Recently, the FDA has requested Sarepta Therapeutics, Inc, Massachusets-based biopharmaceutical company, to suspend all distribution of its DMD gene therapy Elevidys (delandistrogene moxeparvovec), following the deaths of three patients—two non‑ambulatory teenagers and one adult—due …

Introduction If you asked us (and many other industrial consultants) what is one of the most common commercial strategy mistake we see in otherwise well-positioned early stage companies - without a doubt it would …

As of December 2023, 10 FDA-approved gene therapies were available. Based on existing development programs, 30-50 additional gene therapies are expected to be FDA-approved by 2030. The impact on treatment outcomes but also the …

The new managed entry agreement (MEA) and health technology assessment (HTA) regulations adopted in Ukraine over the course of 2020 and 2021 present significant step forward in improving access to treatment in the country. …