Список форумов Дети со Спинальной Мышечной Атрофией Дети со Спинальной Мышечной Атрофией
Children with SMA, Ukraine
 
 FAQFAQ   ПоискПоиск   ПользователиПользователи   ГруппыГруппы   РегистрацияРегистрация 
 ПрофильПрофиль   Войти и проверить личные сообщенияВойти и проверить личные сообщения   ВходВход 

3 новые программы ФСМА

 
Начать новую тему   Ответить на тему    Список форумов Дети со Спинальной Мышечной Атрофией -> Международные события
Предыдущая тема :: Следующая тема  
Автор Сообщение
admin
Site Admin


Зарегистрирован: 30.11.2006
Сообщения: 366
Откуда: Харьков

СообщениеДобавлено: Пн Июн 29, 2009 10:45 am    Заголовок сообщения: 3 новые программы ФСМА Ответить с цитатой

Families of Spinal Muscular Atrophy has just released details of the latest developments for the Families of SMA funded therapeutic pipeline. Families of SMA is currently funding three distinct drug discovery programs:
1) Quinazolines to boost gene expression;
2) Tetracyclines at Paratek Pharmaceuticals to correct gene splicing;
3) Motor neuron replacement program at California Stem Cell and UCI.

Quinazoline Program:

In June 2009, the IND-enabling safety studies were completed for the FSMA-directed Quinazoline program. This is the program previously worked on by Vertex Pharmaceuticals and deCODE genetics, and fully funded by Families of SMA.

The next step for this program is to request a pre-IND meeting with the FDA and to prepare the required data package. Families of SMA will be working over the next several months with toxicology and regulatory experts to prepare for this meeting. According to Jill Jareck
i, Ph.D., Research Director FSMA, "This meeting will be an exciting milestone for the project, and an essential step in the path towards human clinical trials of the drug candidate."

Motor Neuron Replacement Program:

California Stem Cell (CSC) is preparing for a final FDA pre-IND meeting to take place in mid 2009. This is a critical step on the track to submitting a formal Investigational New Drug (IND) application to the FDA to begin clinical trials in SMA Type I. It is the intent of CSC to gain approval to begin FDA-approved clinical trials for the use of these cells in development of a cell replacement therapy for SMA Type I.

Numerous pre-clinical efficacy studies have been completed, demonstrating that the cells work. The pivotal animal safety study, required to support an application to begin clinical trials, was completed in October 2008. According to Chris Airriess, Ph.D., Chief Operating Officer, California Stem Cell, Inc., "CSC is now preparing for a final FDA pre-
IND meeting to take place in mid 2009, keeping us on track for a formal IND application to begin a Phase I/IIA clinical trial in SMA Type I."

Tetracycline Program:

According to Paul Higgins, Ph.D., Director, Inflammation Drug Discovery, Paratek Pharmaceuticals, "FSMA has supported the research at Paratek for the previous three years for our work on tetracycline derivatives as a potential treatment of SMA. These compounds are intended to correct SMN2 splicing and in turn increase SMN protein levels.

Using funding from FSMA to generate the preliminary data for a grant application, Paratek has been awarded a multi-million dollar U01 grant from the NINDS to continue our research that FSMA funded for the past 3 years."

The overall goal for this project is to develop a drug candidate for SMA resulting in an Investigational New Drug (IND) application with the Food and Drug Administration (FDA) within 4 to 5 years.

These three announcements were all made at the 25th A
nniversary SMA Conference. A record of over 900 families and researchers were present at the conference.
Вернуться к началу
Посмотреть профиль Отправить личное сообщение Отправить e-mail Посетить сайт автора
Показать сообщения:   
Начать новую тему   Ответить на тему    Список форумов Дети со Спинальной Мышечной Атрофией -> Международные события Часовой пояс: GMT + 2
Страница 1 из 1

 
Перейти:  
Вы не можете начинать темы
Вы не можете отвечать на сообщения
Вы не можете редактировать свои сообщения
Вы не можете удалять свои сообщения
Вы не можете голосовать в опросах


Powered by phpBB © 2001, 2005 phpBB Group
Русская поддержка phpBB