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Making for another landmark cell and genetherapy approval this month, the US Food and Drug Administration (FDA) has signed off on CellTrans’ Lantidra (donislecel), the first cell therapy for type 1 diabetes. Lantidra is thought to increase insulin secretion through the infusion of allogeneic insulin-producing islet beta cells.
Under the terms of the deal, the company will receive non-exclusive rights to CRISPR/Cas9, a gene-editing technology of CRISPR Therapeutics, for the development of potentially curative T1D cell therapies. The gene-editing technology allows for precise, directed changes to genomic DNA.
VX-880 is an investigational allogeneic stem cell-derived, fully differentiated, insulin-producing islet cell therapy of Vertex for T1D. Cell & GeneTherapy coverage on Pharmaceutical Technology is supported by Cytiva. Through the acquisition, Vertex plans to advance its potentially curative VX-880 programmes in T1D.
Bluebird also recently said it would charge $3 million for Skysona, the genetherapy it just launched for a rare brain-wasting disease. Even a drug-price watchdog, the Institute for Clinical and Economic Review, deemed the $2.8 Continue to STAT+ to read the full story…
The next wave of medicine is well on course to be cell and gene-based. In September 2021, GlobalData figures revealed there to be 1,320 industry-sponsored regenerative medicine and advanced therapy trials ongoing worldwide. Meanwhile, the pharma industry is undergoing somewhat of a transformation itself.
Credit: ETH Zurich Many modern fitness trackers and smartwatches feature integrated LEDs. The green light emitted, whether continuous or pulsed, penetrates the skin and can be used to measure the wearer’s heart rate during physical activity or while at rest. These watches have become extremely popular.
The body’s immune system falsely attacks insulin-making beta cells in the pancreas in Type 1 diabetes. OCA-B, or B cell-specific Oct1/2 coactivator, binds to and controls about 150 genes involved in a process through which T cells are reactivated upon reencountering antigens they have earlier identified and memorised.
publishers New Rochelle, NY, January 26, 2021–Hybrid closed-loop insulintherapy improved glycemic control in adolescents and young adults with type 1 diabetes. Credit: Mary Ann Liebert, Inc.,
Vertex Pharma has ramped up its involvement in gene-editing medicines for the third time in a matter of months, agreeing a partnership with CRISPR specialist Arbor Biotechnologies that could be worth up to $1.2 If approved, it could mount a challenge to bluebird bio’s genetherapy Zynteglo, which is already approved in Europe.
Editas Medicine is pausing its ocular genetherapy program after demonstrating a favorable safety profile and seeking a potential partner to develop EDIT-101, the company announced Thursday.
Revita, an outpatient endoscopic procedural therapy, which aims to eliminate insulin needs and enhance glycemic control by ablating dysfunctional duodenal mucosa. In a recent development, Fractyl Health announced the selection of RJVA-001 as its inaugural clinical T2D candidate in the Rejuva genetherapy platform.
Companion diagnostic for Pfizers hemophilia B genetherapy Beqvez (idanacogene elaparvovec-dzkt). The test is designed for identifying individuals with moderate to severe hemophilia B (congenital factor IX [FIX] deficiency) who may benefit from the genetherapy Beqvez.
Hormones (such as insulin, glucagon, and growth hormone, to name a few) are produced by recombinant DNA, as are blood factors, thrombolytic agents, interferons, monoclonal antibodies, therapeutic enzymes, and more.
While insulin price caps will begin this year, larger effects will be delayed until 2026 – when the US federal government will be allowed to negotiate Medicare prices and demand rebates from drug companies that price gouge. It’s much needed. It’s a hot area.”
Results from a phase 1/2 clinical trial revealed that PolTREGâs regulatory T cell (Treg) therapy, PTG-007, was able to restore insulin secretion in children with type-1 diabetes.
FDA rejects BioMarin’s genetherapy and Gilead’s filgotinib over concerns. The FDA refused to approve BioMarin’s hemophilia A genetherapy valoctocogene roxaparvovec. The removal of an AdComm was seen as a positive. Immune-evading cells with PD-L1 control diabetes in mice.
These breakthroughs, combined with the growing focus on genetherapy, oncology and immunology, have brought these companies to the forefront of global healthcare. The recent partnership to produce affordable insulin in Africa aims to reach over four million people by 2026, reflecting Novo Nordisk’s commitment to global health equity.
The one-shot therapy is expected to provide long-lasting effects, reduce the number of bleeds experienced by patients, and reduce the need Factor VIII replacement therapy.
Dr. Toleikis spoke about the latest clinical trial data for the medical device, and also about the company’s new partnership with Evotec to integrate its insulin-producing cells in the Cell Pouch System. Toleikis about Sernova’s proprietary Cell Pouch System that is designed to house therapeutic cells.
Usually, the desired gene, such as human insulingene, when inserted into the plasmid of the host cell uses transcriptional and translational machinery of the host to express itself. It is worth mentioning that in vitro gene expression requires a suitable host for the production of a specific gene product.
Meanwhile, BioMarin’s genetherapy Roctavian is approved by the FDA for the treatment of hemophilia A. It is a dipeptidyl peptidase-4 inhibitor that keeps insulin levels stable and reduces the amount of glucose produced by the body. But all three drugs have new competitors. Both are anti-CD20 agents. billion in 2022.
Diabetes : These devices offer an effective solution for delivering insulin, improving glycemic control, and enhancing the quality of life for diabetic patients. The historical trends of existing delivery devices, such as prefilled syringes, autoinjectors and insulin pumps, suggest a strong demand in the market.
The first was insulin – Frederick Banting and colleagues managed to isolate insulin that could treat diabetes, up until that point a fatal condition. The period between 1918 and 1939 was marked by two breakthroughs that presaged the arrival of the pharma industry as we know it today.
It is no exaggeration to say that the world changed in the early 80s when insulin was expressed synthetically in microorganisms for the first time. This breakthrough led to a recombinant (biosynthetic) and thereby safer therapy for diabetes than the animal-sourced insulin which had been used since 1922.
CSL closes deal for UniQure genetherapy in hopeful sign for M&A ( BioPharmaDive ).
Dyno raises $100 million to bring artificial intelligence to genetherapy ( STAT ).
Lilly inks smart insulin pen deals with Roche, Dexcom and more ( Fierce ).
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