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
The robust expertise of IASO in monitoring possibly best-in-class CARs leveraging its fully-human antibody discovery platform (IMARS), quickly conducting clinical trials and its in-house GMP facility for producing plasmid, virus vector and CAR-T cells intends to make its new therapies available to larger population across the world.
EDIT-301 is made of patient-derived CD34+ haematopoietic stem and progenitor cells, which are edited using CRISPR at the gamma globin gene (HBG1 and HBG2) promoter sites using a proprietary engineered AsCas12a nuclease, per the company’s website. The first patient had a total haemoglobin level of 16.4 g/dL and 45.4%
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.
The alliance plans to leverage the single-cell adeno-associated virus (AAV) engineering (scAAVengr) platform technology of Avista to develop intravitreal AAV capsids for a Roche-defined profile. This aids in quickly advancing transformative gene therapies to clinical trials for ophthalmic ailments. Avista is entitled to receive $7.5m
As an autologous cell therapy, manufacturing the infusion product is a highly specialised process involving tumour harvest and the extraction and large-scale ex vivo expansion of TILs. The use of engineered immune cytokines that selectively induce the expansion of TILs is being investigated as an alternative to IL-2 stimulation.
California-based Sangamo Therapeutics is one such company that believes in the powerful potential of in vivo genome editing and regulation, together known as genome engineering, and has built up a sizable preclinical pipeline of genome regulation treatments for diseases such as Huntington’s disease and Amyotrophic lateral sclerosis (ALS).
The company is currently progressing its lead drug candidate, RYZ101, into clinical trials for various solid tumour indications. A Phase Ib clinical trial of the drug is underway, enrolling subjects with neuroendocrine tumours, and Phase III trials are anticipated to commence next year.
Innovation S-curve for the pharmaceutical industry Gene splicing using nucleases is a key innovation area in the pharmaceutical industry Nucleases play a fundamental role in the field of recombinant DNA technology, or genetic engineering. However, not all innovations are equal and nor do they follow a constant upward trend.
Quris’ BioAI safety prediction platform – which is based on human tissue samples on chips, nanosensors and machine learning – will be compared to traditional in vitro and in vivo laboratory techniques for spotting toxicity. The post Merck will assess Quris’ AI ‘patient-on-a-chip’ drug safety appeared first on.
With several biologics in the pipeline, including one in early stage clinical trials for head and neck cancer, the company is positioned to engineer highly specific biologics against immune targets in the body. Cell-Based Immunotherapy vs. Immune Biologics. Immunotherapy involves leveraging components of the immune system (i.e.
Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. Launched in 2019, the company raised $700 million in June 2020 in its initial financing.
16, 2020 /PRNewswire/ –: iSTAR Medical , a medtech company developing minimally invasive ophthalmic implants for treatment of patients with glaucoma, today announced positive one-year results from the European STAR-II clinical trial of its micro-invasive glaucoma surgery (MIGS) device, MINIject TM. WAVRE, Belgium , Nov.
XTALKS WEBINAR: How Agile Clinical Trials Unlock Universal Access to Rare Disease Research. Register for this free webinar to learn why the agile clinical trial model is a great fit for many rare disease categories, how to navigate the regulatory landscape for agile clinical trials in rare diseases, how to design studies and more.
Scribe has designed, engineered and tested thousands of evolved CRISPR enzymes to build an advanced platform for creating breakthrough in vivo treatments,” said Oakes. Further back in development are drugs that will be administered to edit genes within the body, but the first of these candidates are now in clinical trials.
This includes analyzing how drug combinations may impact individual patients or groups of patients before even entering a clinical trial. With a staggering 90 percent of drugs failing in clinical trials, AI has the potential to help improve these statistics.
As the area of bringing a digital therapeutic to market means passing the same level of regulatory scrutiny as a traditional, pharmaceutical therapy, this means “prescription digital therapeutics must undergo extensive clinical trials to demonstrate their safety and effectiveness before they ever reach patients,” said McCann.
XTALKS WEBINAR: Cardiac Safety Solutions in Decentralized Clinical Trials (DCT). Cardiovascular diseases are the number one cause of death in the world. According to the World Health Organization (WHO), almost 18 million people died from cardiovascular diseases in 2019 , which was 32 percent of all deaths globally.
CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.
With the launch of Overland, we are creating a robust research and development engine in parallel with the significant growth and opportunity presented by Asia’s evolving biotech industry. A solution could be at hand with Overland Pharmaceuticals. Below is a roundup of some of this week’s interesting news.
Owing to the ability to preserve a wide variety of elements for such a long period of time, including cells, tissues, blood and DNA, cryopreservation has emerged as a key focus area for stakeholders in the biopharmaceutical industry. Therefore, in order to store and preserve such enormous amounts of blood, single-use assemblies are a necessity.
Previous research has shown that SARS-CoV-2 spike-specific monoclonal antibodies play a key role in providing in vivo protection and have been used in developing COVID-19 vaccines. A modified version of the Cv2.1169 antibody was also effective at treating SARS-CoV-2 infection in mice and hamsters.
Our antibody clones in their original IgG format have shown potent neutralization activity in in vitro assays and, in the case of our lead clone, in an in vivo animal model. Headquartered in Mountain View, California, IGM Biosciences is a clinical-stage biotechnology company focused on creating and developing engineered IgM antibodies.
In Silico Clinical Trials, where “device performance is evaluated using a ‘virtual cohort’ of simulated patients with realistic anatomical and physiological variability representing the indicated patient population.”. Draft Guidance at 4. Draft Guidance at 8. Draft Guidance at 5.
XTALKS CLINICAL EDGE: Issue 3 — Interview with CTTI Xtalks Clinical Edge is a magazine for clinical research professionals and all who want to be informed about the latest trends and happenings in clinical trials. Such challenges highlight a significant unmet need and call for further advancements in the field.
TIL vs CAR T-Cell Therapies While CAR T-cell therapies involve inserting a CAR engineered to target a specific cancer cell protein into a patient’s T cells, TIL-based T-cell therapies like Amtagvi involve separating TILs from a patient’s tumor (in autologous therapies) and expanding them ex vivo.
ImmunityBio has engineered the vector to overcome this problem and has shown that its second-generation adenovirus vector can safely and effectively deliver its cargo even in patients with pre-existing adenovirus immunity. This blocking of viral replication was observed in both the lung and nasal passages.
He is currently a professor at the University of Copenhagen where his lab focuses on PNAs in regard to drug discovery, gene targeting, antisense principles, cellular and in vivo delivery and administration of biopharmaceuticals. The Advance awards recognize the work of remarkable global Australians making an extraordinary impact worldwide.
Overview of Gene Switch The notion that genes might be turned on and off was discovered several decades ago when studies revealed that E. coli bacteria, as well as lambda bacteriophage, can adapt to the alterations in the composition of their nutrient medium. These genetic switches assist transcription factors in binding to the promoter region.
In Silico Clinical Trials, where “device performance is evaluated using a ‘virtual cohort’ of simulated patients with realistic anatomical and physiological variability representing the indicated patient population.”. Draft Guidance at 4. Draft Guidance at 8. Draft Guidance at 5.
Applications of mRNA Vaccine and Therapeutics Recent advances using nucleoside-modified mRNA improved protein expression and diminished activation of innate sensors allowing largely overcoming conventional roadblocks concerning mRNA stability, inefficient in vivo delivery and activation of innate sensors by RNA molecules.
The purpose of this exploratory Phase 1b/2 trial was to evaluate the safety and efficacy of the combination of TG4001 and an immune checkpoint inhibitor in a heterogeneous group of patients with aggressive, recurrent and/or metastatic HPV16-positive cancers.
Key findings of the trial:
.
The drug is currently in clinical trials for alcoholic hepatitis (AH), nonalcoholic steatohepatitis (NASH), as well as in COVID-19 patients with acute liver or kidney injury. However, this has since changed, and now, transitioning between academia and industry has become more commonplace. Key Moments. A life-changing moment came for Dr.
It was engineered with an Fc-silent IgG1 isotype to avoid activation of Fc-receptor-mediated non-specific immune. The bispecific antibody was found to conditionally activate the immune pathway in the tumour microenvironment in vitro and in vivotrials. Topic sponsors are not involved in the creation of editorial content.
The acquisition also grants Roche access to Poseida’s P-CD19CD20-ALLO1, a dual-target CAR-T therapy currently in Phase I trials for B-cell malignancies. Additionally, Poseida’s solid tumor program, P-MUC1C-ALLO1, is advancing in Phase I trials, targeting antigen-expressing tumors with promising early results.
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