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NICE recommends gene therapy for severe sickle cell disease
The committee’s approval of the one-time gene therapy means the healthcare inequalities for patients with this genetic blood disorder can begin to be re-addressed.
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Catherine Eckford (European Pharmaceutical Review)
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Takeda plans 2026 CEO succession
The biopharmaceutical company’s executive transition follows a multi-year succession process to support its next chapter.
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Enhancing spectroscopy analysis with machine learning
The spectroscopy-based study highlights the importance of integrating data-driven approaches to enhance isomer discrimination.
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New analysis asserts survival benefit for Roche breast cancer therapy
The new data highlights the potential of the ItovebiTM (inavolisib)-based regimen as a new standard of care in advanced PIK3CA-mutated, HR-positive, HER2-negative breast cancer.
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UK biotech on “positive trajectory”, says BIA
Cutting-edge science and large international investment are driving growth in the UK’s biotech sector, the organisation’s new report reveals.
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AstraZeneca Canadian investment to drive R&D growth
The US$570m investment will contribute to advancing AstraZeneca’s global clinical pipeline, a key goal of its strategy to 2030.
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M&A outlook for pharma in 2025
For a strong 2025, dealmaking should be central to the life sciences strategy, says EY’s annual M&A report.
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Biologic demonstrates “tremendous” potential advance in atrial fibrillation
The Phase II study validated an “incredibly safe bleeding profile” for Factor XI inhibitors as a treatment for the common cardiovascular condition.
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FDA approves innovative monotherapy for treatment-resistant depression
J&J’s standalone small molecule drug was approved based on evidence of improvement of depressive symptoms without the need for patients to take a daily oral antidepressant.
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Sustainability and technology advancement driving spectroscopy market
Integration of industry 4.0 is anticipated to steer growth of the process spectroscopy market into the next decade, a report predicts.
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Early-phase ATMP clinical trials see promising growth
Cell and Gene Therapy Catapult's (CGT Catapult) findings attest to the UK remaining “a highly attractive destination for clinical trials”.
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New guidance to aid cost-effectiveness analysis of new drugs
The report aims to support development of economic models for health technology assessment (HTA) decision making, as there has been “little change in guidance on the use of surrogate endpoints in HTA since 2018”.
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Lilly IBD biologic receives expanded US approval
The drug is the first biologic in over 15 years to have disclosed two-year Phase III efficacy data in Crohn's disease at the time of approval.
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Ensuring packaging integrity of frozen biopharmaceutical products
The study’s observations offers insight for pharmaceutical professionals in choosing the appropriate primary packaging container for frozen biopharmaceutical products.
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Long-term potential revealed for novel modifier gene therapy
The positive two-year findings from the Phase I/II gene therapy trial in retinitis pigmentosa reiterate the potential of mutation-agnostic therapies.
