precision of CRISPR-based genome editing to the gut microbiome of humans and animals, in an ambitious effort to engineer complex microbial communities to achieve outcomes that can benefit human health and the environment.
Function Oncology Inc. emerged from stealth on April 12 with the announcement of a $28 million series A financing that will continue support development of its CRISPR-enabled platform to profile cancer in patient-specific detail. The platform goes beyond next-generation sequencing to measure gene function, potentially allowing identification of new therapeutic targets and better matching of available therapies to vulnerabilities in an individual’s tumors.
The researcher who pioneered prenatal surgery to correct neural tube defects has turned her attention to using CRISPR-edited gene therapies to correct severe monogenic diseases in utero. The availability of prenatal genetic diagnosis and advances in treating fetuses, and also in gene therapy/gene editing, make it possible to repair almost any defect in the genetic code. At the same time, there is a clear rationale for intervening before birth, Tippi MacKenzie, professor of surgery at UCSF’s School of Medicine, told attendees of the third International Human Genome Editing Conference in London on March 7.
