Verve hit by FDA’s gene editing caution

When Beam was slapped with a clinical hold for its base-edited Car-T project BEAM-201, some harboured hopes that Verve’s VERVE-101, which uses the same technology, would escape this fate. For one, BEAM-201 involves multiple edits, while VERVE-101 makes a single base change. However, investors who kept the faith were punished today by an FDA hold on VERVE-101’s IND application; the move raises the question of whether the regulator is cautious about base editing in particular, or in vivo gene editing in general. Without knowing details of the agency’s concerns, it is hard to say. Some groups have managed to get in vivo projects into US trials, including Excision Biotherapeutics, whose EBT-101 uses Crispr to make multiple cuts in the HIV genome. Intellia, however, which reported more data over the weekend on its amyloidosis contender NTLA-2001, has so far only trialled its in vivo assets outside the US. One mark against Verve is that various, more conventional anti-PCSK9 therapies are available, and the FDA has recommended that gene editing therapies be reserved for patients with no other options. The Heart-1 study of VERVE-101 continues outside the US, but Verve’s stock sank 19% this morning.

To read Evaluate Vantage’s report into next-generation genetic medicines, including more information on Verve and Excision, click here.

Status of selected gene edited projects
Project Company Description Status
VERVE-101 Verve Therapeutics In vivo base editing project targeting PCSK9 for high cholesterol Ph1 Heart-1 enrolling in NZ & UK; US IND on clinical hold
BEAM-201 Beam Therapeutics Ex vivo base edited Car-T for leukaemia/ lymphoma US IND on clinical hold; Beam has submitted response to FDA
NTLA-2001 Intellia Therapeutics/Regeneron In vivo Crispr editing project targeting TTR for amyloidosis Ph1 enrolling in NZ, Sweden, UK; data reported
NTLA-2002 Intellia Therapeutics In vivo Crispr editing project targeting KLKB1 for HAE Ph1/2 enrolling in Netherlands, NZ, UK; data reported
EDIT-101 Editas Medicine In vivo Crispr editing project targeting CEP290 for LCA10 IND cleared 2018; in ph1/2 Brilliance; disappointing data reported
EBT-101 Excision Biotherapeutics In vivo Crispr therapy for HIV IND cleared 2021; ph1 (NCT05144386) recruiting
Source: Evaluate Pharma &

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