Gene Therapy

Sanofi invests $30m in MeiraGTx’s gene therapy pipeline

Sanofi invests $30m in MeiraGTx’s gene therapy pipeline

By Jonathan Smith

The London and New York-based gene therapy player MeiraGTx Holdings has received a $30 million investment from Sanofi in addition to potential strategic deals with the big pharma company down the road.

© Getty Images

Gene sequencing identifies rare pediatric disorders in Mexico

By Isabel Cameron

A new genetic sequencing technology from Element Biosciences has helped researchers from the Translational Genomics Research Institute (TGen), part of the City of Hope, identify the likely genetic causes of disorders in six of nine children from Sonora,...

BioMarin opens €38m expansion at Irish manufacturing site

BioMarin opens €38m expansion at Irish manufacturing site

By Rachel Arthur

BioMarin Pharmaceutical has opened the expansion of its manufacturing plant at Shanbally, Co. Cork: with the site now able to provide end-to-end manufacturing for a number of the company’s commercial products.

Pic:getty/stocktrekimages

bluebird bio submits BLA for sickle cell disease gene therapy

By Rachel Arthur

bluebird bio has announced the submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for lovotibeglogene autotemcel (lovo-cel) gene therapy in patients with sickle cell disease (SCD).

Pic:getty/andrejrostek

Novartis releases long-term Zolgensma data

By Rachel Arthur

Novartis has presented new data which ‘underscore the transformational and sustained benefit’ of spinal muscular atrophy (SMA) gene therapy Zolgensma (onasemnogene abeparvovec): with treated children maintaining motor milestones after 7.5 years.

Pic:gettyismagilov

EU approves first gene therapy for hemophilia B

By Jonathan Smith

Three months after receiving the US green light, CSL’s candidate Hemgenix has become the first gene therapy to gain EU approval for the treatment of hemophilia B.

Pic:getty/totojang

CGT Catapult boost for UK life science campus

By Rachel Arthur

The Cell and Gene Therapy Catapult (CGT Catapult) has confirmed its involvement in the development of a new £900m ($1,097m) life science campus in Stevenage, UK: which is set to become one of the largest in Europe.

© GettyImages/AlexanderFord

Opus acquires two compounds for inherited retinal diseases

By Jane Byrne

Last week saw Opus Genetics announce it had acquired the rights to two preclinical-stage adeno-associated virus (AAV)-based gene therapy product candidates for inherited retinal diseases (IRDs) from Iveric Bio.