FirstWord Lists: Bristol-Myers Squibb joins a growing list of gene therapy converts – who else has made the plunge?

Gene therapy’s latest big pharma convert is Bristol-Myers Squibb, which pledged $100 million in near-term payments this week to throw its lot in with uniQure’s efforts in the cardiovascular arena, adding the company’s name to a growing list of peers to have recently made the plunge back into the erstwhile unloved therapeutic modality.

News of the deal sent shares of uniQure skyrocketing up 47 percent on April 6, boosting the company’s market cap to an all-time high of $410 million.

It was not that long ago that news of any deal – let alone one with a nine-figure sum in guaranteed money attached – between a gene therapy company and an established pharma player would have been all but unthinkable. Much has changed over the past couple of years as the field has made a number of important milestones and breakthroughs, including the first-ever commercial approval in the US or Europe thanks to uniQure, which received the go-ahead from the EMA to market Glybera in 2012.

Indeed, it is a testament to the evolution of the gene therapy field, thanks primarily to improvements in the deliverability and an increased appreciation of long-term safety effects, that investors as well as large biotech and pharma partners have been convinced to get involved again after very little success was realised in the modality’s initial heyday in the 1990s.

Specifically, Bristol-Myers Squibb has agreed to pay $50 million in upfront money and a $32-million equity investment (for a 5-percent stake in uniQure), plus three $15 million payments for the selection of additional collaboration targets. The primary focus of the partnership will initially be S100A1, a gene therapy agent for chronic heart failure that uniQure acquired as part of its €3-million takeover of InoCard last fall. The programme is currently in preclinical development and scheduled to enter human testing in 2016.

uniQure is eligible for up to $254 million in R&D and regulatory milestone payments attached to S100A1, and up to $217 million for each other gene therapy programme. The company is also eligible for sales-based milestones and tiered single to double-digit royalties.

All told, the deal could be worth as much as $1 billion (or more) to uniQure, representing a watershed moment for the company – and makes it one of a handful of gene therapy companies to have attracted the attention of significant partners over the last few years. A list of some of the other partnerships in the space include:

Among the other notable companies in the large biotech and pharma space with in-house efforts in the gene therapy space are Baxter, GlaxoSmithKline and Novartis, a reflection of just how far the field has come in a few short years.

See also: Physician Views Poll Results: How comfortable are doctors with the commercial use of gene therapy?

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