Patient dies in Pfizer study of Duchenne gene therapy
Bio Pharma Dive
MAY 7, 2024
Pfizer said the patient, a young boy who was treated earlier last year, had died suddenly. The company is working with trial researchers to investigate further.
Tue.May 07, 2024
356 
AstraZeneca concludes equity investment in Cellectis
Pharmaceutical Technology
MAY 7, 2024
AstraZeneca has concluded an equity investment in clinical-stage biotechnology company Cellectis following a deal signed in November 2023.
Join 32,000+
Insiders
Sign Up for our Newsletter
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
A Healthy Lifestyle Could Offset Genetic Risk For Early Death by a Hopeful 62%
AuroBlog - Aurous Healthcare Clinical Trials blog
MAY 7, 2024
When research tells us that our grandparents’ diets can affect our health decades later, it can be hard to shake the feeling that genetics spells out the trajectory of our wellbeing. But how much of our health is actually determined by our genes, and what effect does our lifestyle have, for better or worse?
Psilocybin effective in treating depression
Pharmaceutical Technology
MAY 7, 2024
A new study has further supported psilocybin's potential to treat depression, which impacts around 300 million people globally.
CDSCO centralises powers to issue NOC for unapproved, banned & new drugs solely for exports
AuroBlog - Aurous Healthcare Clinical Trials blog
MAY 7, 2024
The national drug regulator has withdrawn the powers delegated to the State and Union Territory (UT) Licensing Authorities to issue No Objection Certificates (NOCs) for manufacture of unapproved, banned or new drugs solely for export purpose, asking the industry to file fresh applications with the central authority online from May 15.
Zenas, with new funding, aims dual-targeting antibody at lupus and MS
Bio Pharma Dive
MAY 7, 2024
The $200 million round is the latest evidence that surging interest in autoimmune disease cell therapies could expand to include developers of bispecific antibodies, too.
Sign up to get articles personalized to your interests!
Clinical Research Informer brings together the best content for clinical researchers from the widest variety of industry thought leaders.
More Trending
Prologue, Flagship’s newest startup, looks to mine viruses for new drugs
Bio Pharma Dive
MAY 7, 2024
The company is using what it claims is the largest database of viral protein structures to unearth medicines for a range of diseases, said CEO and Flagship origination partner Lovisa Afzelius.
Gossamer Bio and Chiesi partner for pulmonary hypertension treatment
Pharmaceutical Technology
MAY 7, 2024
Gossamer Bio and Chiesi signed a partnership and licence agreement aimed at developing and marketing seralutinib for pulmonary hypertension.
FDA sets date for advisory meeting on Lilly’s closely watched Alzheimer’s drug
Bio Pharma Dive
MAY 7, 2024
After delaying an approval decision for Lilly’s donanemab, the agency now intends to hold an advisory committee meeting on June 10.
Vertex reports net profits of $2.69bn in Q1 2024
Pharmaceutical Technology
MAY 7, 2024
The increase in revenue was primarily driven by strong product sales of the cystic fibrosis drug Trikafta in ex-US markets.
The New Age of Decentralized Clinical Trials
Advertisement
White paper that delves into the complex topic of Decentralized Clinical Trials and how to master them within the confines of FDA Regulations
Verve moves forward with backup base editing therapy
Bio Pharma Dive
MAY 7, 2024
Weeks after a safety setback derailed its lead candidate, the company has treated the first participant in a trial of its heart disease treatment Verve-102.
China’s NMPA grants breakthrough therapy status for Innovent’s IBI343
Pharmaceutical Technology
MAY 7, 2024
The CDE of China's NMPA has granted breakthrough therapy designation (BTD) for Innovent Biologics’ IBI343 monotherapy.
Life sciences in Ireland: Nurturing talent, innovating excellence
pharmaphorum
MAY 7, 2024
The life sciences sector in Ireland is booming thanks to nurturing talent and fostering innovation. Companies like Eli Lilly and Regeneron have major operations driving excellence.
BioNTech reports net loss of €315.1m in Q1 2024
Pharmaceutical Technology
MAY 7, 2024
BioNTech experienced a significant shift in its financial standing, reporting a net loss of €315.1m ($339.3m) in Q1 2024.
Unlocking Excellence: How Catalent Is Transforming Japan’s Clinical Research
Advertisement
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
Kenvue maps out hundreds of layoffs as service pact with J&J winds down
Fierce Pharma
MAY 7, 2024
Even after consumer outfit Kenvue split from Johnson & Johnson one year ago, the company has been performing certain services for the pharma and medtech giant as part of a transition process. | Kenvue’s board of directors on Monday approved a plan to cut 4% of the company’s global workforce. The J&J spinoff employed about 23,000 staffers at the end of last year, so the layoff initiative could affect some 920 workers.
Broken String Biosciences teams up with Francis Crick Institute for ALS research
Pharmaceutical Technology
MAY 7, 2024
The companies will utilise Broken String’s DNA break mapping platform Induce-seq for ALS research.
Generative AI Finds Its Footing in Drug Development
BioSpace
MAY 7, 2024
Since the 2022 launch of ChatGPT, biopharma has poured money into this new form of artificial intelligence, but companies remain cautious with unproven technology.
Sorcero genAI conjures plain language from scientific papers
pharmaphorum
MAY 7, 2024
Understanding complex scientific studies is often a challenge for patients as well as healthcare professionals, but one that may become easier thanks to artificial intelligence.
The New Age of Decentralized Clinical Trials
Advertisement
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.
AAV Manufacturing Takes Center Stage at ASGCT24
BioSpace
MAY 7, 2024
Tuesday afternoon’s session was standing room only as representatives from various biopharma companies presented on their work to improve the efficiency and quality of AAV production.
Antidote to be announced as Corporate Honoree at MSAA Improving Lives Benefit
Antidote
MAY 7, 2024
We are thrilled to be honored as the Multiple Sclerosis Association of America’s Corporate Honoree at their 10th annual Improving Lives Benefit on May 15! This inspiring event supports MSAA’s free programs and services and honors key players in the MS research space.
Vertex Touts 13% Revenue Jump, Beats Profit Estimates on Strong Cystic Fibrosis Performance
BioSpace
MAY 7, 2024
With a total revenue of nearly $2.7 billion, Vertex exceeded analyst estimates in the first quarter, bolstered by strong uptake of its cystic fibrosis products.
NHS to roll out fibre optic laser therapy to prevent seizures in epileptic patients
Pharma Times
MAY 7, 2024
The chronic brain disease that causes seizures currently affects around 600,000 people living in the UK
Roles and Responsibilities of Specialized Clinical Supply Experts
Advertisement
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.
Sandoz delivers 6% sales growth with Humira biosimilar leading the charge
Fierce Pharma
MAY 7, 2024
In its opening months, after spinning off as a separate business from Novartis, Sandoz is riding the wave of a strong launch of its biosimilar version of AbbVie’s Humira (adalimumab). | After spinning off as a separate business from Novartis, Sandoz is riding the wave of a strong launch of its biosimilar version of AbbVie’s Humira (adalimumab). With Hyrimoz leading the way, sales of the company’s biosimilars reached $623 million in the first three months of this year.
Pfizer pauses DMD gene therapy trial after boy’s death
pharmaphorum
MAY 7, 2024
Pfizer has paused dosing in a study of its Duchenne muscular dystrophy gene therapy after a sudden patient death in another trial
Vertex's Casgevy launch progresses as CF business posts impressive growth
Fierce Pharma
MAY 7, 2024
While cystic fibrosis has long been Vertex Pharmaceuticals’ bread and butter, the company has been eager to diversify its revenue base in recent years. | As of mid-April, Vertex said it had collected 5 patients' cells for eventual treatment with sickle cell disease therapy Casgevy. That puts Vertex ahead of its gene therapy rival bluebird bio, which recently said it had collected just one patient's cells for its own SCD treatment Lyfgenia.
ADC Therapeutics Posts Early Phase II Lymphoma Data for Zynlonta as Revenue Drops 5.8%
BioSpace
MAY 7, 2024
ADC Therapeutics is positioning Zynlonta for a label expansion with new Phase II data showing that the treatment can elicit high rates of complete response in patients with relapsed or refractory marginal zone lymphoma.
How Machine Learning Drives Clinical Trial Efficiency
Advertisement
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.
eyeDNA plans pivotal trial of retinitis pigmentosa therapy
pharmaphorum
MAY 7, 2024
Two-year data from a phase 1/2 trial of eyeDNA Therapeutics' gene therapy for retinitis pigmentosa sets up pivotal trials, says the new biotech.
Mapping the Vertical Integration of Insurers, PBMs, Specialty Pharmacies, and Providers: A May 2024 Update
Drug Channels
MAY 7, 2024
It's time for Drug Channels ’ annual update of vertical integration among insurers, PBMs, specialty pharmacies, and providers within U.S. drug channels. Below you’ll find our latest illustration of the major vertical business relationships among the largest companies along with some of the notable activity since our previous update. These organizations continue to exert greater control over patient access, sites of care/dispensing, and pricing, although some have started to unwind their vert
Francis Crick Institute and Broken String Biosciences partner for ALS research
Pharma Times
MAY 7, 2024
The rare and fatal progressive neurodegenerative disease is estimated to affect 5,000 people in the UK
Krystal Biotech touts topical gene therapy uptake, plots overseas launches
Fierce Pharma
MAY 7, 2024
After a 2023 approval from the FDA, Krystal Biotech has collected more than $95 million from its launch of Vyjuvek, the first treatment for the rare skin disease dystrophic epidermolysis bullo | The topical gene therapy has pulled in nearly $100 million during its first three quarters on the U.S. market. Meanwhile, Vyjuvek is currently under review in Europe, and a filing in Japan is planned for later this year.
Accelerating Clinical Supply Through Integrated Drug Development
Advertisement
As the development pipeline for new drugs continues to grow, biopharmaceutical companies are re-evaluating how to best manage and balance resources across an increasing number of development projects and complex clinical trials. There are two approaches that can be used to speed a drug from development to clinic faster: timeline compression and parallel processing, but only one that considers the benefits of integrating clinical supply into the overall drug development process.
Let's personalize your content