Skip Therapeutics and Sheba Medical Center to Collaborate for the Development of RNA-based Treatments for Rare Diseases
Skip Therapeutics ("Skip"), developer of computational platform for design of Antisense Oligonucleotide (ASO) based therapies and Sheba Medical Center ("Sheba"), one of the leading medical centers in the world, announced today that they have entered a collaboration...
Moderna loses a COVID vaccine patent in Europe amid heated clash with BioNTech, Pfizer
With litigation spanning many countries, the COVID-19 vaccine giants remain engaged in a heated patent war. In the latest twist, Pfizer’s Comirnaty partner BioNTech chalked up a win as the European Patent Office (EPO) snatched back one of Moderna’s patents, ruling it...
Flagship Pioneering Launches First UK Biotech with $50M Investment
Investment firm Flagship Pioneering on Tuesday launched its first U.K. biotech. Quotient Therapeutics, a company studying genetic variation on the cellular level, joins 41 other companies in the firm’s ecosystem. After a two-year incubation period in the investment...
FDA Delays Decision on Bristol Myers and 2seventy bio’s CAR-T Therapy for Multiple Myeloma
The FDA has postponed its decision on approving Bristol Myers Squibb and 2seventy bio's CAR-T cell therapy for multiple myeloma in earlier treatment lines. Originally expected by December 16, the decision's delay is due to the FDA's plan to convene a public meeting...
UK Approves World’s First CRISPR Gene Therapy for Sickle Cell Disease
The UK's Medicines and Healthcare Regulatory Agency (MHRA) has granted approval for Casgevy, the world's first gene therapy for sickle cell disease, utilizing the CRISPR gene-editing tool. This landmark decision could bring relief to thousands of individuals in the UK...
RNA Editing Is Having a Moment
RNA could be considered DNA’s younger sibling. From the very beginning, RNA’s milestones came later and attracted less attention than DNA’s milestones. For example, the discovery of DNA’s structure was first published in 1953; the corresponding discovery for RNA’s...
CRISPR Revolution: Unveiling BATF3 as a Key to Enhancing Cancer T-Cell Therapy
Researchers at Duke University have advanced cancer T-cell therapy using CRISPR for high-throughput screening of human immune cells. They discovered BATF3, a master regulator transcription factor gene, which reprograms thousands of genes in T cells, enhancing their...
Enhancing AAV6-Mediated Transgene Delivery into T Cells with Cell-Penetrating Peptides: A New Approach to Efficient Gene Delivery for Cellular Immunotherapy
Over the past decade, CAR-T cell therapy, a form of cancer immunotherapy, has shown significant promise, largely due to advances in gene delivery methods. Adeno-associated virus (AAV), particularly AAV6, is notable for its high affinity to immune cells, but its...
VectorY’s $138M Series A to Advance Vectorized Antibody Programs for Neurodegenerative Diseases
Not all drug targets are created equal. For those that result in a tissue specific gain-of-function, the strategy can be not-so-straightforward. That’s exactly what’s going on in some forms of amyotrophic lateral sclerosis (ALS), such as those with a mutation in the...
Astellas Sees Path Forward for Gene Therapy Despite Four Patient Deaths
Despite four pediatric patient deaths and an ongoing clinical hold by the FDA, new trial data released Wednesday by Astellas suggests its gene replacement therapy AT132 for X-linked myotubular myopathy is still viable as a potential treatment option. A detailed...
Astellas Announces Data from ASPIRO Study in X-linked Myotubular Myopathy Published in The Lancet Neurology
Preliminary data analysis on ventilator dependence and motor improvement are reported alongside overall safety findingsAstellas Pharma US, Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") today announced that The Lancet Neurology published a preliminary...
Kyverna Therapeutics Announces Publication in The Lancet Neurology of Case Report of Patient with Refractory Myasthenia Gravis Treated with Investigational KYV-101
No adverse events related to CAR T-cell therapy 60 days post-infusion in patient suffering from severe, treatment-refractory, generalized myasthenia gravis KYV-101 is a fully human CD19 CAR T-cell therapy designed for use in patients with B cell-driven autoimmune...
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