Adverum Biotechnologies Inc. CEO Laurent Fischer said the firm chose the more prudent route in scrapping development of gene therapy ADVM-022 (AAV.7m8-aflibercept) for diabetic macular edema (DME) as a result of dose-limiting toxicity in the phase II Infinity trial.
NHS England has announced a new £340 million (US$481 million) Innovative Medicines Fund, as the country grapples with the issue of bringing ultra-pricey specialist medicines to patients on the country’s taxpayer-funded health care system.
Cell and gene therapy have seen much progress in recent times, with the product pipelines in those areas bursting with more than 1,200 therapies. Naturally, the challenges, opportunities and essential development strategies in those fields were the focus on the last day of the BIO Asia-Taiwan Conference 2021.
DUBLIN – Coave Therapeutics unveiled a new identity and a new gene therapy platform, as it closed a €21.2 million (US$25 million) extension to its long-running series B round, which takes the total raise to €33 million.
Private biopharma investment in 2021 continues to outpace that of the past two years as two gene therapy companies, Shape Therapeutics Inc. and Kriya Therapeutics Inc., posted series B financings this week totaling more than $200 million.
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.
Biogen Inc. has announced contrasting results from phase III trials of therapies for a rare eye disease and depression, following last week’s controversial FDA approval of Alzheimer’s drug Aduhelm. The Cambridge, Mass.-based firm said a phase III gene therapy study in the rare retinal disease choroideremia missed its primary and secondary endpoints, although the news was better from a potential therapy for major depressive disorder.
LONDON – The pioneers of gene therapy in Europe now aim to take the field to the next level, moving into the development of virally delivered antibodies in the treatment of central nervous system and muscular diseases, with Alzheimer’s disease one of the first targets.
The FDA has lifted clinical holds on four studies from Bluebird Bio Inc., following recent similar actions with other gene therapy programs. Two of the studies concern phase I/II and phase III clinical trials of the gene therapy Lentiglobin (BB-1111) in treating sickle cell disease. The remaining two studies are phase III clinical trials of betibeglogene autotemcel gene therapy, which share a vector with Lentiglobin, for treating transfusion-dependent beta-thalassemia.
China is making strides in cell and gene therapy, notably so with a 61% surge in the number of clinical trials in six years, a new report by Ernst & Young showed. While analysts noted the increasing innovation efforts and cross-border collaborations, concerns remain if quality will be compromised by speed.
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