new Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing. — pasadenanow.com, 1d ago

new BioNTech is best known for developing therapies that use messenger RNA to turn on immune responses to infectious diseases and cancer. However, it has quietly built an arsenal of experimental antibodies, some of which emerged from a collaboration with the Danish biotech Genmab, which first developed the Johnson & Johnson cancer drug Darzalex. — BioPharma Dive, 1d ago

new To tackle this gene alteration, QurAlis has developed its proprietary FlexASO splice modulator platform. This technology uses antisense oligonucleotides (ASOs) to correct mis-splicing, restore UNC13A protein production, and reduce cryptic exons that may contribute to disease progression. Kasper Roet, QurAlis CEO, explained that “FlexASOs operate on the same mechanism of action as other splice-switching ASOs. However, incorporation of FlexASO technology improves the potency, safety, and biodistribution of oligos.”... — Drug Discovery and Development, 1d ago

new Gene therapy has been heralded as the new frontier of medicine, but there are still many limitations to current technologies; among them, how to deliver therapeutic genes to specific cells, and only activate them in the right context. A team has created a new RNA-based tool called DART VADAR to bring gene editing out of the 'dark side' of those problems and into the light. Using an engineered form of a naturally occurring enzyme, their sensors can automatically sense the presence of a trigger molecule and initiate the translation of payload genes within cells. The advance broadens the scope of conditions that can be treated with RNA-based therapeutics and enables the development of highly specific treatments for a variety of diseases. — ScienceDaily, 1d ago

new ...technology, but researchers have been trying to master targeted treatments for decades, too. Now scientists believe they’re on the cusp of the next stage, with the development of a novel engineered RNA sense-and-respond circuit they’ve named Detection and Amplification of RNA Triggers via ADAR, or DART VADAR, which seeks out a specific molecular marker of disease or cell type involving the RNA-editing enzyme ADAR for highly specialized treatment. — New Atlas, 16h ago

new Nurix Therapeutics is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of small molecule and cell therapies based on the modulation of cellular protein levels as a novel treatment approach for cancer and other challenging diseases. Leveraging extensive expertise in E3 ligases together with proprietary DNA-encoded libraries, Nurix has built DELigase, an integrated discovery platform to identify and advance novel therapeutic candidates targeting E3 ligases, a broad class of enzymes that can modulate proteins within the cell. Nurix’s drug discovery approach is to either harness or inhibit the natural function of E3 ligases within the ubiquitin-proteasome system to selectively decrease or increase cellular protein levels. Nurix’s DELigase platform is broadly applicable across multiple therapeutic areas. Nurix’s wholly owned, clinical stage oncology and immunology pipeline includes targeted protein degraders of Bruton’s tyrosine kinase, a B-cell signaling protein, and inhibitors of Casitas B-lineage lymphoma proto-oncogene B, an E3 ligase that regulates activation of multiple immune cell types including T cells and NK cells. Nurix is headquartered in San Francisco, California. For additional information visit... — gilead.com, 2d ago

new ...“Among the most promising translational strategies of gene therapy for neurodegenerative diseases, ASOs have emerged as a viable, life-extending therapeutic approach by correcting fatal gene expression or RNA processing defects. This work demonstrates the... — Massachusetts General Hospital, 8h ago

new XNK Therapeutics is a clinical stage, immunotherapy company focusing its efforts on preventing and treating cancer by developing novel NK cell-based therapies. The company is at the forefront of the development of autologous NK cell-based products using its proprietary technology platform. The company's platform technology and lead investigational candidate drug was developed specifically to target cancers, including settings where allogeneic cell products are not readily applicable. The Company's objective is for its investigational candidate drug and proprietary platform technology to constitute key components in the cancer treatments of tomorrow. XNK Therapeutics is headquartered in Stockholm,... — prnewswire.co.uk, 1d ago

new ...explained, “A: ‘CRISPR’ (pronounced ‘crisper’) stands for Clustered Regularly Interspaced Short Palindromic Repeats, which are the hallmark of a bacterial defense system that forms the basis for CRISPR-Cas9 genome editing technology.”... — Conservative Firing Line, 16h ago

new ..., the researchers showed that a new genome editing technique called base editing can correct the mutation that causes CD3 delta SCID in blood stem cells and restore their ability to produce T cells. — medicalxpress.com, 1d ago

new RNAi-Based Therapeutics and Novel RNA Bioengineering Technologies... — securities.io, 2d ago

new Using an RNA sensor, MIT engineers have designed a new way to trigger cells to turn on a synthetic gene. The researchers demonstrated that their sensor could accurately identify cells expressing a mutated version of the p53 gene, which drives cancer development, and turn on a gene encoding a fluorescent protein only within those cells. Their novel approach may lead to targeted therapies for cancer and other diseases. — GEN - Genetic Engineering and Biotechnology News, 1d ago

new Aspect Biosystems is a biotechnology company pioneering the development of bioprinted tissue therapeutics to transform how we treat currently incurable diseases. Aspect is creating these next generation cell therapies by applying its full-stack tissue therapeutic platform, which combines the company’s proprietary bioprinting technology, therapeutic cells, biomaterials, and computational design. Aspect’s bioprinted tissue therapeutics are designed to replace, repair, or supplement biological function inside the body with the aim of treating currently incurable diseases such as type 1 diabetes as well as genetic and acquired liver diseases. Learn more at... — aspectbiosystems.com, 2d ago

new In the context of other gene therapies for spinal muscular atrophy or similar conditions, Biogen’s (Nasdaq:BIIB) Spinraza (nusinersen) is an FDA-approved treatment for SMA. Although not a gene therapy, it is an antisense oligonucleotide that helps improve motor function in SMA patients through a series of regular spinal injections. Another promising gene therapy is SRK-015 by Scholar Rock (Nasdaq:SRRK), currently in clinical development to treat SMA patients by promoting muscle growth. For other similar conditions, such as Duchenne muscular dystrophy (DMD), several gene therapies are in development, like Sarepta Therapeutics’ (Nasdaq:SRPT) SRP-9001, which is currently in clinical trials. Solid Biosciences (Nasdaq:SLDB) is also developing SGT-001, a gene therapy for DMD patients that aims to restore dystrophin expression and improve muscle function. — Drug Discovery and Development, 1d ago

new Collins and team used the RNA editing ability of ADARs to create the new sensor and programmable therapy. Each single-stranded “circuit” contains the RNA sequence needed, a “stop” codon sequence of uracil-adenosine-guanine (UAG) in the middle of the RNA strand and a sequence coding for a fluorescent green protein. They also added the ADAR sequence to a later model to allow it to work in more tissues, as ADARs are not naturally found at high levels in all cells. — Inside Precision Medicine, 1d ago

new Now, researchers have successfully restored the vision of mice with retinitis pigmentosa using a genome editing technique they developed (referred to as PE... — GEN - Genetic Engineering and Biotechnology News, 1d ago

new ..."Among the most promising translational strategies of gene therapy for neurodegenerative diseases, ASOs have emerged as a viable, life-extending therapeutic approach by correcting fatal gene expression or RNA processing defects. This work demonstrates the in vitro and in vivo efficacy of ASOs in preventing missplicing of stathmin-2 in TDP-43– deficient neurons," says Lagier-Tourenne. — phys.org, 1d ago

new TAK-279 came from the labs of Nimbus, a biotech company that places computational methods at the heart of its drug discovery approach. Cambridge, Massachusetts-based Nimbus designed the molecule, which it called NDI-034858, to selectively bind to one particular domain of TYK2 without hitting closely related JAK proteins. — MedCity News, 1d ago

new Led by Professor Kai Yao, the team attempted to rescue the vision of mice with RP caused by mutations in the gene encoding a critical enzyme – called PDE6β – by engineering a new use for the CRISPR genome editing tool. — Good News Network, 2d ago

new ThermoGenesis Holdings, Inc. develops, commercializes, and markets a range of automated technologies for CAR-T and other cell-based therapies. The Company currently markets a full suite of solutions for automated clinical biobanking, point-of-care applications, and automation for immuno-oncology, including its semi-automated, functionally closed CAR-TXpress™ platform, which streamlines the manufacturing process for the emerging CAR-T immunotherapy market. For more information about ThermoGenesis, please visit:... — prnewswire.com, 1d ago

new Absci Corp. is the drug and target discovery company harnessing deep learning AI and synthetic biology to expand the therapeutic potential of proteins. It partners with pharma and biotech innovators to create the next generation of protein-based drugs. The company was founded by Sean McClain in 2011 and is headquartered in Vancouver, WA. — markets.businessinsider.com, 1d ago

new Depending on drug class, the leukemia therapeutics market analysis is segmented into chemotherapy and targeted therapy & immunotherapy. The targeted therapy & immunotherapy segment accounted the largest share in 2021. This is attributed to the identification of molecular targets of cancer cells, fewer adverse effects, and the availability of a number of targeted therapeutics. In addition, recent advances in cellular technology have contributed to improving the cells and their metabolism at a molecular level, thus driving the need for targeted drug therapies for leukemia cancer treatment. — alliedmarketresearch.com, 1d ago

new AGT is also conducting preclinical work on a drug to treat the rare, inherited monogenic disease phenylketonuria, as well as an immuno-oncology program, dubbed ImmunoTox, to deliver therapy to solid tumors using lentiviral vectors. Down the line, Galvin sees potential for treating a range of monogenic diseases. — PharmaVoice, 2d ago

new Oxford Nanopore and Cyclomics entered a multi-year research collaboration and licensing agreement to fine-tune the method with the goal of enabling robust detection of rare mutations that are present at below 0.5% in cfDNA. Further optimizations, including the detection of methylation—which provides critical information on cancer and can be elucidated in real time using nanopore sequencing—are now underway. — Inside Precision Medicine, 1d ago

new Kangpu Biopharmaceuticals, Ltd. is a clinical-stage company focused on the discovery and development of innovative small molecules for the treatment of solid tumors, hematologic malignancies, autoimmune diseases, and inflammatory disorders through novel solutions, including targeted protein ubiquitination and degradation. Kangpu has developed a robust pipeline of potential first-in-class and best-in-class drug candidates based on proprietary technology platforms, including NeoMIDES... — prnewswire.com, 2d ago

new BioVaxys Technology, a clinical stage biotechnology company that is developing viral and oncology vaccine platforms,... — VatorNews, 1d ago

new While cell and gene therapy, today, focuses primarily on rare diseases, emerging therapies are targeting larger patient populations and more chronic conditions, which means the benefits could multiply. As that happens, viral vector manufacturing — a critical component in gene therapy, which relies on modified viruses to efficiently introduce specific DNA sequences into cells — is in demand. And MilliporeSigma, which has been manufacturing viral vectors for more than 25 years, including those found in four commercially available viral vector-based gene therapies on the market, has seen an increase in inquiries from biopharma innovators interested in a CDMO partner that can support them from development through commercialization, to bring new treatments to market. — BioPharma Dive, 2d ago

new Novel UNCD™-based scaffolds provide order of magnitude better surfaces for growing pluripotent stem cells and inducing electric-field-based diferentiation into other human cells for developmental biology and biological treatment of human medical conditions. — Open Access Government, 1d ago

new Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing, and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology. — prnewswire.com, 2d ago

new PharmaCyte Biotech, Inc. a global biotechnology company focused on developing cellular therapies for cancer and diabetes using its signature live-cell encapsulation technology. — WriteUpCafe.com, 1d ago