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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.
As we step into 2024, the lifesciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. These companies are at various stages of research and clinical trials.
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.
Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. Meanwhile, others are researching the link between gene variation and different responses to treatments.
Drs Emmanuelle Charpentier and Jennifer Doudna have won this year’s Nobel Prize for chemistry in recognition of their work on the gene-editing technology CRISPR/Cas9. There is enormous power in this genetic tool, which affects us all. What started as a curiosity?driven, — The Nobel Prize (@NobelPrize) October 7, 2020.
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.
The lifesciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the lifesciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.
Innovations in Cancer Therapy CRISPR/Cas9, a groundbreaking gene-editing technology, has demonstrated significant potential in oncology, offering new avenues for cancer treatment. By tailoring treatments based on an individual’s genetic makeup, it may allow for more effective and targeted therapies.
Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.
Hemophilia B, also known as congenital Factor IX (FIX) deficiency or Christmas disease, is a rare genetic bleeding disorder caused by a deficiency or defect in the FIX clotting protein. Pfizer said that according to the World Federation of Hemophilia, more than 38,000 people worldwide were living with hemophilia B in 2021.
Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself.
One area of the gene therapy space that could become a major treatment modality is CRISPR technology. The key to why this technology could become central to geneediting is due to the specificity with which it can edit DNA and that it is also a relatively cheap process, compared with existing technologies.
A downstream approach has traditionally been used by scientists to map networks, wherein a gene for a protein is removed one at a time and the impact on the immune cell’s function is observed. CRISPR-Cas9 geneediting was used to simultaneously perturb thousands of genes by targeting specific points of genetic code.
For CRC, treatment options that are targeting genetic mutations such as BRAF , KRAS and HER2 have been developed. Building Cancer Awareness and Empowering Métis Youth: Featuring Jordyn Playne, President, Métis Nation of Ontario Youth Council – Xtalks LifeScience Podcast Ep.
Technologists are getting better at coding biology, and venture firms are flooding a new generation of startups with cash to commercialize their technology bringing in the next wave of genetic innovation. Verve Therapeutics unveils its lead program for treating genetically high cholesterol.
More Investigational Therapies for HIV Are on the Way American Gene Technologies, a pioneering biotech company headquartered in Rockville, Maryland, has released promising results from its Phase I trial of AGT103-T, a gene therapy designed for individuals with chronic HIV disease.
CRISPR works as genetic scissors to edit parts of the genome. The CRISPR-Cas9 geneediting system was first discovered to be endogenous in bacteria. Given the system’s powerful ability to make cuts in genes in a precise manner, the system is being leveraged in gene therapies.
The HELIOS-B study’s findings are timely as new gene-editing therapies, like the one being studied in MedStar Health’s Phase III MAGNITUDE trial for ATTR-CM, are also being explored. The MAGNITUDE trial uses CRISPR/Cas9 technology to target the genetic source of the disease, potentially offering a one-time treatment.
Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself.
Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US. In 2022, bluebird won approvals for two gene therapies — Skysona for the treatment of the rare neurological disorder cerebral adrenoleukodystrophy (CALD) and Zynteglo for beta-thalassemia.
There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes.
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.
This has ushered in a new era of genomics that is fostering rapid, detailed and personalized insights into human genetics. Xtalks is celebrating the International Day of Women and Girls in Science with a special overview of notable female scientists who have made revolutionary contributions to the field of genetics.
A study from the University of California San Francisco (UCSF) has revealed that women with Alzheimer’s do better than men with the disease due to a genetic advantage conferred by their extra X chromosome. Genetic Sex Differences in Disease.
Tripling its production capacity, Touchlight can now manufacture 8kg plasmid DNA, a key component for mRNA gene therapies and vaccines. As per the company, dbDNA is suitable for rapid, scalable manufacture of GMP DNA and can incorporate gene sequences of sizes ranging from 500bp to 20kb, which are typically unstable as pDNA.
GeneEditing CRISPR-Cas9 and related gene-editing technologies continue to advance, and today they are widely used to study and develop therapeutic approaches for a broad range of human diseases. Furthermore, CRISPR/Cas9 presents a promising avenue for overcoming genetic diseases in the near future.
Kristin Yarema, President of Cell Therapy at Poseida Therapeutics – Xtalks LifeScience Podcast Ep. 129 Through this acquisition, Roche secures proprietary technologies such as Poseida’s Cas-CLOVER geneediting platform and in-house good manufacturing practice (GMP) manufacturing capabilities.
Casgevy: A Game-Changer for Beta Thalassemia CRISPR Therapeutics and Vertex are transforming blood disorder treatment with their gene-editing solution Casgevy (exagamglogene autotemcel [exa-cel]). This one-time gene therapy tackles transfusion-dependent beta-thalassemia (TDT), a disorder causing severe anemia.
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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