Top Tweets for #IsaVec
Alongside other established companies in the field of rare diseases, Astellas appears to be a potentially suitable partner for #Isavec in the Asian markets.
$SGMO
There's another meeting in Taiwan this week—Taiwan has one of the highest incidences of Fabry disease in the world. I don't understand why no geographic licenses for #Isavec are being granted.
$SGMO
The next FIN #Fabry Expert meeting will take place from April 24th to 25th, 2026 in Taiwan.
Taiwan has an unusually high reported incidence of Fabry disease. In particular, the IVS4+919G>A (GLA gene) mutation is very common in Taiwan. A Taiwanese licensee for $SGMO s ST-920 would certainly make sense.
@yusufhameed @kaysaaa81 @BiotechAnalysst @sandymacrae23 $SGMO will stay afloat with ATM until the BLA from #Isavec/ST-920 is submitted.
#Fabry $SGMO #ST920 #Isavec #STAAR
An interim analysis (data cut-off: September 12, 2024) showed a positive average annual eGFR increase of +3.061 mL/min/1.73 m² per year in a cohort of 23 patients who were followed for at least 12 months. This indicates a significant improvement in kidney function. An earlier interim analysis (cut-off: September 19, 2023) showed nearly stable kidney function in 13 patients with an eGFR increase of -0.915, indicating a slowing of the typical functional decline in FD. The improvement in the average eGFR from 13 to 23 patients who were followed up for AT LEAST 12 months suggests that the dramatic improvement in kidney function only becomes apparent over time (>12 months). If 10 additional study participants are included in the next analysis, then, depending on their study duration, a lower average eGFR value than in the interim analysis is possible, especially if the eGFR values are consolidated after exactly 12 months. It is important that the eGFR values remain positive over time, thus fulfilling the requirements for accelerated approval.
cut-off: September 19, 2023:
https://t.co/fQMfiUTscj
cut-off: September 12, 2024
https://t.co/ro5vYaP2F6
All #Fabry disease patients given ST-920 in trial have reached the one-year mark needed for the next step in getting #accelerated #FDA #approval.
$SGMO @SangamoTx #isaralgagene #civaparvovec #isavec
https://t.co/k1NiM707Vr
@yusufhameed Regarding $SGMO / #ST920 I will say it again: #Isavec will win prizes, such as the Prix Galien. This is a prestigious international award for outstanding innovations in pharmaceutical research and medicine. Mark my words!
I stick to the facts:
There is a life-shortening (approx. 20y) indication (FD), in which the previous medications (ERT) only slow down the deterioration (kidney and heart function). Now there is a $SGMO gene therapy (#Isavec/#ST920), of which the interim data has convinced the drug regulatory authorities (FDA and EMA; several FDA members attended a EMA meeting!) to such an extent that a phase 1/2 study (STAAR) is being approved as a registration study and a phase 3 study is not required for approval. A potential licensee thus has no development costs for a ready-to-use gene therapy (usually up to $1b) that also has hardly any side effects. Every pharmaceutical company doing gene therapy research is a potential takeover candidate, because it is hard to imagine a better therapy outcome. For Fabry patients, there are many advantages (single application, improvement of kidney and heart function), but also from an overall economic point of view: an economic benefit is very likely despite the expected high price of the gene therapy (> $1m). Isavec comes very close to a cure!
@SchoolyardPS7 Why $SGMO should secured a $100M term loan when it's #Fabry #Gene #Therapy will generate within the next 7 weeks upfront payment much over $200M ?
#isaralgagene #civaparvovec #IsaVec
https://t.co/E9rPfFMwzh
Updated Data Suggests @SangamoTx Gene Therapy Improves Renal Function in Fabry Disease
With these results, it feels like commercial success based on potential Partnership/Deal is right around the corner.
$SGMO #Fabry #Isaralgagene #civaparvovec #IsaVec
https://t.co/HVCC9YGjI1
Updated Data Suggests @SangamoTx Gene Therapy Improves Renal Function in Fabry Disease
With these results, it feels like commercial success based on potential Partnership/Deal is right around the corner.
$SGMO #Fabry #Isaralgagene #civaparvovec #IsaVec
https://t.co/HVCC9YGjI1
$SGMO - that is incredible!
"...typical untreated adults with classic fabry experience an annual eGFR decline of 5 mL/min/1.73m2/year. ERT treated patients experience a decline around 1. Even normal healthy adults experience a decline close to 1. But patients treated with Isa-vec experience an average INCREASE of 3. That is incredible."
@Chipoo86 Sanofi mishandles Sangamo once with Sickle cell , i dont want stinkin sanofi as a partner , i want to see their fabrazyme sales decline hard once $SGMO #ISAVEC on market , they dont deserv us as a partner ... i would only sell them the Tregs business in france
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