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- PMLiVE

UniQure

Biomarin

FDA keeps BioMarin’s gene therapy for phenylketonuria on hold

The decision follows data suggesting BioMarin’s experimental gene therapies may have the potential to progress cancers

- PMLiVE

bluebird bio’s beti-cel accepted for priority review by FDA

The tough journey to approval for beta-thalassemia gene therapy (beti-cel) in the US may be coming to an end, with approval possible by 2022

Disrupting Cell Engineering with Non-Viral Modalities

Jessica Schwaber, Scientific Director, Cell & Gene Therapy, Avectas, explores her company’s unique cell engineering platform, the recent shift in cell & gene therapy commercial needs, current barriers to realizing...

Impetus Digital

- PMLiVE

AbbVie signs gene therapy deal in eye care

AbbVie and REGENXBIO have teamed up on RGX-314, a potential one-time gene therapy against two leading causes of blindness, wet AMD and diabetic retinopathy.

Biomarin

FDA pauses BioMarin gene therapy trial

The study for BMN 307 in genetic disease phenylketonuria will be paused while malignancies in mice are explored

Realizing the Potential of Genomics-Driven Precision Medicine

Jennifer Helfer, Senior Director, Patient Advocacy & Engagement at Encoded Therapeutics Inc., discusses genomics-driven precision medicine, gene therapy misconceptions and fears, involving patients and caregivers in designing gene therapy trials,...

Impetus Digital

- PMLiVE

Researchers publish promising results for ultra-rare disease gene therapy method

Gene therapy method evaluated in seven children with aromatic amino acid decarboxylase deficiency

- PMLiVE

bluebird bio lodges counterclaim against Spark Therapeutics

Spark previously accused bluebird of misusing trademarks relating to the word ‘spark’ in marketing/educational materials

- PMLiVE

FDA grants priority review for AZ, Amgen’s tezepelumab for asthma

Drug 'has the potential to transform treatment for a broad population of severe asthma patients'

- PMLiVE

Merck & Co withdraws Keytruda’s US approval in third-line stomach cancer

The FDA initially approved Keytruda in 2017 for stomach cancer based on overall response rate data

- PMLiVE

New data shows benefits for presymptomatic SMA patients treated with Zolgensma

Zolgensma was initially approved in 2019 for the treatment of SMA, launching with a list price of $2.125m

- PMLiVE

Sarepta Therapeutics’ DMD gene therapy shows early success

Most patients with Duchenne muscular dystrophy require full-time use of a wheelchair by their early teens

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