As Medicine Man has correctly pointed out, Sarepta’s clinical trial failure (no statistically significant improvement in muscle function was seen after 48 weeks) was in SRP-9001 in DMD.
SRP-9001 has no connection to Sue Fletcher. It is a once-off experimental AAV-vector delivered gene therapy, rather than a PMO therapy.
As SoT has correctly pointed out, Sarepta’s Amondys 45 (SRP-4045) has just been approved by the FDA for DMD patients amenable to exon-45 skipping. It is a PMO therapy and Sue Fletcher was part of the team instrumental in the drug’s design.
Although the benefits of Amondys 45 may be seen as modest, it’s important to note that this drug does not have the benefit of cell-penetrating peptide delivery.
PYC Price at posting:
14.5¢ Sentiment: Hold Disclosure: Held
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