This site uses cookies to improve your experience. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country, we will assume you are from the United States. Select your Cookie Settings or view our Privacy Policy and Terms of Use.
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Used for the proper function of the website
Used for monitoring website traffic and interactions
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Strictly Necessary: Used for the proper function of the website
Performance/Analytics: Used for monitoring website traffic and interactions
In this continuing series, BioPharma Dive examines new areas of biotech investment and startup activity, such as next-generation RNAmedicines, TYK2 inhibitors and in vivo CAR-T.
Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. By self-circularisation, Orna’s oRNA technology makes circular ribonucleic acids (oRNAs) from linear RNAs.
So far, human trial data for gene-editing drugs have been generated for ex vivo therapies, meaning the CRISPR technology is used to modify cells outside the body, which are then reinfused into the patient. — Eric Topol (@EricTopol) June 26, 2021. — Eric Topol (@EricTopol) June 26, 2021.
Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Our novel editing systems have the potential to precisely modify gene targets for both in vivo and ex vivo therapeutic development.”
Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. dosing of *first patient* with VERVE-101, an in vivo CRISPR base editing medicine. Today: we are announcing.
Singapore scientists uncover potential role of long non-coding RNAs in pancreatic cancer Credit: From Figure 4 in Liu, S., Wnt-regulated lncRNA discovery enhanced by in vivo identification and CRISPRi functional validation. Harmston, N., Glaser, T.L. Genome Med 12, 89 (2020).
In recent webinars by Genuity Science, formerly known as WuXi NextCODE, experts from the biotech and pharma industries spoke about leveraging the power of single cell RNA sequencing platforms and solutions in conjunction with machine learning technologies such as AI in cell biology and disease research. The Power of Single Cell Technology.
Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.
This program included assays such as Big Blue and PIG-a designed to provide a robust measure of a drug or chemical’s ability to induce mutations in vivo. The totality of the data from these studies indicates that molnupiravir is not mutagenic or genotoxic in in vivo mammalian systems. “We
Acuitas’ LNP technology will support Bayer’s in vivo gene editing and protein replacement programs by specifically delivering RNA payloads to the desired target organ, the liver. a biotechnology company specializing in the development of lipid nanoparticle (LNP) delivery systems for molecular therapeutics.
The intracellular barriers include endosomal escape, RNA sensors, and endonucleases. Ben Hargreaves speaks to Liberate Bio, a company that is hoping to address the delivery issues that could prevent mRNA, as well as siRNA and ASO therapeutics, from reaching their full potential.
Beam Therapeutics has taken over a startup whose technology could proffer the biotech’s genetic medicines to more tissues in the body, widening the potential to approach more diseases. Cambridge, Massachusetts-based Beam aims to make more precise edits with genetic medicines, which employ base-editing.
Back in September, Vertex Pharmaceuticals and CRISPR Therapeutics – the company behind the CRISPR Cas-9 platform – announced that the European Medicines Agency (EMA) had granted Priority Medicines (PRIME) designation to CTX001, an investigational ex vivo CRISPR Cas-9 gene-edited therapy for the treatment of severe sickle cell disease.
She received a master’s degree in Cardiovascular Pharmacology from West China Medical Center of Sichuan University and a bachelor’s degree in Clinical Medicine from Soochow University. Dr. Zhang has around 20 years of experience in research and drug development in both industry and academia in the US.
In recent years, mRNA technology has rocked the world of medicine, making it possible to prevent and treat a vast array of diseases and disorders. Vaccinations in the past have always been either weak or inactive forms of a pathogen which was given to the body so that our immune system is trained to recognize that given pathogen.
Studies have identified the presence of two types of HIV-infected CD4+ T cells: ‘Transcriptionally inactive’ cells that do not typically produce viral RNA or viral proteins. Transcriptionally active’ cells in which HIV RNA is actively transcribed to make copies of the virus (despite long-term ART).
The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies. Recent headlines have highlighted the potential for Chimeric Antigen Receptor (CAR)-based therapies to provide clinical benefits to persons affected by lupus. In these cases B cell depletion is a feature, not a bug.
.–( BUSINESS WIRE )– ImmunityBio , a privately-held immunotherapy company, today announced its COVID-19 vaccine candidate protected nasal and lung airways of non-human primates against coronavirus (SARS-CoV-2) in a challenge study. This blocking of viral replication was observed in both the lung and nasal passages.
We organize all of the trending information in your field so you don't have to. Join 21,000+ users and stay up to date on the latest articles your peers are reading.
You know about us, now we want to get to know you!
Let's personalize your content
Let's get even more personalized
We recognize your account from another site in our network, please click 'Send Email' below to continue with verifying your account and setting a password.
Let's personalize your content