Good news for patients with a Stop-Mutation

[Guest guest]

Hi Maia,

sorry, I have followed these study for so long now that I didn't

recognize that they weren't mentioning stop-mutations this time.

>>Some 5 percent to 10 percent of cystic fibrosis patients in

general -- but the majority of patients of Ashkenazi Jewish

descent -- have the kind of defect gentamicin targets.<<

The Ashkenazi Jews Mutation is W1282X and 60% of the Ashkenazi pwcf

carry that mutation.

CPX

CPX is a small molecule protein-repair therapy for cystic fibrosis

(CF), the most common fatal genetic disorder among Caucasians. CPX is

an orally-administered drug with the potential to treat the

underlying cause of CF. Current treatments for CF address only the

symptoms, which ultimately include a build-up of viscous mucus in the

lungs that harbor infections and which lead to chronic pulmonary

disease and death in most patients.

SciClone has been granted Orphan Drug Status for CPX in both the U.S.

and Europe, and has received financial support for the clinical

program from both the FDA and the Cystic Fibrosis Foundation.

SciClone has licensed CPX from the National Institutes of Health

(NIH).

In previous preclinical studies conducted at the National Institutes

of Health (NIH), CPX demonstrated the ability to repair the two

principal protein defects underlying the cause of cystic fibrosis

(CF). CPX appears to enable the defective protein to travel through

the cell and reach the epithelial cell membrane ( " trafficking " ) and

to improve an originally impaired transport of chloride ions across

the cell membrane. This mechanism of action is intended to prevent

the build-up of viscous mucus in the first place, and thus remove the

danger of fatal infection common in CF patients.

SciClone's first U.S. phase 2 trial aimed at demonstrating this

protein repair activity in CF patients did not produce the sustained

circulatory drug levels required to assess efficacy because of the

erratic digestive absorption patterns of the CF patients. SciClone

worked with the Cystic Fibrosis Foundation's Therapeutic Development

Network and reformulated CPX to prepare for additional studies.

Development activities aimed at new phase 2 studies for CPX continue.

> Torsten

> Where exactly in the article it says it will help

> patients with stop-mutations?

>

> What is CPX?

>

> Maia

>

>

>

> > this cure (if it is one) will only work for

> > stop-mutations. But other

> > meds like CPX are designed especially for the dF508

> > mutation.

> >

> > Torsten

[Guest guest]

Hi Maia,

sorry, I have followed these study for so long now that I didn't

recognize that they weren't mentioning stop-mutations this time.

>>Some 5 percent to 10 percent of cystic fibrosis patients in

general -- but the majority of patients of Ashkenazi Jewish

descent -- have the kind of defect gentamicin targets.<<

The Ashkenazi Jews Mutation is W1282X and 60% of the Ashkenazi pwcf

carry that mutation.

CPX

CPX is a small molecule protein-repair therapy for cystic fibrosis

(CF), the most common fatal genetic disorder among Caucasians. CPX is

an orally-administered drug with the potential to treat the

underlying cause of CF. Current treatments for CF address only the

symptoms, which ultimately include a build-up of viscous mucus in the

lungs that harbor infections and which lead to chronic pulmonary

disease and death in most patients.

SciClone has been granted Orphan Drug Status for CPX in both the U.S.

and Europe, and has received financial support for the clinical

program from both the FDA and the Cystic Fibrosis Foundation.

SciClone has licensed CPX from the National Institutes of Health

(NIH).

In previous preclinical studies conducted at the National Institutes

of Health (NIH), CPX demonstrated the ability to repair the two

principal protein defects underlying the cause of cystic fibrosis

(CF). CPX appears to enable the defective protein to travel through

the cell and reach the epithelial cell membrane ( " trafficking " ) and

to improve an originally impaired transport of chloride ions across

the cell membrane. This mechanism of action is intended to prevent

the build-up of viscous mucus in the first place, and thus remove the

danger of fatal infection common in CF patients.

SciClone's first U.S. phase 2 trial aimed at demonstrating this

protein repair activity in CF patients did not produce the sustained

circulatory drug levels required to assess efficacy because of the

erratic digestive absorption patterns of the CF patients. SciClone

worked with the Cystic Fibrosis Foundation's Therapeutic Development

Network and reformulated CPX to prepare for additional studies.

Development activities aimed at new phase 2 studies for CPX continue.

> Torsten

> Where exactly in the article it says it will help

> patients with stop-mutations?

>

> What is CPX?

>

> Maia

>

>

>

> > this cure (if it is one) will only work for

> > stop-mutations. But other

> > meds like CPX are designed especially for the dF508

> > mutation.

> >

> > Torsten

[Guest guest]

Hi Maia,

sorry, I have followed these study for so long now that I didn't

recognize that they weren't mentioning stop-mutations this time.

>>Some 5 percent to 10 percent of cystic fibrosis patients in

general -- but the majority of patients of Ashkenazi Jewish

descent -- have the kind of defect gentamicin targets.<<

The Ashkenazi Jews Mutation is W1282X and 60% of the Ashkenazi pwcf

carry that mutation.

CPX

CPX is a small molecule protein-repair therapy for cystic fibrosis

(CF), the most common fatal genetic disorder among Caucasians. CPX is

an orally-administered drug with the potential to treat the

underlying cause of CF. Current treatments for CF address only the

symptoms, which ultimately include a build-up of viscous mucus in the

lungs that harbor infections and which lead to chronic pulmonary

disease and death in most patients.

SciClone has been granted Orphan Drug Status for CPX in both the U.S.

and Europe, and has received financial support for the clinical

program from both the FDA and the Cystic Fibrosis Foundation.

SciClone has licensed CPX from the National Institutes of Health

(NIH).

In previous preclinical studies conducted at the National Institutes

of Health (NIH), CPX demonstrated the ability to repair the two

principal protein defects underlying the cause of cystic fibrosis

(CF). CPX appears to enable the defective protein to travel through

the cell and reach the epithelial cell membrane ( " trafficking " ) and

to improve an originally impaired transport of chloride ions across

the cell membrane. This mechanism of action is intended to prevent

the build-up of viscous mucus in the first place, and thus remove the

danger of fatal infection common in CF patients.

SciClone's first U.S. phase 2 trial aimed at demonstrating this

protein repair activity in CF patients did not produce the sustained

circulatory drug levels required to assess efficacy because of the

erratic digestive absorption patterns of the CF patients. SciClone

worked with the Cystic Fibrosis Foundation's Therapeutic Development

Network and reformulated CPX to prepare for additional studies.

Development activities aimed at new phase 2 studies for CPX continue.

> Torsten

> Where exactly in the article it says it will help

> patients with stop-mutations?

>

> What is CPX?

>

> Maia

>

>

>

> > this cure (if it is one) will only work for

> > stop-mutations. But other

> > meds like CPX are designed especially for the dF508

> > mutation.

> >

> > Torsten