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Why Sarepta’s most recent failure in DMD was entirely predictable

DrugBaron

But before doing so, its worth reading a few paragraphs from a Commentary on gene therapy that DrugBaron wrote in 2001, marking the first publication of the completed human genome sequence, just to remind us that the central flaw in the Sarepta story was well-known two decades ago.

DNA 100
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Medicinal Chemistry In The Age Of Artificial Intelligence

LifeSciVC

In 2001, I was at Millennium Pharmaceuticals, one of the major players in the nascent field of genomics drug discovery, and that time has strong parallels to the changing state of drug development today. In fact, the Millennium of 2001 (pre-Takeda acquisition) had one drug approved which was Velcade (bortezomib).

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Rapid delivery of toxicological material

Drug Target Review

To achieve an aggressive timeline from DNA to investigational new drug (IND) application, drug developers have strengthened collaborations with contract development & manufacturing organisations (CDMOs) to expedite drug development.

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Maui Fires: How Mitochondrial DNA Will Identify Human Remains

PLOS: DNA Science

The Maui firestorm was so vast and fast that most identification of human remains will come from bits of persisting DNA from mitochondria. MtDNA in Forensics and Genealogy Mitochondrial DNA (mtDNA) is used in forensics to match the tiniest bits of human remains to families. Mitochondrial DNA is also far more abundant than nuclear DNA.

DNA 97
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Fast Biology

Codon

Thanks to decades of progress in molecular sequencing technologies, it is simple to read out the order of nucleotides in a DNA sequence, for example, or to quantify messenger RNAs as they are made by a cell. The ATP synthase “bead” experiment was part of a 2001 study published in the journal Nature.

RNA 98
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Intellia Aims Big with CRISPR Therapy to Cure ATTR

The Pharma Data

Intellia has received approval to launch their Phase I trial of NTLA-2001 in hereditary ATTR. Their NTLA-2001 candidate uses their proprietary non-viral lipid nanoparticle platform, which will deliver this CRISPR/Cas9-based therapy. hATTR is an inherited DNA mutation of the TTR gene.

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Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy

PLOS: DNA Science

The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. The post Older Siblings Made Possible Just-Approved Gene Therapy for Metachromatic Leukodystrophy appeared first on DNA Science. That would enable identification of children before symptoms begin.