Drug Discovery World

APRIL 15, 2024

With clinical trials in North America and Europe for the treatment of Parkinson’s disease via the injection of dopaminergic neurons, the first patient data from Vertex on the delivery of beta-islet cells and continued progress in the eye, we can expect to see this grow in 2024.

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Drug Discovery World

OCTOBER 3, 2023

The US Food and Drug Administration (FDA) has announced a pilot programme with the aim of accelerating the development of novel drug and biological products for rare diseases. The FDA will be accepting applications to the START programme between 2 January 2024 and 1 March 2024.

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Drug Discovery World

MAY 2, 2024

Hailed as a revolution in the treatment of many diseases, cell and gene therapy (CGT) is the fastest growing area of therapeutics. 2023 was a record year for CGTs, with seven new CGTs approved by the US Food and Drug Administration (FDA) across a variety of different disease areas.

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Drug Discovery World

FEBRUARY 19, 2024

However, despite these challenges, there are some positive developments; for instance, potential signs of improvement in the financial situation were flagged in late 2023 with AstraZeneca’s acquisition of cell therapy company Gracell for $1.2bn. antibody-drug conjugate [ADC] pipelines in some oncology settings).

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Drug Discovery World

MARCH 18, 2024

Following the recent AD/PD 2024 conference, which took place on 5-9 March in Lisbon, Portugal, there have been a number of key announcements regarding investigational therapies for neurological disorders, particularly Alzheimer’s disease and Parkinson’s disease.

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Drug Discovery World

MARCH 18, 2024

Following the recent AD/PD 2024 conference, which took place on 5-9 March in Lisbon, Portugal, there have been a number of key announcements regarding investigational therapies for neurological disorders, particularly Alzheimer’s disease and Parkinson’s disease.

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Drug Discovery World

MAY 21, 2024

From a weight loss drug that prevents heart attacks and a gene therapy that restores hearing, to a vaccine that can treat viruses that don’t exist yet, our chosen news stories this week all represent potential breakthroughs in their respective fields.

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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

FEBRUARY 21, 2024

DDW Editor Reece Armstrong speaks to Dr Paul Song , MD, NKGen and Dr Vince DeOrchis , clinician, to find out more about the investigational natural killer therapy SNK01 and the benefit of using a patient’s own cells to treat Alzheimer’s. PS : Most, if not all, current therapies are focused on MCI. RA: How does SNK01 work?

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Drug Discovery World

MAY 8, 2024

Whether it is successful seed funding or dedicated investment, this money enables scientists to produce top-quality research which will in turn result in discoveries and drugs that help tackle disease and areas of unmet need. The post New DDW Highlights episode: 8 May 2024 appeared first on Drug Discovery World (DDW).

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Drug Discovery World

NOVEMBER 8, 2023

The first set of speakers for Rejuvenation Startup Summit 2024 (Berlin, 10-11 May 2024) has been announced. The networking event aims to bring together startups and members of the longevity venture capital/investor ecosystem to create therapies to vastly extend the healthy human lifespan.

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Drug Discovery World

APRIL 12, 2024

The US Food and Drug Administration (FDA) has accepted an Investigational New Drug (IND) application for Tr1X’s TRX103 for the prevention of Graft versus Host Disease (GvHD) in patients undergoing HLA-mismatched haematopoietic stem cell transplantation (HSCT). “As a result, innovative treatments are urgently needed.”

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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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Drug Discovery World

JANUARY 15, 2024

Perhaps most importantly, a new antibiotic was found that targets a Gram-negative pathogen, mRNA was used as an effective therapy for a rare liver disease, and a common immune cell was found to attack cancer. The post DDW Highlights: 15 January 2024 appeared first on Drug Discovery World (DDW).

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Drug Discovery World

JANUARY 15, 2024

Perhaps most importantly, a new antibiotic was found that targets a Gram-negative pathogen, mRNA was used as an effective therapy for a rare liver disease, and a common immune cell was found to attack cancer. The post New DDW Highlights podcast: 15 January 2024 appeared first on Drug Discovery World (DDW).

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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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Fierce BioTech

MAY 14, 2024

Strategies for Expanding Access to Rare Disease Therapies Join us as we explore strategic approaches employed by industry leaders, who are bridging the diagnostic gap to improve patient outcomes and accelerate the development of precision therapies.

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KIF1A

FEBRUARY 10, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. This highlights the heterogeneity of KAND, and the importance of identifying KIF1A mutations in other rare disease groups.

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Vial

DECEMBER 8, 2023

With technological innovation and scientific research propelling the development of new therapies, anticipation for the future is high. As we look forward to 2024, several groundbreaking therapies are poised to radically change the landscape of ophthalmology, providing hope and improved treatment options for patients with ocular conditions.

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Drug Discovery World

FEBRUARY 16, 2024

The first programme, focused on RNAi therapies to tackle highly genetically mediated cancers, has been awarded £835,000 by Innovate UK and Innosuisse to develop a new RNAi therapy for acute myeloid leukaemia. in a full seed funding round in the second quarter of 2024. The post Belfast techbio raises £1.4m

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Drugs.com

JUNE 18, 2024

TUESDAY, June 18, 2024 -- There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.“It’s It’s encouraging that this gene-editing treatment con.

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Fierce BioTech

MAY 13, 2024

In this panel discussion, rare disease experts discuss the significant challenges with achieving a timely and accurate rare disease diagnosis and strategies to engage both patients and providers to expand understanding of and access to therapies that benefit patients. On Demand Start Date Tue, 06/18/2024 - 14:00

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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

MAY 15, 2024

Dr Catherine Beech , CEO at Exonate and Dr Pete Adamson , CSO at Breye Therapeutics discuss the need for better therapies to treat diabetic retinal disease and current progress in the field. Specific numerical values (steps) denote the severity of NPDR, with 47-53 describing the moderately severe disease.

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Drug Discovery World

DECEMBER 8, 2023

ONK Therapeutics and NAYA Biosciences have agreed to develop a combination therapy of ONK’s optimally-engineered natural killer (NK) cell therapies and NAYA’s FLEX-NK bispecific antibodies. The post Partners explore NK cell therapy and bispecific antibodies combination appeared first on Drug Discovery World (DDW).

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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). Join the club.

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KIF1A

MARCH 2, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Animal models have given researchers profound insights into disease, assisting in the creation and testing of new therapies that have changed lives across the world.

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Drug Discovery World

MARCH 15, 2024

Biotechnology company Allyx Therapeutics has reported positive Phase Ib clinical data for its lead compound, ALX-001, a first-in-class, synapse-targeted, disease-modifying oral therapy in development for neurodegenerative diseases. The findings were presented at the AD/PD 2024 Conference in Lisbon.

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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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Drug Discovery World

JANUARY 23, 2024

Breast cancer was the most studied disease in 2023 for the third consecutive year, followed by solid tumours, stroke, Covid-19 and prostate cancer. Meanwhile, we have seen a marked reduction in investment into Covid-19 therapies in 2023, likely due to there being fewer available patients. Read the full report.

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Drug Discovery World

AUGUST 29, 2023

A stem cell derived investigational therapy for treating Parkinson’s disease has shown promise in early clinical trials. The data were presented at the International Congress of Parkinson’s Disease and Movement Disorders in Copenhagen, Denmark.

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Drug Discovery World

SEPTEMBER 28, 2023

We are currently preparing a marketing authorisation application for Kinselby in the EU, which remains on track for submission in Q1 2024.” CTCL is a rare disease with approximately 5,000 patients being newly diagnosed in Europe each year. Currently, CTCL is incurable and treatment options for advanced-stage CTCL are limited.

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KIF1A

MARCH 9, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. During her undergraduate studies, she worked on heart disease research in Chi Kueng Lam’s laboratory at the University of Delaware. Meet our Guest Bloggers!

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Drug Discovery World

FEBRUARY 15, 2024

DDW Editor Reece Armstrong speaks to Tracy M Woody , President and Chief Executive Officer of Cessation Therapeutics, about the company’s goal to develop an antibody therapy to prevent fentanyl overdoses. The American Medical Association (AMA) acknowledged that alcohol and drug dependence are diseases, a quarter of a century ago.

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Drug Target Review

JANUARY 23, 2024

What innovative approaches and technologies are anticipated to reshape immuno-oncology treatments in 2024? In April 2023, we saw the first randomised data applying an individualised vaccine in combination with checkpoint inhibition for resectable melanoma, demonstrating benefit in terms of time to disease recurrence.

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Drug Discovery World

MARCH 2, 2023

With our pre|CISION and Affimer platforms we have an unparalleled opportunity to develop really meaningful new cancer therapies, by developing targeted therapies, multi-specific immunotherapies, difficult to drug targets, and next-generation cell therapies.”

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Broad Institute

APRIL 2, 2024

Scientists link certain gut bacteria to lower heart disease risk By Allessandra DiCorato April 2, 2024 Breadcrumb Home Scientists link certain gut bacteria to lower heart disease risk Study finds several species of cholesterol-metabolizing bacteria in people with lower cholesterol levels. Online April 2, 2024.

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