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Neurogene falls again on Rett gene therapy side effects

Neurogene has halted patient enrolment in the high-dose arm of its trial of Rett syndrome gene therapy, sparking another run on its shares.

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AbbVie bags EU nod for key drug from ImmunoGen takeover

AbbVie's $10.1 billion takeover of ImmunoGen has been rewarded with EU approval for Elahere, the first drug specifically cleared for folate receptor-alpha (FRα) positive, platinum-resistant

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Tirzepatide gets a big win in obesity-related heart failure

Eli Lilly's tirzepatide has been shown to significantly cut cardiovascular risks in patients with obesity and a form of heart failure with few treatment options, in another big win for the

News

Trump pick of RFK for top US health job astounds critics

Last night, news broke that President-Elect Donald Trump had nominated Robert F Kennedy Jr (aka RFK Jr) as US Health Secretary.

News

MSD joins PD-1/VEGF push in cancer with LaNova deal

There has been a lot of talk about drugs targeting both PD-1 and VEGF stealing the crown held by MSD's Keytruda in cancer immunotherapy.

News

ASH: AZ’s danicopan shows long-term benefit in PNH

New data with AstraZeneca’s oral Factor D inhibitor danicopan have built the case for the drug as an add-on to complement C5 antibodies for the treatment of paroxysmal noc

News

ASH: Pfizer builds case for long-acting haemophilia drug

Pfizer’s once-weekly antibody injection for haemophilia A and B, marstacimab, could be on track for regulatory approvals based on solid results in a phase 3 trial reported

R&D

Predictive genetic testing offers the greatest promise and e...

If your genetic makeup put you at greater risk of disease, would you want to know?

Debates & Insights

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Synthetic control arms in clinical trials: Making it happen

Across the industry, pharma companies are turning to AI and real-world data to address many of the challenges of running clinical trials.

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How synthetic control arms are changing clinical trials

Clinical trials are involved, expensive, and have been done largely the same way for decades but with today’s technology all that can change.

News

Neurogene falls again on Rett gene therapy side effects

Neurogene has halted patient enrolment in the high-dose arm of its trial of Rett syndrome gene therapy, sparking another run on its shares.

News

AbbVie bags EU nod for key drug from ImmunoGen takeover

AbbVie's $10.1 billion takeover of ImmunoGen has been rewarded with EU approval for Elahere, the first drug specifically cleared for folate receptor-alpha (FRα) positive, platinum-resistant

Press Releases

Views & Analysis

Touring Chiesi's vision for the future at the new Biotech Ce...

Following an invite to fly into Parma, Italy to witness first-hand the press launch of the considerable structure – black-painted and as yet under construction, gigantic cranes and all – th

News

Vir awarded $1 billion multi-year BARDA influenza contract

The US Government’s Biomedical Advanced Research and Development Authority (BARDA) has made an

Views & Analysis

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Returning... The 5th Operationalize Expanded Access Programs...

Welcome to, or welcome back to, the 5th Operationalize: Expanded Access Programs Summit 2025!

R&D

Growing threat of AMR: UN calls for urgent global action

In response to the rise of AMR, world leaders recently convened at the United Nations General Assembly to forge a landmark commitment, setting ambitious targets and funding initiatives to c

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Sponsored

Webinar recap: Boosting recruitment and retention with SDOH ...

As the pharmaceutical industry continues its shift towards more patient-centric models, the incorporation of social determinants of health (SDOH) data has become increasingly valuable.

Deep Dive

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The many trends reshaping clinical trials: An interview with...

Over the last five years, a confluence of trends have combined to reshape the reality of clinical research. COVID-19 pushed decentralised trials into the mainstream.

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Heard on the pipeline: AI in R&D

As has become tradition in the pharma and biotech world, 2024 kicked off with a bang as experts from around the globe gathered in San Francisco for the 42nd annual JP Morgan Health Conferen

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Accelerating innovation in rare diseases

Millions of people living with rare diseases are urgently waiting for diagnosis, care, and life-changing treatment options.

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Sponsored

Beyond clinical: Boosting recruitment and retention with SDO...

The success of clinical trials hinges on a complex array of factors, many of which extend beyond traditional clinical care.

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evolution of clinical research in the UK

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The evolution of clinical research: Embracing new delivery m...

The UK is welcoming a new era of clinical trial delivery, evolving beyond the traditional site-centric approach to also embrace remote, decentralised, virtual, and hybrid trials and enablin

R&D

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EDI in clinical research: The case for adaptive strategies a...

The topic of Equality, Diversity, and Inclusion (EDI) in health and care research participation has been gaining traction in the past few years.

Podcasts

R&D

From neurosurgery to focused ultrasound: Not if, but when

Therapeutic or focused ultrasound began being applied to neurologic conditions less than a decade ago, but its potential in a wide spectrum of brain applications is high.

R&D

LSX USA Jonah Comstock and Eloise McLennan Wrapup

Investment outlook: Key takeaways from LSX USA

Investment and strategic partnerships are the lifeblood of innovation in healthcare, driving groundbreaking discoveries and accelerating the development of life-changing therapies.

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Tips for on-time tech transfers that stay on budget

When it comes to life sciences manufacturing, there's nothing quite so daunting as a tech transfer - moving a product from development to commercialisation, or to a new facility or CDMO, pr

Video

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Jonah Comstock interview with Dr Tadaaki Taniguchi

LSX 2024 - Dr Tadaaki Taniguchi

At LSX in Boston last month, editor-in-chief Jonah Comstock caught up with Dr Tadaaki Taniguchi, chief medical officer at Astellas Pharmaceuticals to talk a bit about the company and some r

R&D

Interview with James Graham Recce Pharma

LSX 2024 - James Graham, Recce Pharma

A lot of ink has been spilled about the anti-microbial resistance space and why it’s so hard to create a profitable company in it.

R&D

Richard Young, Nicole Raleigh, Daniel Schwenk and Andrea Kloeble

Boehringer Ingelheim and Veeva: A data partnership for One M...

Boehringer’s Medicine Excellence initiative harnesses technology with the aim to build a ‘One Medicine Platform’, unifying development processes and data in a centralised platform.

White Papers

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Rare Disease Diagnostics: Advancing Orphan Drug Development ...

This white paper is free and can be downloaded and read at your convenience

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Envisioning a World Without Disease

In June, pharmaphorum came together with the Connected World Without Disease Accelerator for the inaugural World Without Disease Summit, a two-day event that brought together stakeholders f

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Partner Content

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Partner Content

LEAP HR: Life Sciences Europe

Save the Date for LEAP HR: Life Sciences Europe! 18th-20th February, 2025, London, UK.

R&D

3rd Molecular Glue Drug Development Summit

Partner Content

Welcome to the Molecular Glue Drug Development Summit!

The Molecular Glue Drug Development Summit is back in January, 2025!

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Partner Content

Welcome to the 6th RNAi-Based Therapeutics Summit!

RNAi-Based Therapeutics Returns!

Neurogene falls again on Rett gene therapy side effects

AbbVie bags EU nod for key drug from ImmunoGen takeover

Tirzepatide gets a big win in obesity-related heart failure

Trump pick of RFK for top US health job astounds critics

MSD joins PD-1/VEGF push in cancer with LaNova deal

ASH: AZ’s danicopan shows long-term benefit in PNH

ASH: Pfizer builds case for long-acting haemophilia drug

Predictive genetic testing offers the greatest promise and e...

Synthetic control arms in clinical trials: Making it happen

How synthetic control arms are changing clinical trials

Neurogene falls again on Rett gene therapy side effects

AbbVie bags EU nod for key drug from ImmunoGen takeover

Touring Chiesi's vision for the future at the new Biotech Ce...

Vir awarded $1 billion multi-year BARDA influenza contract

Returning... The 5th Operationalize Expanded Access Programs...

Growing threat of AMR: UN calls for urgent global action

Webinar recap: Boosting recruitment and retention with SDOH ...

The many trends reshaping clinical trials: An interview with...

Heard on the pipeline: AI in R&D

Accelerating innovation in rare diseases

Beyond clinical: Boosting recruitment and retention with SDO...

The evolution of clinical research: Embracing new delivery m...

EDI in clinical research: The case for adaptive strategies a...

From neurosurgery to focused ultrasound: Not if, but when

Investment outlook: Key takeaways from LSX USA

Tips for on-time tech transfers that stay on budget

LSX 2024 - Dr Tadaaki Taniguchi

LSX 2024 - James Graham, Recce Pharma

Boehringer Ingelheim and Veeva: A data partnership for One M...

Rare Disease Diagnostics: Advancing Orphan Drug Development ...

Envisioning a World Without Disease

FT US Pharma and Biotech Summit: Live coverage

FT Live US Pharma and Biotech Summit 2023 - Day 2

FT Live US Pharma and Biotech Summit 2023

LEAP HR: Life Sciences Europe

Welcome to the Molecular Glue Drug Development Summit!

Welcome to the 6th RNAi-Based Therapeutics Summit!

Neurogene falls again on Rett gene therapy side effects

Decoding physician prescribing behaviour: A data-driven jour...

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