Pharmafile Logo
  • - PMLiVE
  • - PMLiVE
  • Diversity, Equity and Inclusion
  • Communiqué 53 Cover Image

Lenti-D

- PMLiVE

Lilly and MeiraGTx partner in $475m ophthalmology deal

The companies will develop and commercialise therapies for genetic conditions

The (clinical) trials and tribulations of gene therapy – why meaningful patient engagement strategies matter

By Sarah Mohamad and Lauren Coleman-Bennett

Amiculum

What do people look for in gene therapy materials? Results from our survey of patients and relatives

By Sam Fraser

Amiculum

- PMLiVE

CSL Behring announces four-year results for haemophilia B gene therapy Hemgenix

The genetic bleeding disorder affects an estimated one per every 19,283 male births in the US

- PMLiVE

NICE approves Vertex’s Casgevy one-time gene therapy for sickle cell disease

About 15,000 people in England are living with the inherited blood disorder

- PMLiVE

Vertex and Orum partner on gene editing conditioning drugs in deal worth $945m

The agreement gives Vertex the rights to use Orum’s targeted protein degradation technology

- PMLiVE

FDA publishes final industry guidance for CAR-T and gene therapies

The new guidance can also be applied to other genetically modified lymphocyte products, including  CAR NK cells

- PMLiVE

FDA approves two cell-based gene therapies for sickle cell disease

Vertex/CRISPR and bluebird bio’s gene therapies have been approved to treat patients aged 12 years and older

- PMLiVE

bluebird bio presents positive results for inherited blood disorder gene therapies

The company’s sickle cell disease therapy was accepted for FDA priority review earlier this year

- PMLiVE

AstraZeneca and Cellectis enter gene therapy partnership worth over $2.2bn

The deal gives AZ access to Cellectis’ gene editing technologies and manufacturing capabilities

- PMLiVE

bluebird bio to sell FDA priority review voucher for $103m

The company previously sold its second voucher to Bristol Myers Squibb for $95m

Biomarin

BioMarin announces first patient dosed with haemophilia A gene therapy in Europe

The therapy was granted conditional marketing authorisation by the EC in August 2022

Subscribe to our email news alerts

Latest content

Latest intelligence

Quick links