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gene therapies

This page shows the latest gene therapies news and features for those working in and with pharma, biotech and healthcare.

bluebird bio reveals plans to launch two gene therapies

Both therapies were endorsed by the US FDA Cell Tissue &Gene Therapy Advisory Committee. ... elivaldogene autotemcel (eli-cel) gene therapies in the fourth quarter of the year.

Latest news

Merck KGaA announces launch of start-up collaboration programme in Asia

Merck. The company noted that there will be a strong focus on companies working in manufacturing, utilising cellular, molecular- and immuno-assays or cell and gene therapy tools, engaging in manufacturing
Novartis and Precision BioSciences announce in vivo gene editing collaboration

Novartis and Precision BioSciences (Precision) have announced an in vivo gene editing research and development collaboration to develop potentially curative treatments for disorders including sickle cell disease (SCD) and beta thalassaemia.
Novartis publishes final positive phase 3 results of Zolgensma in Nature Medicine

Almost all children involved with copies of the SMN2 gene treated pre-symptomatically met key milestones. ... presymptomatic babies with SMA,” said Shephard Mpofu, senior vice president, chief medical officer, Novartis Gene Therapies.
bluebird bio maintains its focus on gene therapy opportunities during structural changes

The company intends to increase its focus on near-term catalysts – including anticipated FDA approvals for beta-thalassemia and cerebral adrenoleukodystrophy gene therapies. ... Over more than a decade, Bluebird has made remarkable advances across
Novartis and Voyager Therapeutics agree on gene therapy deal worth 1.7bn

therapies for neurological diseases, with options to access capsids for two other targets. ... The challenge of biodistribution of AAV gene therapies to target cell types in the brain is longstanding, but we refuse to believe it is insurmountable,”

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Latest Intelligence

Charting patient pathways with precision for better healthcare

We will see faster progress in areas such as CAR-T and gene therapies because these are personalised medicines and the patient is going to have as much of a
How early-stage clinical research is facilitating the new wave of personalised medicines

Designing studies for gene therapies is not simply ‘business as usual’ for contract research organisations (CRO) and clinical investigators. ... It is important that physicians seek to develop more advanced and specialist skills via such schemes to
EU cross-border healthcare

and this is becoming increasingly clear as more and more gene therapies come to market. ... The reality for many patients and their families is sobering. Gene therapies and other Advanced Therapy Medicinal Products (ATMPs) require specialist treatment
Mapping the patient journey: eight ways to improve support programmes

In some areas, such as gene therapies and high-cost cancer treatments, accelerated approval and early access schemes are conditional on capturing real-world outcomes data – and patient support tools provide
Rare genetic diseases – what’s in store for 2021?

In my opinion, these new pathways will establish themselves and be extended to other treatments, including cell and gene therapies. ... Mike Fraser is General Manager, EMEA at Novartis Gene Therapies. 16th February 2021. .

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Latest appointments

Health comms agency emotive hires new cell and gene therapy leader

An expert in cell and gene therapies, Dr Ferrari joins emotive following 20 years in research as well as holding medical affairs and commercial roles in biopharma and diagnostics. ... The group has ongoing work in preparing for the global launch of one
Prevail Therapeutics adds to its board of directors

Francois Nader joins the biotech. Prevail Therapeutics, a biotechnology company focused on the development of gene therapies for patients with neurodegenerative diseases, has strengthened its board of directors with the addition ... advance our pipeline
Abeona Therapeutics appoints new additions to board of directors

Stefano Buono and Richard Van Duyne join the biopharma. Abeona Therapeutics, a clinical stage biopharmaceutical company focused on developing cell and gene therapies, has expanded its board of directors through the ... He said: “Abeona’s ambitious
Avita Medical appoints CEO

chief scientific officer, global business development and licensing, and chief scientific officer for its cell and gene therapy unit. ... and gene therapies for Novartis subsidiaries Systemix Inc.
apceth Biopharma appoints Dusan Kosijer as CFO

In parallel, apceth’s pipeline has recently entered a very thrilling phase, encouraging us to accelerate further the development of our first-in-class, first-in-man cell-based gene therapies.”. ... successful track record as one of the leading CDMOs

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Latest from PMHub

The desire for connected customer experiences unites stakeholders

There are more therapies to choose from and different kinds of treatments such as cell and gene therapies that require entirely different engagement models.
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 the full ... potential of cell &gene
Addressing the Gendered Health Data Gap

When thinking about personalised treatment today, we think of targeted cancer treatments, or gene therapies.
Gene Editing and Precision Medicine: The Future of Healthcare?

Dr. Oscar Segurado, Chief Medical Officer at ASC Therapeutics, dives into advanced gene therapies, gene editing, and personalized medicine.
What NORD’s RareInsights® 30-year comparative analysis means to us and our clients at OPEN Health

Generate and disseminate comparative effectiveness evidence, when available, to highlight the value of new therapies and raise awareness across stakeholders and support access and adoption. ... This hope and expectation is driven by the further