WhatsApp Logo Subscribe
For News Alert

News

Girl aged 5 dies in US during French gene therapy trial

October 15, 2020 07:09 PM


Twitter Share Facebook Share WhatsApp Share

A five-year-old girl with a rare neurodegenerative disease died in the US while taking part in a gene therapy trial run by French biotechnology company Lysogene, the firm said Thursday.

The little girl was suffering from Sanfilippo syndrome or mucopolysaccharidosis type III, a rare genetic disease that alters brain development after birth and leads to premature death.

In a statement Lysogene, a company developing gene therapy for central nervous system diseases in children, said "the immediate cause of death is currently unknown" and that there was as yet "no evidence that the event is linked to the study drug administration".

It said it was "profoundly saddened by the passing of this child" and was collecting "additional information" about the circumstances.

The share price of the company dropped 19 percent to 2.05 euros in morning trading in Paris.

The girl was one of 19 people being treated in the trial conducted at eight hospitals in Europe and the United States.

She died at home several months after receiving the therapy, consisting of a single injection, at one of four treatment sites in the US, Lysogene told AFP.

In its statement the company said it was following the remaining 18 patients closely and remained "committed to the LYS-SAF302 development program".

On June 5, the US Food and Drug Administration (FDA) ordered a clinical hold on the trial after observing "localized findings on MRI images at the intracerebral injection sites" suggesting "a potential connection to delivery".

In a statement at the time the company said "no clinical symptoms have been observed that could be directly attributed to the observed MRI findings".

 



Most Read

  1. Hania Aamir and Zaviyar Nauman's Nikkah glam gives ‘heart attack’ to many Hania Aamir and Zaviyar Nauman's Nikkah glam gives ‘heart attack’ to many
  2. Actress Khushboo shot dead in Nowshera Actress Khushboo shot dead in Nowshera
  3. Guard shoots dead his employer in Lahore’s Ghalib Market Guard shoots dead his employer in Lahore’s Ghalib Market
  4. Sania Mirza seeks forgiveness ahead of Hajj pilgrimage Sania Mirza seeks forgiveness ahead of Hajj pilgrimage
  5. Wajdan’s high demands from Chahat Fateh Ali Khan shock everyone Wajdan’s high demands from Chahat Fateh Ali Khan shock everyone
  6. Ex-CJP Saqib Nisar uses influence for grandson at Aitchison College Ex-CJP Saqib Nisar uses influence for grandson at Aitchison College

Opinion

  1. An Unmissable Opportunity: The Time for Modi and Islamabad leadership to Resume Bilateral Dialogue is Now!
    An Unmissable Opportunity: The Time for Modi and Islamabad leadership to Resume Bilateral Dialogue is Now!

    By Sudheendra Kulkarni

  2. Premier Shehbaz and Chinese model
    Premier Shehbaz and Chinese model

    By Naveed Aman Khan

  3. Modi's myth broken, yet in power
    Modi's myth broken, yet in power

    By Naveed Aman Khan

  4. Xi, Shehbaz and CPEC phase - 2
    Xi, Shehbaz and CPEC phase - 2

    By Naveed Aman Khan

  5. Pakistan's UNSC Triumph: A New Era
    Pakistan's UNSC Triumph: A New Era

    By Dr Asif Channer

  6. Shehbaz, Deng, Xi and transformation
    Shehbaz, Deng, Xi and transformation

    By Naveed Aman Khan