While U.S. lawmakers continue their debate on reducing spending for prescription drugs, government payers are exploring innovative reimbursement ideas to cover gene and cell therapies that could cost millions of dollars for a cure or a durable effect against rare diseases.
Collectively, lysosomal storage disorders (LSDs) are caused by malfunctions in metabolic enzymes in the lysosome system. Depending on which enzyme is missing, toxic metabolites accumulate. While the LSDs are highly heterogenous – even within one disease, presentation can vary widely – neurodegeneration is a common feature in these disorders.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: AGTC, Dyne, Genenta, Luye, Metagenomi, Otonomy, Regenxbio, Tessa, Ultragenyx.
Collectively, lysosomal storage disorders (LSDs) are caused by malfunctions in metabolic enzymes in the lysosome system. Depending on which enzyme is missing, toxic metabolites accumulate. While the LSDs are highly heterogenous – even within one disease, presentation can vary widely – neurodegeneration is a common feature in these disorders.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: 4D, Affinia, Arcellx, Generation, Homology, Rocket, Sio Gene, Uniqure.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: Antion, Biomarin, Enochian.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Gene & Cell Therapy annual meeting, including: Adaptimmune, Akouos, Beam, Catamaran, Codiak, Dyno, Freeline, M6P, Neurogene, Passage, Phio, Poseida, Precision, Senti, Verve.
LONDON – A breakthrough technology for generating fully human T-cell receptors (TCR) is set to deliver next-generation T-cell therapies for treating solid tumors, following the €66 million (US$78.3 million) series A funding of T-knife GmbH.
By deleting the gene for uridine monophosphate synthetase (UMPS), an enzyme in the pathway for uridine synthesis, researchers have made cells, including embryonic stem cells and T cells, dependent on dietary uridine. The work, which was published in the July 13, 2020, online issue of Nature Biotechnology, adds a new potential way of controlling cell therapies.
By deleting the gene for uridine monophosphate synthetase (UMPS), an enzyme in the pathway for uridine synthesis, researchers have made cells, including embryonic stem cells and T cells, dependent on dietary uridine. The work, which was published in the July 13, 2020, online issue of Nature Biotechnology, adds a new potential way of controlling cell therapies.
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