Israeli company BioBlast targets orphan diseases that traditional pharmaceutical companies won’t touch.
By Abigail Klein Leichman for Israel21c
Treatments for extremely rare medical conditions are few and far between. The number of cases of “orphan diseases” doesn’t justify the amount of cash needed to get a pharmaceutical developed, tested and approved.
This is exactly the niche that Tel Aviv-based BioBlast Pharma was created to fill in 2012. Now its three experimental platforms are moving closer to market.
Cabaletta, BioBlast’s lead product for treating two rare and currently untreatable conditions — oculopharyngeal muscular dystrophy (OPMD) and spinocerebellar ataxia type 3 (SCA3) — received Fast Track approval in June from the US Food and Drug Administration (FDA) to expedite the drug’s development, review and potential approval specifically for treating OPMD.
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