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Expanding upon the CRISPR-Cas9 geneediting system, researchers at MIT have designed a new technique called PASTE geneediting that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE geneediting technique was recently published in Nature Biotechnology.
If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the lifesciences. Here is a look at some lifescience trends to expect in the coming year, including key themes and topics in the pharmaceutical, biotechnology and medical device industries to keep an eye out for in 2022.
Now a common geneediting tool, the popularity of the CRISPR-Cas9 system has increased over the past decade. CRISPR is notable for engineering living cells, allowing scientists to edit, turn off, delete, or replace genes in a cell’s genome. Harnessing the Cellular Engineering Potential of CRISPR.
Alnylam Pharmaceuticals announced promising results from its HELIOS-B Phase III clinical trial evaluating vutrisiran, an investigational RNA interference (RNAi) therapeutic for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM). As of March 2024, the FDA has approved six small interfering RNA (siRNA) therapies.
Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the LifeSciences. The CRISPR geneediting system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing.
Over 26 weeks of Sunlenca combined with other antiretroviral drugs, 81 percent of participants achieved HIV RNA suppression, reaching levels low enough to be considered undetectable. These findings signify a significant step forward in the development of AGT103-T as a potential gene therapy for HIV. percent in the placebo group.
Altitude LifeScience Ventures, and the second SoftBank Vision Fund, with participation from the Qatari Investment Authority and other undisclosed investors. Verve Therapeutics has an aim to bring one-and-done geneediting treatments to heart disease. The round was led by Alaska Permanent Fund Corp.,
Related: 2020 Year in Review: COVID-19, CRISPR and Immunotherapies Define the Year for the LifeSciences. The CRISPR geneediting system consists of the Cas9 enzyme, which serves as molecular scissors to cleave double-stranded DNA, and a guide RNA template targeted to a specific genomic sequence, which allows for precise editing.
With Blackstone LifeSciences, up to €300 million (around $315 million USD) will be invested to accelerate pivotal studies and clinical development programs for formulating the subcutaneous delivery of the anti-CD38 antibody Sarclisa (isatuximab) for the treatment of multiple myeloma. Veklury generated a revenue of $3.91 billion ($19.19
The pandemic propelled the lifescience and healthcare sectors onto center stage, and they rose to the occasion against the most unprecedented health challenge in recent times. While COVID-19 has undoubtedly been the biggest story in the lifescience industry in 2020, it was a busy and positive year in many other areas.
A female’s second X chromosome is typically silenced (so that females don’t get an overdose of X chromosome genes) through X-linked inactivation, a process where the chromosome is coated with an outer layer of non-coding RNA. One of these doubly active genes is KDM6A.
From leveraging artificial intelligence (AI) to streamline diagnostics and treatments to exploring the untapped potential of RNA-based therapeutics, biotechnology is shaping the future of healthcare and beyond. As of January 31, 2024, approximately 131 unique RNA-based therapies are in clinical development across various therapeutic areas.
Gene therapy has shown promise in treating cancers that are particularly difficult to manage such as neuroblastoma and Wilms tumor using CAR-T cell therapy, CRISPR-Cas9 geneediting and RNA-based interventions to target genetic drivers of disease.
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