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We are a leader in the field of gene therapy, seeking to deliver to patients suffering from rare and other devastating diseases single treatments with potentially curative results. We are advancing a focused pipeline of innovative gene therapies, including our clinical candidates for the treatment of Huntington s disease, refractory mesial temporal lobe epilepsy ( MTLE ), Fabry disease, and amy…
The business is unprofitable at the operating level (-1151.38% margin). The thesis depends entirely on whether and when it reaches sustainable profitability.
Revenue declined 40.6% YoY. Margins deteriorated 471.8pp alongside, both lines moving the wrong way.
ROIC dropped from -70.65% to -89.19%, capital efficiency is deteriorating. Negative free cash flow of -$178M. The business is consuming cash, not generating it.
Profitability & Returns
Revenue (TTM)
$18M
▼ -40.6% YoY
Net Income (TTM)
-$209M
▲ +16.9% YoY
Op. Margin
-1059.74%
▼ -471.8pp YoY
ROIC
-72.43%
▼ -18.5pp YoY
Cash Flow & Balance Sheet
FCF (TTM)
-$173M
▲ +4.1% YoY
Op. Cash Flow (TTM)
-$172M
▲ +2.6% YoY
Net Debt
-$523M
Net Cash Position
Cash & Equiv.
$587M
5Y CAGR: -15.6%
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