Mustang Bio sets its sights on 2021 approval
Its gene therapy is three years away from market, but Mustang is already thinking about outcomes-based pricing.
AAN 2019 – Roche confirms its place in spinal muscular atrophy
A slew of data on spinal muscular atrophy projects over the weekend shows that this tiny market is set for some big competition, with Novartis, Roche and Biogen jostling…
Novartis and Biogen face spinal muscular atrophy reckoning
The upcoming American Academy of Neurology meeting could provide clues on how the market will shake out.
Mustang and the bubble boy bubble
The value of Mustang Bio has tripled on data on its immune deficiency gene therapy. But investors might be too keen – and also too late.
Big-bucks first quarter masks drop in deal volume
Bristol-Myers Squibb’s $74bn bid for Celgene led to a huge quarter, but a drop in the deal count could give pause to those hoping for a bumper year.
Two haemophilia A subjects help Sangamo erase 2019's losses
Promising early data with Sangamo and Pfizer’s haemophilia A gene therapy could set up a battle with Roche and Biomarin.
Should gene therapy patients be asked to pay?
The price in value-based payment deals should by definition be justified, so why is patient cost-sharing part of the calculation? Spark covers this cost, but there is no…
Fibrocell gets a reality check
Promising data with Fibrocell’s epidermolysis bullosa gene therapy send the company down as funding worries kick in.
How to pay for gene therapies in developing nations
With rich countries like the US finding it hard to fund gene therapies, it is worth asking whether these projects will ever reach patients in developing countries. And if…
Kickstarting ideas
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