Gene therapy
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BioMarin executives sought to calm an anxious investor base Wednesday with a public address and pledge to achieve a nearly 50% bump in annual revenue by 2027. But analysts were left wanting.
Eli Lilly offers weight loss drug Zepbound directly to consumers while Novo Nordisk continues to struggle with supply challenges for its own GLP-1s. Meanwhile, gene therapies for retinal diseases target competitive market, and layoffs persist.
The intellectual property landscape for newer gene-editing technologies, like that for CRISPR-Cas9, remains unclear and hard to navigate.
With promising early results, cell and gene therapies are making headway against both rare and common ocular and auditory diseases.
With gene therapies by REGENXBIO and AbbVie, Adverum and others in mid- or late-stage trials, this therapeutic class could soon be an option for this common cause of blindness in the elderly.
Quotient Therapeutics’ platform targets somatic mutations, which the startup contends can help identify a broader scope of genes potentially associated with disease phenotypes. Wednesday’s agreement is part of an existing partnership between Pfizer and Flagship Pioneering.
As the biotech implements a more focused strategy for its Roctavian hemophilia A gene therapy, BioMarin has recruited two seasoned pharma executives to bolster its C-suite.
With the help of third-party investors, the new venture will focus on three genetic and rare diseases: tuberous sclerosis complex, erythropoietic protoporphyria and alpha-A1 antitrypsin deficiency.
The recent invalidation of an AAV gene therapy patent overlooks the complexity of innovation in biotechnology and could put a broad swath of intellectual property at risk.
Regeneron, Akouos and Mass Eye and Ear are testing therapies that can reverse genetic protein deficiency to restore hearing, with promising early results.
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