Top Tweets for #Isaralgagene
A BLA (eg for #Isaralgagene civaparvovec prepared by $SGMO ) consists of several key components, and many underestimate the Chemistry, Manufacturing, and Controls (CMC) section in particular. While it is often assumed that successful clinical trials alone constitute the decisive step toward approval, they are in reality only a prerequisite for approval by the FDA.
The CMC section describes in detail how a biologic drug is manufactured, controlled, and produced with consistent quality. This includes, among other things, the manufacturing process, analytical methods, quality controls, stability data, and the validation of production steps. This area is particularly complex for biologics because even small changes in the manufacturing process can affect product properties.
For smaller biotech companies like Sangamo, CMC therefore often poses a major challenge. Production and analytics are frequently outsourced to external partners such as CMOs (in Isavec’s case, Brammer Bio, a subsidiary of $TMO Thermo Fisher Scientific), which requires additional coordination and extensive documentation. As a result, delays in regulatory approval processes often arise in the CMC area, even though the clinical data is already compelling.
Over the next three months, $SGMO is likely to focus primarily on its #Fabry program with ST-920 (#isaralgagene civaparvovec). Updates are expected on the ongoing approval process with the U.S. FDA, such as formal feedback or progress in the review process.
In addition, clinical progress in the ST-503 pain program could be reported, particularly regarding the ongoing Phase 1/2 study (e.g., recruitment or initial safety data).
The focus is therefore clearly on regulatory milestones and clinical updates.
There is no official list price for $SGMO's #isaralgagene civaparvovec (ST-920) in the US yet, as the therapy is currently still undergoing the approval process. However, based on comparable one-time gene therapies for rare diseases, the price per patient is expected to be in the high six to seven-figure range, i.e., approximately $1–2 million. The manufacturing costs for AAV-based gene therapies such as ST-920 are significantly lower and are estimated in the industry to be around $50,000–150,000 per dose, depending on factors such as vector production, purity requirements, quality assurance, and clinical preparation.
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
@mchilberg Is there a requirement to publish the data from the $SGMO #Pivotal #Fabry #GT trial (#Isaralgagene #Civaparvovec)?
When do you expect the data to be released?
Also, is there a regulatory deadline by which the results must be disclosed?
$SGMO `s ST-920 resp. #Isaralgagene Civaparvovec is an experimental gene therapy for Fabry disease that delivers a functional copy of the GLA gene into liver cells, allowing for continuous production of α-galactosidase A (α-Gal A). This approach aims to provide a stable, long-term treatment, eliminating the need for repeated enzyme replacement therapy (ERT) infusions. In clinical trials, ST-920 has demonstrated sustained normal α-Gal A enzyme levels, indicating effective and lasting enzyme production. Additionally, gene therapy may enable a more uniform distribution of the enzyme throughout the body, leading to a more effective reduction of Gb3 deposits in various tissues, including kidney podocytes, where ERT has shown less effective Gb3 reduction.
https://t.co/eShAEHlk7l
@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."
@pick1998_2 @mchilberg Not only safer - @SangamoTx $SGMO #fabry #genetherapy improves renal function.. an effect you do not see in #ERT.
#isaralgagene #civaparvovec
$SGMO in one week:
#Peter #Marks Keynote Address: "Advancing the Frontier of Gene Therapy" #Isaralgagene #civaparvovec gene therapy in adults w #Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)
good chance of deal by / then / around
2 weeks: $SGMO February 6 #Peter #Marks Keynote Address: "Advancing the Frontier of Gene Therapy" #Isaralgagene #civaparvovec gene therapy in adults w Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)
only 3 weeks to go:
$SGMO says expects deal in Q1 per Jefferies:
$SGMO February 6 #Peter #Marks Keynote Address: "Advancing the Frontier of Gene Therapy" #Isaralgagene #civaparvovec gene therapy in adults w Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)
@SchoolyardPS7 @digdeeper1976 @mr_cleanstl @andrewpaleogos @GamalielBaer @Sh24363159 correct - but the Fabry Meeting first week in February might even be a better place for $SGMO #Fabry updates and announcements!
#Isaralgagene #civaparvovec #genetherapy
https://t.co/amdFPiqHth
$SGMO says expects deal in Q1 per Jefferies:
could come early Feb.
$SGMO February 6 #Peter #Marks Keynote Address: "Advancing the Frontier of Gene Therapy" #Isaralgagene #civaparvovec gene therapy in adults w Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)
$SGMO says expects deal in Q1 per Jefferies:
could come early Feb.
$SGMO February 6 #Peter #Marks Keynote Address: "Advancing the Frontier of Gene Therapy" #Isaralgagene #civaparvovec gene therapy in adults w Fabry disease: Updated results from an ongoing phase 1/2 study (STAAR)
@yusufhameed @GamalielBaer @BiotechAnalysst In case of an Conditional Marketing Authorisation by the European Agencies for $SGMO #Fabry #Genetherapy (#isaralgagene #civaparvovec)
$10 per share would be more reasonable
https://t.co/CjwkF30iXz
criteria for Conditional Marketing Authorisation:
$SGMO
https://t.co/svq91dx2aZ
$SGMO is the leading patent holder for zinc-finger nucleases. By using its proprietary zinc finger platform, the company is engaged in the development of various pipeline candidates. The company's lead asset involving this technology is #isaralgagene #civaparvovec (ST 920) #Fabry
@Franca_ole #Fabry Market size is projected to value USD 2.4 billion in 2024 and reach USD 4.9 billion by 2033, growing at a CAGR of 8.3%. #ZiFi (Zinc fingers) based #Genetherapy ( #isaralgagene #civaparvovec [ST-920]) with fast track designation from @SangamoTx https://t.co/5URTuvlJSo
![ZinselmeyerB's tweet photo. @Franca_ole #Fabry Market size is projected to value USD 2.4 billion in 2024 and reach USD 4.9 billion by 2033, growing at a CAGR of 8.3%. #ZiFi (Zinc fingers) based #Genetherapy ( #isaralgagene #civaparvovec [ST-920]) with fast track designation from @SangamoTx https://t.co/5URTuvlJSo https://t.co/0c5Z0i8onY](https://pbs.twimg.com/media/GTLW0ezX0AABD_v.png)
@biotech_cash i think #Sanofi needs to #protect their $1B+ ERT business ...
or substitute it a with a one time fix drug AKA
#Isaralgagene #civaparvovec
@BiotechAnalysst $JNJ looking for first and best in class: #Fabry $SGMO #isaralgagene #civaparvovec https://t.co/uKdlsu0uTS
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