Thu.Nov 16, 2023

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Not-for-profit alliance advocates for life sciences collaboration

BioPharma Reporter

The Pistoia Alliance is a global, not-for-profit alliance that advocates for greater collaboration in life sciences R&D. It has just announced its four new strategic priorities made up of global pharma and tech representatives to deliver new project ideas to drive industry change.

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World’s first CRISPR medicine approved in UK for sickle cell, beta thalassemia

Bio Pharma Dive

Clearance of Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy in the U.K. comes ahead of expected regulatory decisions in the U.S. and Europe.

Medicine 306
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World’s first CRISPR-based gene therapy approved for sickle cell anaemia

Pharmaceutical Technology

Vertex and CRISPR's Casgevy has received conditional approval in the UK for treating sickle cell and transfusion-dependent beta-thalassemia.

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FDA approves new Bristol Myers drug for lung cancer

Bio Pharma Dive

Acquired via a $4 billion biotech buyout, Augtyro is one of an array of new products the pharma hopes will offset patent expirations for current top sellers.

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Optimizing Clinical Supply Strategy: Navigating Challenges & Finding Your Ideal Model

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Xphozah wins orphan drug designation for paediatric hyperphosphatemia

Pharmaceutical Technology

Ardelyx scores another FDA win after Xphyozah was approved by the FDA in October as an add-on therapy for chronic kidney disease patients.

Drugs 246
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West Coast investor Red Tree on its San Diego expansion and staying bullish in biotech

Bio Pharma Dive

Red Tree Venture Capital plans to tap into San Diego’s biotech pipeline as it works to build a West Coast competitor to Boston-area investors.

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Homology enters reverse merger with Q32 Bio

Bio Pharma Dive

The deal, which will result in a combined company operating under Q32's name, comes months after Homology laid off much of its staff and halted research.

Research 264
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An ‘Extra Fold’ in The Human Brain May Delay Early Dementia by Years

AuroBlog - Aurous Healthcare Clinical Trials blog

Few people had probably heard of frontotemporal dementia until earlier this year, when the family of actor Bruce Willis announced the 68-year-old had been diagnosed with the condition. Frontotemporal dementia is a rare disease – thought to account for only one in every 20 cases of dementia.

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Paper details Astellas gene therapy study that led to patient deaths

Bio Pharma Dive

The company, which is working with regulators to lift a clinical hold, said the treatment could still help people with X-linked myotubular myopathy.

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Kite and Arcellx expand strategic collaboration

Pharmaceutical Technology

Kite has expanded its existing partnership with Arcellx for the latter’s CART-ddBCMA candidate to include lymphomas.

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The New Age of Decentralized Clinical Trials

White paper that delves into the complex topic of Decentralized Clinical Trials and how to master them within the confines of FDA Regulations

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Top FDA official Woodcock to retire early next year

Bio Pharma Dive

The 37-year veteran helmed the agency’s pandemic response, and was a key decision-maker in controversial calls on opioids, an Alzheimer's therapy and muscular dystrophy drugs.

Drugs 161
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State govts should carry forward all projects introduced by Centre to promote Ayurveda: Dr Ramanathan

AuroBlog - Aurous Healthcare Clinical Trials blog

On the occasion of National Ayurveda Day, the Ayurveda Medicine Manufacturers Association of India (AMMOI) has called upon the state governments that the constructive steps being taken by the Union Ayush Ministry to make Ayurveda system of treatment as an alternative treatment method for the world population should be taken forward by all state governments […]

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UK MHRA extends licence for Vertex’s children’s cystic fibrosis drugs

Pharmaceutical Technology

The UK MHRA extended the licence of Vertex Pharmaceuticals’ cystic fibrosis drugs, Kaftrio and Kalydeco for children aged two to five years.

Drugs 130
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Welcome to the Brave New World of CRISPR Gene Editing

BioSpace

The U.K.’s conditional marketing authorization for Vertex Pharmaceuticals and CRISPR Therapeutics’ gene-edited therapy exa-cel raises some potential safety concerns with the risk of off-target effects.

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European Clinical Supply Planning: Balancing Cost, Flexibility and Time

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Certa plans pivotal scleroderma trial after Phase II data readout

Pharmaceutical Technology

Certa plans to discuss trial plans with the FDA in early 2024, with the trial set to begin later in the year.

Trials 147
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AbbVie’s Migraine Drug Ubrelvy Scores Victory in Phase III Study

BioSpace

When given hours ahead of an expected episode, a late-stage study showed the preventive benefits of Ubrelvy in safely reducing moderate or severe headaches within 24 hours of treatment.

Drugs 122
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BMS strengthens NSCLC market share with ROS1 drug Augtyro

Pharmaceutical Technology

Augtyro (repotrectinib) is the only FDA-approved tumour kinase inhibitor (TKI) for ROS1-positive non-small cell lung cancer (NSCLC).

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Bristol Myers' $4.1B Turning Point buy yields FDA approval for lung cancer drug Augtyro

Fierce Pharma

Watch out, Pfizer and Roche. Thanks to an acquisition and now an FDA approval, Bristol Myers Squibb is entering a niche lung cancer market. | Watch out, Pfizer and Roche. Thanks to an acquisition and now an FDA approval, Bristol Myers Squibb is entering a niche lung cancer market.

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The New Age of Decentralized Clinical Trials

This new white paper defines and details the impact of Decentralized Clinical Trials on the Pharmaceutical industry and how the impact can be measured along with steps companies can take to ensure adoption.

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LENZ Therapeutics and Graphite Bio agree to merge

Pharmaceutical Technology

LENZ Therapeutics has signed a definitive agreement with Graphite Bio to merge the two businesses in an all-stock deal.

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Pfizer, Astellas’ Xtandi Gets FDA Approval for Earlier Prostate Cancer Treatment

BioSpace

Xtandi is now approved to treat nonmetastatic castration-sensitive prostate cancer with biochemical recurrence at high risk for metastasis with or without a gonadotropin-releasing hormone analog therapy.

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BT-7480 by Bicycle Therapeutics for Gastric Cancer: Likelihood of Approval

Pharmaceutical Technology

BT-7480 is under clinical development by Bicycle Therapeutics and currently in Phase II for Gastric Cancer.

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Pharma is racing to oncology's first line, causing a rethink of early-stage disease treatment

Fierce Pharma

Timothy Yap, Ph.D., used to see patients nearing the end of their cancer journey in his phase 1 department at the University of Texas' MD Anderson Cancer Center. | Cancer drugs are getting better thanks to innovation, driven by the data and regulatory forces like Project FrontRunner.

Drugs 113
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Unlocking Excellence: How Catalent Is Transforming Japan’s Clinical Research

Planning on running clinical trials in Japan? How can you reliably supply these studies? Discover Catalent’s clinical supply packaging facility in Shiga, Japan. Strategically located between Tokyo and Osaka, and one of largest in Japan, this 6,000 square meter facility offers comprehensive services including primary and secondary clinical packaging and labelling, comparator sourcing, cold chain storage, local and global distribution, local language support and white glove service to support stud

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Astellas agrees to take over Propella Therapeutics for $175m

Pharmaceutical Technology

Astellas Pharma has signed an agreement for the acquisition of Propella Therapeutics in a deal worth $175m.

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Retaining the human element of AI within pharma and healthcare

pharmaphorum

In this episode of the pharmaphorum podcast, web editor Nicole Raleigh speaks with Venu Mallarapu, vice president of global strategy and operations at eClinical Solutions, about the ins and outs of successful AI implementation within pharma and healthcare.

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BT-7480 by Bicycle Therapeutics for Solid Tumor: Likelihood of Approval

Pharmaceutical Technology

BT-7480 is under clinical development by Bicycle Therapeutics and currently in Phase II for Solid Tumor.

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Great Britain first to approve CRISPR-based gene therapy

Drug Discovery World

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy (exagamglogene autotemcel, or exa-cel) is the first medicine to be licensed that uses gene editing tool CRISPR, for which its inventors were awarded the Nobel Prize in 2020.

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How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

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BT-7480 by Bicycle Therapeutics for Solid Tumor: Likelihood of Approval

Pharmaceutical Technology

BT-7480 is under clinical development by Bicycle Therapeutics and currently in Phase II for Solid Tumor.

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FDA Raises Efficacy Concerns About Merck’s Chronic Cough Drug Ahead of Adcomm Meeting

BioSpace

In briefing documents for Friday’s advisory committee meeting, the regulator has expressed concerns about the effectiveness of the company’s P2X3 receptor antagonist gefapixant.

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Glox Therapeutics secures £4.3m seed funding to combat AMR

Pharma Times

The bacteriocin development programme will first target pseudomonas aeruginosa - News - PharmaTimes

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AstraZeneca’s AKT Inhibitor Wins FDA Approval for Combo Breast Cancer Treatment

BioSpace

The company's Truqap, in combination with its endocrine therapy Faslodex, has been approved by the regulator for adult patients with advanced HR-positive breast cancer with specific biomarker alterations.

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Roles and Responsibilities of Specialized Clinical Supply Experts

When selecting a clinical supply provider, consideration often focuses upon the manufacturing, packaging, storage and distribution capabilities available that will, at face-value, be sufficient to meet the needs of the sponsor and their trial. However, there are human-based and knowledge-driven factors that are often overlooked that go beyond these basic physical capabilities and are integral to the development and delivery of high performing clinical supply chains.