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BioMed X has entered a research partnership with Sanofi to leverage artificial intelligence (AI) for drug development. This team will focus on creating a versatile computational platform that can precisely forecast the first-in-class drug candidates’ efficacy in virtual patient groups.
The US Food and Drug Administration (FDA) has granted an Orphan Drug Designation to Editas Medicine’s gene therapy EDIT-301 in sickle cell disease, based on an April 27 announcement. The US agency previously granted the Orphan Drug Designation to EDIT-301 for its study in beta thalassemia, in May 2022.
In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Artificial Intelligence in Pharmaceuticals: In-silico drug discovery. However, not all innovations are equal nor do they follow a constant upward trend.
TFF Pharmaceuticals and NIEHS will investigate the TFF-HMW-HA formulations’ therapeutic efficacy and pharmacokinetics using in vitro and in vivo models of respiratory diseases.
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 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.
Since that discovery, a flurry of gene-editing focused biopharma companies have launched – including Intellia Therapeutics, CRISPR Therapeutics, Caribou Biosciences and Mammoth Biosciences – and the first drug therapies based on the technology are now in human testing for diseases like cancer. Zinc fingers. billion in funding.
Under the agreement, Sanofi will receive non-exclusive rights to Scribe’s CRISPR by Design platform of wholly-owned enzymes for developing ex vivo NK cell therapies. The alliance will leverage the CRISPR genome editing technologies of Scribe to facilitate in genetic modification of new natural killer (NK) cell therapeutics for cancer.
German drugmaker Merck KGaA has reached an agreement to pilot an artificial intelligence platform developed by Quris that aims to identify potential safety problems with new therapeutic candidates as early as possible in the drug discovery process.
Single cell sequencing has revolutionized the study of biological tissues and systems at the cellular and molecular level. Recent advances in the technology have allowed for the interrogation of distinct subsets of cell populations within tissues, and associated molecular markers that may function as important disease drivers.
Innovation S-curve for the pharmaceutical industry Transgenic murine models is a key innovation area in pharmaceutical s Transgenic murine models refer to mice that have been genetically altered for the purposes of understanding the in vivo functions of genes. Regeneron Pharmaceuticals is the leading patent filer in transgenic murine models.
As we step into 2024, the life sciences 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. Regulatory bodies are also taking note of the applications of AI in drug development.
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.
Cell therapies hold life-saving potential for patients, and the race is on to develop a best-in-class, living, ‘thinking’ drugs. Implement logic and develop ‘decision-making’ cells: create robust, controllable activation in vivo with University of Pennsylvania and Ludwig Institute for Cancer Research Lausanne.
Its main platform – called X-editing – can generate CRISPR drugs with improved activity, specificity and deliverability compared to rivals, according to the start-up’s website. Scribe has designed, engineered and tested thousands of evolved CRISPR enzymes to build an advanced platform for creating breakthrough in vivo treatments,” said Oakes.
There are three main types of coronary or carotid artery stents : bare metal stents, drug-eluting stents that are coated with drugs to be slowly released into the artery to help prevent the artery from clogging again and biodegradable stents for temporary needs. Cardiovascular diseases are the number one cause of death in the world.
Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Bluebird has a total of three gene therapies in its pipeline.
This Hanson Wade event is aimed at experts looking to deliver CNS drug candidates safely and successfully across the blood-brain barrier (BBB). Discover the role of neural extracellular vesicles in non-invasive CNS drug delivery, and how the properties of extracellular vesicles can be enhanced with unique surface modifications.
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.
“KSQ’s CRISPRomics discovery platform is a powerful technology to help us identify novel targets in line with our immuno-oncology strategy,” said Loïc Vincent, Head, Oncology Drug Discovery Unit and Immunology Unit at Takeda. Takeda will assume responsibility for funding all development and commercialization activities.
Beam adds to drug delivery stable with USD 120 Million GuideTx buy. Electroporation is used for ex vivo delivery of therapies to blood and immune cells. The company’s in vivo therapies reach the eye and central nervous system via adeno-associated viruses, while its liver therapies employ lipid nanoparticles (LNPs).
The companies will use ViGeneron’s proprietary vgAAV, novel engineered AAV capsids, to efficiently transduce retinal cells via intravitreal injections. The companies will work together on the in vivo proof of concept (POC). Gene therapy has become a clinical reality. For further information, please visit www.vigeneron.com.
Biopreservation is the exclusive biological approach designated for the preservation of cells, tissues and organs ex-vivo for the purpose of storage, shipment and transportation. Biopreservation media is one of the most crucial entity in conferring effectiveness. F) to 8°C (46.4°F).
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.
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.
Drawing upon 50 years of expertise in clinical research, biopharmaceuticals, biotechnology and drug discovery, Dr. Van Kampen excels as the chief executive officer of The Van Kampen Group, which assists other companies in research and liaisons with government and industry regarding licensing products for commercial use.
Such challenges highlight a significant unmet need and call for further advancements in the field. Vittoria Biotherapeutics is a clinical-stage cell therapy company that incorporates the latest advancements in cell therapy and gene editing with a unique approach that potentially overcomes some of the issues with this therapeutic modality.
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. BeyondSpring – BeyondSpring Inc. At Spark, he served as head of U.S. marketing and diagnostics.
Iovance Biotherapeutics’ Amtagvi (lifileucel) won US Food and Drug Administration (FDA) approval last week for the treatment of advanced melanoma, making it the first individualized tumor-infiltrating lymphocyte (TIL) therapy and the first T-cell therapy for a solid tumor to win US regulatory approval.
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.
While no cure is available for MS, existing disease-modifying therapies in the form of small molecule and protein drugs either directly target the self-reactive immune cells or broadly dampen inflammation. Now, a research team at the Wyss Institute for Biologically Inspired Engineering at Harvard University and Harvard John A.
The synergistic behavior achieved from the process circumvents the inherent limitation of the individual counterparts, such as short in vivo half-life, low solubility and immunogenicity. Moreover, it provides a better understanding of the disposition of the drug and its role in causing organ toxicity.
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.
Xtalks spoke with WeiQi Lin, MD, PhD, executive vice president, research & development, and principal scientist at DURECT Corporation — a company focused on developing drugs and drug delivery methods in therapeutic areas with significant unmet need — about her transition from academia to industry. Key Moments.
Kiromic chPD1 has shown in preclinical data to show a cytotoxic response in 9 different in vivo models with 100% long-term PFS with the induction of host memory responses.
chPD1 will be used in the Company’s proprietary chimeric antigen receptor therapy (CAR-T) platform using gamma-delta T-cells (GD-T).
About Kiromic.
Following the unauthorized download of all abstracts on the SITC website, Transgene is communicating the content of the late-breaking poster abstract that will be presented at the SITC 35th Anniversary Annual Meeting (SITC 2020), to be held virtually November 9-14, 2020.
Key findings of the trial:
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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 vivo trials. Topic sponsors are not involved in the creation of editorial content.
Their pipeline includes P-BCMA-ALLO1, a promising therapy targeting multiple myeloma that has received Regenerative Medicine Advanced Therapy designation and Orphan Drug designation from the US Food and Drug Administration (FDA). Poseida’s expertise in donor-derived, allogeneic CAR-T therapies offers a transformative edge.
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