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Gene Editing
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Gene Editing is the ability to make highly specific changes in the DNA sequence of a living organism, essentially customizing its genetic makeup. It demonstrates great potential in gene research, gene therapy and genetic improvement. The Gene Editing concept includes major US-listed companies whose businesses are involved in gene editing.
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Should be interesting to see what we get in up front. It's been given Accelerated Approval by FDA so BLA will be filed next year sometime once we get final patient data. I'm guessing 100 to 200 million up front.
nfa
SiteOne’s efforts in non-opioid pain management put it in a unique position relative to Vertex Pharmaceuticals, a major player in the field known for its successful development of cystic fibrosis treatments. Vertex has also been working on non-opioid pain drugs, specifically targeting the NaV1.8 sodium channel, a focal point shared by SiteOne’s lead pro...
The STAC-BBB capsid technology addresses one of gene therapy's most challenging obstacles - crossing the blood-brain barrier efficiently. Traditional AAV vectors struggle with CNS penetration, often requiring invasive direct administration. An effective IV-delivered solution could revolutionize neurological gene therapy by enabling broader distribution and simplified administration.
The non-human primate data showing potent BBB ...
The $20 million upfront payment and potential $1.3 billion in milestone payments represent a significant cash infusion for Sangamo, whose current market cap is around $486 million. The deal structure, with tiered mid-to-high single-digit royalties, follows industry standards for early-stage biotech licensing agreements. The immediate cash boost strengthens Sangamo's balance sheet and extends their operational runway.
The agreement's va...
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