Video Interview: Abeona Breaking Into Rare Diseases
This article was originally published in Scrip
Three-year-old Abeona Therapeutics is poised to enter the clinic with its lead gene therapy programs targeting some devastating lysosomal diseases.
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Companies doing groundbreaking work in cell and gene therapies should look to how rare disease drug developers partnered with patient advocacy groups and disease foundations to advance their shared goals, suggested speakers at the Cell & Gene Exchange conference.
The WORLD Symposium Feb. 13 to 16 in San Diego offered everything from basic research through late-stage clinical data in lysosomal diseases, including ArmaGen's blood-brain barrier-crossing treatment for Hurler syndrome and updates from ongoing development programs from rare disease specialists, such as Genzyme, Amicus and Protalix.