FDA approves first gene therapy for hemophilia B
Bio Pharma Dive
NOVEMBER 22, 2022
The treatment, which is for the less common “B” form of the bleeding disorder, will be sold in the U.S. by maker CSL for $3.5 million.
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AI will continue to attract investment in near future in the healthcare industry
Pharmaceutical Technology
NOVEMBER 25, 2022
Artificial intelligence (AI) was seen as one of the top current investment priorities and was thought to continue to attract investment in the healthcare sector in the upcoming two years, according to GlobalData's latest report ‘Digital Transformation and Emerging Technology in the Healthcare Industry - 2022 Edition’. In this survey-based report tracker, digital media was prioritised as a top current investment target, with 53% of surveyed respondents confirming that their companies are currentl
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As biotech retreats, gene therapy companies retrench and redraw plans
Bio Pharma Dive
MARCH 24, 2022
At least nine biotechs working in cell or gene therapy have announced layoffs, cost cuts or restructured their research since December — restructurings that have coincided with a stock market downturn.
2022 in review: Regulation starts to catch up with AI in pharma
Pharmaceutical Technology
DECEMBER 21, 2022
Artificial intelligence (AI) continued to stay in the news with several high-profile deals this year, as the pharmaceutical industry readily took to adopting AI models to improve drug discovery. But as the field grows in leaps and bounds, many authorities have prioritised the release of new guidelines, frameworks, and regulations to keep pace with these advances.
The New Age of Decentralized Clinical Trials
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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.
What to make of Eisai and Biogen’s latest Alzheimer’s drug data
Bio Pharma Dive
NOVEMBER 29, 2022
At a medical conference, the companies detailed clinical trial results that could help support approval of their drug, lecanemab. However, some doctors aren’t yet convinced the medicine’s risks are worth its potential benefits.
In surprise result, Alzheimer’s drug from Eisai, Biogen shows benefit in large trial
Bio Pharma Dive
SEPTEMBER 28, 2022
The companies said the drug, called lecanemab, met all of its goals in the Phase 3 study. The data are a significant finding and provide stronger support for a much-debated hypothesis for treating Alzheimer’s.
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‘One needs to feel safe’: Racism can put sleep — and its health benefits — out of reach
STAT News
DECEMBER 20, 2022
Lots of people struggle to get enough sleep — and the responsibility for fixing the problem tends to fall on the individual. Experts offer advice like reducing screen time, exercising more, or just going to bed earlier in the evening. But many restless nights can’t be solved with blackout curtains, ear plugs, or other typical suggestions.
Developing a digital marker for coronary artery disease
Medical Xpress
DECEMBER 20, 2022
Using machine learning and clinical data from electronic health records, researchers at the Icahn School of Medicine at Mount Sinai in New York constructed an in silico, or computer-derived, marker for coronary artery disease (CAD) to better measure clinically important characterizations of the disease.
UK digital health project aims to tackle dysfunctional breathing
pharmaphorum
DECEMBER 19, 2022
A collaboration between academic centres in the UK has won government funding for a digital approach to dysfunctional breathing or dyspnoea, a symptom that affects around 10% of the population. The Engineering and Physical Sciences Research Council (EPSRC) has set aside £400,000 (almost $490,000) for the project, which will help fund work at the Universities of Plymouth, Salford and Stirling, and the Glasgow School of Art.
Long-COVID personalized medicine enters Phase 2 trial
BioPharma Reporter
NOVEMBER 22, 2022
GeNeuro has recruited the first patients in a Phase 2 trial evaluating temelimab against long-COVID: assessing the efficacy of the treatment for improving cognitive impairment and fatigue.
Commercial Drug Sourcing for Clinical Trials
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The sourcing of commercial drugs for use in clinical trials either as a comparator, rescue or co-medication can be a complex process. Sponsors can face a number of challenges when trying to obtain these critical supplies for their studies, including addressing product lead times, availability, expiry limitations, safety and cost, among others. Choosing the right partner with global sourcing, regulatory, quality and clinical packaging expertise can provide the necessary guidance to help sponsors
Bridging the Translational Gap between Alzheimer’s Advances and Patients
BioSpace
OCTOBER 26, 2022
With diagnostics for Alzheimer's already approved and therapeutics in late-stage clinical trials, the next hurdle is to translate these advances into clinical practice. The Davos Alzheimer's Collaborative is working on it.
A Cancer Trial’s Unexpected Result: Remission in Every Patient
NY Times
JUNE 5, 2022
The study was small, and experts say it needs to be replicated. But for 18 people with rectal cancer, the outcome led to “happy tears.”.
AstraZeneca withdraws US COVID vaccine application, shifts focus to antibody treatments
Fierce Pharma
NOVEMBER 10, 2022
AstraZeneca withdraws US COVID vaccine application, shifts focus to antibody treatments. aliu. Thu, 11/10/2022 - 09:02.
Early cell therapy successes start to turn the tide in lupus
Pharmaceutical Technology
NOVEMBER 14, 2022
In a field dominated by antibodies and small molecules, two cell-therapy based approaches have come under the spotlight for showing early signs of efficacy in treating lupus. In September, a group from Friedrich Alexander University Erlangen-Nuremberg reported that five patients with lupus achieved remission after an infusion of autologous chimeric antigen receptor (CAR)-T cells led to a deep depletion of B cells.
How Machine Learning Drives Clinical Trial Efficiency
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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.
Building a biotech in a downturn: 3 lessons from VCs and startup CEOs
Bio Pharma Dive
NOVEMBER 3, 2022
In a panel discussion hosted by BioPharma Dive, venture capitalists and CEOs discussed how startups can navigate a challenging market as well as possible ripple effects from the new U.S. drug pricing law.
ALS drug approved by FDA in closely watched decision, marking win for patients, developer
Bio Pharma Dive
SEPTEMBER 30, 2022
The drug, which will be sold as Relyvrio, showed modest benefits in function and survival in testing. It also became the latest test of the FDA's flexibility toward new therapies for neurological disorders.
Bluebird gene therapy approved by FDA for rare blood disease
Bio Pharma Dive
AUGUST 17, 2022
The regulator cleared the biotech’s medicine Zynteglo for transfusion-dependent beta thalassemia, giving patients a powerful new treatment option. But it will come at a very high cost of $2.8 million in the U.S.
‘In vivo’ cell therapy: expanding beyond CAR-T
Bio Pharma Dive
OCTOBER 18, 2022
At least five startups have emerged with new ways to genetically modify immune cells within the body, an approach that, if successful, could widen the field of CAR-T treatment.
What the FDA's New Dosage Guidance Means for the Future of Clinical Research
Speaker: Dr. Ben Locwin - Biopharmaceutical Executive & Healthcare Futurist
What will the future hold for clinical research? A recent draft from the FDA provides valuable insight. In "Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases," the FDA notes that "targeted therapies demonstrate different dose-response relationships compared to cytotoxic chemotherapy, such that doses below the Maximum Tolerated Dose (MTD) may have similar efficacy to the MTD but with fewer toxicities.
Alnylam drug succeeds in key heart disease study, boosting company
Bio Pharma Dive
AUGUST 3, 2022
The success of Alnylam's treatment in a trial known as APOLLO-B could pave the way for the biotech to reach consistent profitability, should the FDA sign off on a planned approval application.
CAR-T pioneer Carl June on founding biotech startups and cell therapy’s next act
Bio Pharma Dive
OCTOBER 18, 2022
The University of Pennsylvania immunologist and inventor of Kymriah spoke with BioPharma Dive about working with pharma, starting companies and the future of the cell therapy field.
A Versant-backed biotech emerges to take on ‘overlooked’ cancer targets
Bio Pharma Dive
OCTOBER 6, 2022
Nested Therapeutics touts a deep bench of scientific leaders and advisers, including Kevan Shokat, whose work drugging KRAS — a cancer-related gene once thought to be undruggable — helped lead to the development of Amgen’s Lumakras.
Centerview grows role as go-to adviser for biopharma dealmaking
Bio Pharma Dive
NOVEMBER 8, 2022
Over the past several years, the investment firm has served as a financial adviser on biopharma acquisitions worth more than $200 billion in total, with the latest announced Monday.
2022 Research: The Rapid Rise of Ocean Freight Visibility
Advertiser: FourKites
A research study conducted by The Journal of Commerce and FourKites surveyed hundreds of international shippers, exploring how their usage of global supply chain visibility technology has evolved since the onset of global disruptions caused by COVID-19. For international shippers, ocean freight visibility has evolved from optional to essential and satisfaction with visibility varies greatly depending on how it is obtained and delivered.
Pfizer study results show Paxlovid benefit less clear in lower-risk patients
Bio Pharma Dive
JUNE 15, 2022
A closely watched study missed its goal, failing to prove the antiviral pill’s benefit in a broader population than the high-risk individuals for which it’s currently cleared.
Ferring wins FDA approval for bladder cancer gene therapy
Bio Pharma Dive
DECEMBER 19, 2022
The OK caps a long development journey for the treatment, which was previously turned back by the agency. Ferring doesn’t expect it to be available until the second half of next year, however.
'The music stopped': Biotech rout leaves drug startups grounded as demand slumps for IPOs
Bio Pharma Dive
FEBRUARY 6, 2022
For the first time in years, biotechs no longer have an easy path onto Wall Street, a market reversal that could change what the next generation of young drugmakers looks like.
Tapping into pharma’s growing opportunities in digital health
Pharmaceutical Technology
AUGUST 4, 2022
Soaring patient numbers are stretching hospital capacity across the globe, forcing healthcare providers and their partners to think laterally about how to meet the demand without doubling their resources. Smarter diagnostic tools and more sophisticated remote- and self-care models will have an increasingly important role to play, as long as any advances are seen to deliver excellent outcomes and a better patient experience.
An Innovative & Creative Problem Solver Approach to Selling in the Medical Device Space
Speaker: Steve Goldstein, Sales Leader
Are you currently in sales, or involved in a business that depends on strong sales results? What about the extremely competitive world of medical device sales? What are some of the top challenges your customers face and how do you approach understanding what’s most important to them? Join Steve Goldstein, Sales Success Coach, Motivational Speaker and Medical Device Sales Leader from Gold Selling LLC., to discover critical strategies and approaches you can take to engage your customers, achieve g
Sangamo presses ahead with Fabry disease gene therapy
Bio Pharma Dive
SEPTEMBER 2, 2022
Preliminary results from a Phase 1 study show Sangamo’s treatment to be safe and suggest it is working as intended, leading the biotech to move into the trial’s next phase.
Moderna and UK government sign deal to establish mRNA facility
Pharmaceutical Technology
DECEMBER 21, 2022
Moderna and the UK government have entered a ten-year strategic collaboration to build a messenger ribonucleic acid (mRNA) research, development and manufacturing facility in the country. The latest development comes after the parties announced an agreement in principle in June this year. This Moderna Innovation and Technology Centre (MITC) is expected to offer access to a locally produced future mRNA vaccine portfolio against respiratory viruses, subject to regulatory evaluation and licensure.
AstraZeneca to acquire gene editing biotech LogicBio
Bio Pharma Dive
OCTOBER 3, 2022
Shares in LogicBio had lost nearly all their value since a 2018 IPO, bringing the company to the brink of Nasdaq delisting. The buyout deal represents a premium of over 600%.
Prime Medicine raises $175M in one of the year’s largest biotech IPOs
Bio Pharma Dive
OCTOBER 20, 2022
The gene editing startup is one of less than two dozen biotech companies to debut on Wall Street this year, as the pace of IPOs remains far off last year’s trajectory.
Making the Case for Case Management for Cell and Gene Therapies
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As demand for advanced therapies increases, so does the need for more specialized supply chain support, as these products have strict transportation and handling requirements. Deviations from the intended supply chain can delay timelines and affect these high value products, which can be detrimental to budgets, but more importantly can have dire effects on the patients whose lives depend on receiving these critical doses safely and on time.
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