Drug Discovery World

JANUARY 9, 2024

Thirteen new-to-market therapeutics and drugs poised to launch in 2024 will achieve ‘blockbuster’ status by 2029 or deliver game-changing benefits to patients. The 2024 Drugs to Watch, are: 1. The therapy has shown positive results in Phase III trials in both breast and lung cancer.

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Drug Discovery World

JANUARY 30, 2024

Viral vector manufacturing At the Advanced Therapies Awards ceremony, Dr Luigi Naldini, Director of the San Raffaele Telethon Institute for Gene Therapy, was awarded a Lifetime Achievement Award in recognition of ‘extraordinary contributions to gene therapy’, a field he has helped shape through his pioneering work on lentiviral vectors.

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FDA Law Blog: Drug Discovery

MAY 19, 2024

By Sarah Wicks — On May 21, 2024, the EveryLife Foundation for Rare Diseases (ELF) will host a Scientific Workshop at the National Press Club in Washington, D.C. aimed at identifying and characterizing the challenges in developing therapies for ultra-rare diseases and conditions that affect exceedingly small populations.

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Drug Discovery World

APRIL 23, 2024

Over the past 20 years research has increasingly linked concussions to neurogenerative conditions, including dementia, Alzheimer’s disease and CTE, a degenerative brain disease. While those therapies provide some relief to concussed patients, 20% or more continue to have persistent symptoms for weeks to months to years after injury.

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Drug Discovery World

OCTOBER 3, 2024

The US Food and Drug Administration (FDA) has announced seven new clinical trial grants awarded under the Orphan Products Grants Program for clinical trials to address the unmet needs of people affected by rare diseases. Targeted Therapy Technologies received $2.6 In total, clinical researchers will receive approximately $17.2

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Drug Discovery World

FEBRUARY 28, 2024

Research into new cancer drugs remain strong. A Phase I clinical trial, sponsored and managed by Cancer Research UK’s Centre for Drug Development, tested whether a type of antibody called IgE could be used to treat cancer. She has over 25 years’ experience in B2B journalism, ten of those being in life sciences.

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Drug Discovery World

JANUARY 19, 2024

BPGbio, an AI-powered biopharma that focuses on oncology, neurology, and rare diseases, has announced new data from a Phase IIa clinical trial of its lead candidate, BPM31510, in patients with advanced pancreatic cancers at the annual ASCO Gastrointestinal Cancers Symposium, being held 18-20 January 2024, in San Francisco.

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KIF1A

MARCH 2, 2024

#ScienceSaturday posts share exciting scientific developments and educational resources with the KAND community. Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Want to learn more about translational research?

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LifeSciVC

JANUARY 16, 2024

These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. recent announcements from Merck and Sanofi for obesity and broader metabolic disease).

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Drug Target Review

JULY 18, 2024

Why is detecting cancers via liquid biopsy before they become visible on imaging important for drug development, not just diagnostics? We believe liquid biopsy is very helpful for oncology drug development because the reduction and clearance of circulating DNA occurs quickly before radiographic imaging is available.

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Alta Sciences

AUGUST 29, 2024

Engaging at the outset with a fully integrated and experienced drug development partner can ensure safety, with timely data sharing at every step of the drug development plan, and facilitate agile, flexible decision-making and planning.

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Alta Sciences

JULY 25, 2024

Top 10 Life Science Resources for Summer 2024 pmjackson Thu, 07/25/2024 - 14:44 There’s a lot of life science content out there, which is why we’ve curated a selection of our expert insights, tips, case studies, and scientific and regulatory information for your convenience. Catch up on what you might have missed below! Read the eBook.

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Drug Target Review

SEPTEMBER 25, 2024

While these approaches often produce encouraging initial results, the development of drug resistance remains a major obstacle for long-term patient survival. Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades.

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PPD

NOVEMBER 30, 2023

As we approach the end of 2023, it’s time to reflect on the past 12 months and how advances in drug development shaped the pharma and biotech industries. These shifts are a prelude to further change and progress in the clinical trial landscape in 2024. Five Predictions for the Drug Development Industry in 2024 1.

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Drug Discovery World

FEBRUARY 13, 2024

Often this can be drug approvals, exciting research into hard-to-treat indications, or even regulatory updates that enable players within the sector to utilise the latest technologies and drive therapies to market faster, ultimately benefitting patients. Progress isn’t stagnant though.

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Alta Sciences

JANUARY 11, 2024

It is estimated that there are over 6,000 monogenic diseases, affecting over 350 million people worldwide; for these diseases, cell and gene therapy may provide hope for a cure. However, there are significant challenges associated with the successful development of these complex, leading-edge therapies.

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Agency IQ

AUGUST 2, 2024

What We Expect the FDA to do in August and September 2024 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the months ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

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Drug Discovery World

AUGUST 14, 2024

Even with an approved therapy or a late-stage clinical trial, it’s still not a given that patients or doctors will have access to comprehensive and cost-effective genetic testing. DS: What is needed, in your view, to enable successful recruitment for clinical trials for personalised drugs?

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Drug Discovery World

JULY 23, 2024

Dr Daniel Turner, Chief Scientific Officer, Enhanc3D Genomics, outlines how 3D genomics offers a radically novel approach to address challenges in drug target discovery and how it can affect change in drug target identification, cell therapy and personalised medicine.

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PPD

JANUARY 5, 2023

In an industry survey led by PPD, Thermo Fisher Scientific’s clinical research business, rare diseases emerged as a top therapeutic area in drug developers’ pipelines, with 39% of global biotech and biopharma leaders pursuing therapies in the broad rare disease space.

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Agency IQ

MAY 3, 2024

What We Expect the FDA to do in May and June 2024 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the months ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

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Drug Discovery World

MAY 6, 2024

Even before this, however, Amsterdam was established as an important centre for drug development. The company is now investigating a promising gene therapy for Huntingdon’s disease in a Phase I/II trial, with patients already showing signs of preserved neurological function.

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Alta Sciences

SEPTEMBER 27, 2024

The growing understanding of the immune system's role in various diseases, along with advancements in drug development, have led to a natural evolution from traditional vaccines, small molecules , and cytokines , to increased targeted biologics and innovative approaches in gene therapy and vaccines.

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Drug Discovery World

JUNE 7, 2023

Panellists will discuss the following questions: Where will the opportunities be in 2024? How important will mRNA-based vaccines be in treating viruses and diseases such as cancer? What can we expect from the cell & gene therapy market? Is it true that the next era will belong to antibody drug conjugates?

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Drug Discovery World

JUNE 27, 2023

Panelists will discuss the following questions: Where will the opportunities be in 2024? How important will mRNA-based vaccines be in treating viruses and diseases such as cancer? What can we expect from the cell & gene therapy market? Is it true that the next era will belong to antibody drug conjugates?

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Drug Discovery World

JUNE 22, 2023

Panelists will discuss the following questions: Where will the opportunities be in 2024? How important will mRNA-based vaccines be in treating viruses and diseases such as cancer? What can we expect from the cell & gene therapy market? Is it true that the next era will belong to antibody drug conjugates?

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Agency IQ

JULY 8, 2024

What we expect European regulators to do in July 2024 In this recurring feature, AgencyIQ, through public data and previous analysis, determines what European medicine and device regulators will likely do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods, and more. and national regulators.

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Drug Discovery World

JUNE 15, 2023

Panelists will discuss the following questions: Where will the opportunities be in 2024? How important will mRNA-based vaccines be in treating viruses and diseases such as cancer? What can we expect from the cell & gene therapy market? Is it true that the next era will belong to antibody drug conjugates?

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Drug Discovery World

JUNE 8, 2023

Panelists will discuss the following questions: Where will the opportunities be in 2024? How important will mRNA-based vaccines be in treating viruses and diseases such as cancer? What can we expect from the cell & gene therapy market? Is it true that the next era will belong to antibody drug conjugates?

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Drug Discovery World

JANUARY 22, 2024

It states: ” An integrated appreciation of the physiological consequences of DUB modulation in relevant cancer models will eventually lead to the identification of patient populations that will most likely benefit from DUB-targeted therapies” 1. appeared first on Drug Discovery World (DDW).

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Drug Discovery World

DECEMBER 26, 2023

Otsuka Pharmaceutical and Ionis Pharmaceuticals Otsuka Pharmaceutical and RNA therapy company Ionis have entered into a licence agreement for Otsuka to acquire exclusive marketing rights to Ionis’ hereditary angioedema (HAE) drug candidate donidalorsen in Europe.

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Agency IQ

FEBRUARY 12, 2024

BY RACHEL COE, MSC | FEB 7, 2024 10:21 PM CST The Bespoke Gene Therapy Consortium A collaborative effort, the Bespoke Gene Therapy Consortium (BGTC) was launched in October 2021 to “accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease.”

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Agency IQ

MARCH 1, 2024

What We Expect the FDA to do in March and April 2024 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the months ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

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Agency IQ

FEBRUARY 2, 2024

What We Expect the FDA to do in February and March 2024 (Updated) In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the months ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

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Agency IQ

JUNE 26, 2023

FDA quietly kicks off process that could inform future rare disease regulatory reforms Reforms buried within the FY2023 omnibus spending bill require the development of a new report looking at the regulatory processes of the U.S. Meanwhile, in the EU rare diseases are defined using a different set of criteria. In the U.S.,

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PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Muscle makes up about 40 percent of body weight. million DNA bases.

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Conversations in Drug Development Trends

NOVEMBER 27, 2023

Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel. In recognition of the unmet need and medical urgency for innovative therapies in the sickle cell space, the FDA granted exa-cel Priority Review, with a formal decision expected by December 8, 2023.

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