From natural killer cells to off-the-shelf CAR-Ts, biopharma targets cancer with next-gen cell therapies Sarepta cites 'overburdened' FDA as factor in DMD gene therapy delay FDA gets out its red pen again, rejecting BioMarin's gene therapy valrox amid durability worries Using CRISPR to improve viral vectors for gene therapy Astellas' Audentes reports 3rd death in gene therapy trial In the face of COVID-19, cell and gene therapy space shows ‘remarkable resilience:’ report Functioning liver cells regenerate in pig lymph nodes, and a human trial is coming Bayer backs Triumvira to take CAR alternative into clinic Immune-evading cells with PD-L1 control diabetes in mice Spark Therapeutics nabs CMO from new owner Roche Taysha Gene Therapies guns for $100M IPO as it eyes 2020 clinical trial Featured Story | By Arlene Weintraub Following the success of personalized CAR-T treatments like Gilead's Yescarta and Tecartus for blood cancers, several companies are working on the next generation of cell therapies to treat a range of tumor types. They include off-the-shelf CAR-Ts and therapies that boost the innate ability of natural killer (NK) cells to destroy cancer. read more |
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Top Stories By Nick Paul Taylor Burdens placed on the FDA by COVID-19 and the rapid proliferation of cell and gene therapies are a factor in the recent setback to Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) prospect, according to CEO Douglas Ingram. read more By Ben Adams BioMarin’s shares plummeted in early Wednesday trading after the FDA refused to approve its hemophilia A gene therapy valoctocogene roxaparvovec. read more By Angus Liu To overcome preexisting immunity that cripples the adenovirus vectors used in gene therapy and vaccines, scientists at the University of Pittsburgh created a CRISPR-based system that briefly suppresses the genes that cause the problem. The method improved gene therapy uptake in mice and is being developed by startup SafeGen. read more By Nick Paul Taylor Audentes Therapeutics has reported a third death in a clinical trial of its gene therapy against a rare genetic neuromuscular disorder. The Astellas Pharma subsidiary said it “remains committed” to the program despite the rising death rate in the higher-dose cohort of the phase 1/2 clinical trial. read more By Amirah Al Idrus In the early days of COVID-19, the Alliance for Regenerative Medicine was unsure how the pandemic and its accompanying economic downturn would affect the cell and gene therapy space. Now, the numbers are in—and they’re better than ever. In the first half of 2020, the sector raised $10.7 billion, more than the total it raised in 2019. read more By Arlene Weintraub Pitt researchers demonstrated that pigs can grow functioning auxiliary livers in their abdominal lymph nodes after their own hepatocytes are isolated and injected into them. Startup LyGenesis is working to bring the method into human clinical trials later this year. read more By Nick Paul Taylor Bayer has co-led a $55 million investment in cell therapy startup Triumvira Immunologics. Triumvira will use the series A funding to run clinical trials of cell therapies that are directed by an alternative to CARs. read more By Angus Liu Scientists at the Salk Institute created functional, three-dimensional human islet-like organoids that restored glucose control in mice without triggering immune rejection. The trick was to induce the expression of the PD-L1 immune checkpoint protein. read more By Ben Adams After its recent $4.8 billion buyout of gene therapy specialist Spark Therapeutics, Roche is now handing over one of its execs to help lead its new acquisition’s R&D. read more By Amirah Al Idrus Taysha Gene Therapies launched just four months ago, but it plans to ride the biotech wave to a $100 million IPO. 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