While share prices of most small-to-medium biotechs have been taking a hammering in 2022 so far, gene therapy companies have been particularly hard hit.
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Michael Parini talks to Scrip about how a new focus at the company can help it prove the value of its AAV-based gene therapy platform.

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Public Company Edition: Stock valuations are falling due to political, economic and regulatory uncertainty, resulting in fewer large public offerings, more alternative financings and cost cuts. Carisma, Tenaya, BioAtla, Arbutus, Nkarta, Alector and Adaptimmune announced layoffs.
The US FDA approved anti-CD19 antibody Uplizna, from Amgen’s $27.8bn purchase of Horizon in 2023, for IgG4-related disease – a larger market than its original NMOSD indication.
BeiGene’s Phase III ociperlimab joins the list of failed TIGIT inhibitors, as candidates from Roche, Merck & Co. and others have failed late-stage studies.
It might be the beginning of the end for the orphan drugs party but there is still sales growth enjoyment to be had for the sector, whose star performers are now looking increasingly like mainstream drugs.
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BeiGene’s Phase III ociperlimab joins the list of failed TIGIT inhibitors, as candidates from Roche, Merck & Co. and others have failed late-stage studies.
The approval of another Novartis drug with a different mechanism means the company is further cornering multiple parts of IgAN pathogenesis.
It might be the beginning of the end for the orphan drugs party but there is still sales growth enjoyment to be had for the sector, whose star performers are now looking increasingly like mainstream drugs.