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- PMLiVE

Sarepta

- PMLiVE

Sarepta to submit application for Duchenne muscular dystrophy gene therapy

The condition occurs in approximately one in every 3,500-5,000 newborn males worldwide

- PMLiVE

bluebird bio maintains its focus on gene therapy opportunities during structural changes

The company plans to reduce its workforce amid efforts to reduce its operating costs

- PMLiVE

Novartis and Voyager Therapeutics agree on gene therapy deal worth 1.7bn

The licence option agreement is for next-generation gene therapy vectors for neurological diseases

- PMLiVE

bluebird bio shares therapy updates for 2022

The company provided information for its first two gene therapies and its lovo-cel BLA submission for sickle cell disease

Takeda agrees on $2bn deal with Code Bio to expand its gene therapy programmes

The pharma company will gain opt-in rights for four rare diseases candidates

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.

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

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