The FDA today released draft guidance designed to make it easier for researchers to seek approvals for drugs designed for individuals with severe genetic diseases. The draft guidance focuses on investigational new drug submissions for antisense oligonucleotide (ASO) products because these are the most common drugs being developed for severely debilitating or life-threatening genetic diseases.…
FDA OKs intentional genomic alteration in pigs — with potential therapeutic applications
The FDA has approved an intentional genomic alteration (IGA) in a line of domestic pigs known as GalSafe. The decision marks the first time the agency has signed off on a biotechnology product for both food applications and potential therapeutic use. The news “represents a tremendous milestone for scientific innovation,” said FDA Commissioner Dr. Stephen…
Oxford Gene Technology expands next-generation sequencing line
Oxford Gene Technology (OGT) has expanded its SureSeq line of next-generation sequencing (NGS) panels to detect genetic variants associated with breast and ovarian cancer and myeloid disorders. One of the offerings is a myeloid panel that can detect abnormalities in 70 genes associated with a range of diseases, including acute myeloid leukemia, myelodysplastic syndrome and…
The top 10 medical disruptors of 2021
For the past 18 years, the Cleveland Clinic has predicted what the top 10 medical disruptors will be for the following year. The list of technologies, cultivated by a panel of physicians and scientists at the clinic, was led by Dr. Will Morris, executive medical director of Cleveland Clinic Innovations, and Dr. Akhil Saklecha, managing…
Illumina may buy back Grail
Illumina (NSDQ:ILMN) may acquire Grail for more than $8 billion — four years after it spun out the cancer detection startup. That’s according to a Bloomberg report out yesterday, which cited people familiar with the matter. Analysts told Bloomberg that the acquisition would put Illumina — a giant in the DNA sequencing space — in direct…
Mission Bio completes $70M Series C for its tech to enable better cancer treatments
Mission Bio has secured $70 million in Series C financing to scale its single-cell multi-omics technology, according to the company. Novo Growth, the growth equity of Novo Holdings, led the round that included new investor Soleus Capital. Previous investors Mayfield, Cota, and Agilent also participated in the financing, which brings the company’s total funding to…
Sherlock, Binx to develop first CRISPR-based POC test for COVID-19
Sherlock Biosciences and Binx Health recently signed a strategic partnership to develop a point-of-care diagnostic test for COVID-19 that uses CRISPR technology. Through the collaboration, Binx’s IO diagnostic platform will be combined with the Sherlock CRISPR technology to develop a test that is simple to use. The test is designed to provide rapid and accurate…
New web resource keeps vaccine researchers up-to-date on COVID-19 protein structures
Wladek Minor of the University of Virginia and other top structural biologists have created a Web resource so that scientists can track the progress of investigations into COVID-19 protein structures. The website also includes the research team’s assessment of the quality of the individual models. It includes enhanced versions of the protein structures when possible.…
Sandia researchers look for genetic bullet against COVID-19
Researchers at Sandia National Laboratories are using genetic resequencing tools to find ways to stop the virus behind the COVID-19 pandemic. Biochemist Joe Schoeniger and virologist Oscar Negrete’s work involves genetically engineering a deployable antiviral countermeasure for COVID-19 using CRISPR-based technology. “The goal is to find new ‘reloadable’ countermeasures for viral outbreaks that do not…
NIH says still no drug proven safe, effective for treating COVID-19
The National Institutes of Health today announced the publishing of new COVID-19 treatment guidelines, and the expert panel producing the guidelines says no drug has yet been proven safe and effective against the virus. NIH has the new guidelines posted online at covid19treatmentguidelines.nih.gov — and says it will update the guidelines often as new peer-reviewed…
Sanofi, GSK seek to have an adjuvanted COVID-19 vaccine ready by late 2021
Sanofi and GSK recently announced that they have signed a letter of intent to collaborate on an adjuvanted vaccine for COVID-19 — with a goal of having the vaccine available by the second half of 2021. Sanofi is contributing its S-protein COVID-19 antigen. The company’s recombinant DNA technology produced an exact genetic match to proteins…
Mammoth preparing to seek EUA for CRISPR COVID-19 test
CRISPR-startup Mammoth Biosciences will file for an emergency use application from the FDA after publishing a study in Nature Biotechnology that suggests its platform can detect SARS-CoV-2 from a respiratory swab RNA extracts in less than 45 minutes. The company says the paper contains the first peer-reviewed data using CRISPR diagnostics for COVID-19. In a…
SGI-DNA touts its automated gene printer for coronavirus vaccine development
Synthetic biology company SGI-DNA is touting its efforts to accelerate research in the creation of vaccines for SARS-CoV-2, the virus causing the COVID-19 pandemic. The San Diego-based company said in a news release last month that it built the genes thought to be critical to developing successful vaccines against the virus and made those tools…
Evonetix raises $30m for desktop gene synthesis tech
Evonetix announced today that it closed a $30 million Series B funding round as it develops technology for enabling gene synthesis on a chip. The Cambridge, U.K.-based synthetic biology company said the funding round, led by new investor Foresite Capital, is slated to fund the company through to the introduction of its desktop DNA platform,…
Sanofi eyes Synthrox purchase
Sanofi (NYSE:SNY) announced today that it is planning to tender an offer to acquire all outstanding common stock shares of clinical-stage biotech company Synthorx. The agreement and plan of merger between the two companies was announced on Dec. 7 and the offer is being made pursuant to that. Sanofi said it stands to acquire the common…
Horizon Discovery announces partnership with Human Protein Atlas
Horizon Discovery Group (LSE: HZD) announced this week that the Human Protein Atlas will use Horizon’s CRISPR-edited knockout cell models to further expand the knowledge available in its Cell Atlas program. Horizon specialized in the application of gene editing and gene modulation for cell line engineering. Knockout cell models offer the possibility for an enhanced…
‘Hubble Space Telescope’ for genomics could yield new drugs, materials to save species
Researchers in Spain have developed a new genomics tool that can classify how species are related to each other at far larger scales than previously possible and may yield new drugs, materials and foods — and pinpoint strategies for saving species at risk of extinction. Heralded as a “Hubble Space Telescope” of genomics, the technology…
GenapSys secures $90 million in bid to upend sequencing
Following the raising of a $90 million Series C, privately held GenapSys will begin rolling out a line of low cost electronic-based genetic sequencing machines that it says could upend the research and clinical testing industries. CEO Hesaam Esfandyarpour says the Redwood City, Calif.–based company’s Sequencer, a table-top device that carries a price tag that’s…
New Genetic Weapons Challenge Sickle Cell Disease
Help for patients with sickle cell disease may soon come from gene editing to fix the mutation that causes the disease and boost the patient’s own protective fetal hemoglobin. New research shows that using CRISPR-Cas9 and a corrective short DNA template to repair the sickle cell mutation in a patient’s hematopoietic stem cells (HSCs) could…
Natural Gene Therapy for Intractable Skin Disease Discovered
Pathogenic gene mutations causing a type of intractable skin disease can be eliminated from some parts of patients’ skin as they age, according to Hokkaido University researchers and their collaborators in Japan. This represents a form of natural gene therapy. In general, there is no fundamental treatment method capable of curing diseases caused by gene…
Estimating the Efficacy and Cost of Curative Gene Therapy for Beta-Thalassemia
Gene therapy offers the promise of a cure for beta-thalassemia and a new study has shown that it is associated with fewer complications and hospital admissions over 2 years than treatment by allogeneic hematopoietic stem cell transplantation (HSCT). The study, which analyzes and compares the effectiveness and cost of gene therapy versus (HSCT) in patients…
Using CRISPR, Researchers Discover Treatment for Box Jellyfish Sting
Gene-Editing Technology May Produce Resistant Virus in Cassava Plant
New ‘Hairpin’ Approach Takes CRISPR to Next-Level Accuracy
Researchers may have found a way to significantly improve CRISPR/Cas9 gene editing technology. Biomedical engineers from Duke University have added a short tail to the guide RNA that folds back and binds onto itself to create a lock that can only be undone by the targeted DNA sequence, ultimately improving CRISPR’s accuracy by an average…
Russian Scientists Alter 3D Genome Using ‘Small Molecules’
Researchers have discovered that the spatial organization of the genome can be altered using small molecule compounds which are considered as promising anti-cancer drugs. This work opens up the prospect of developing a new class of anticancer epigenetic drugs that alter the 3D genome. The results were published in Nature Communications. The properties of a living…