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

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 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 DDW Highlights: 8 May 2024 appeared first on Drug Discovery World (DDW).

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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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Broad Institute

JUNE 27, 2024

By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. CHARMs are an elegant solution to the problem of silencing disease genes, and they have the potential to have an important position in the future of genetic medicines.”

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Drug Discovery World

MARCH 27, 2024

The vaccines currently in late-stage development hold promise in tackling the disease burden of tuberculosis (TB), says data and analytics company GlobalData on World Tuberculosis Day 2024. The day aims to raise awareness about the devastating health, social, and economic consequences of the disease.

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Drug Discovery World

FEBRUARY 19, 2024

There was a significant increase in cell and gene therapy product approvals by the FDA last year, and with the potential for up to 17 gene therapy approvals in the EU and US in 2024, the field is likely to continue to be at the forefront of drug development this year.

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Drug Development Small Molecule Drugs Therapies 147

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. Due to a delayed US launch it remains a drug to watch for 2024.

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Drugs Therapies Treatment Virus 173

On Medicine

FEBRUARY 28, 2024

What is Perthes’ disease? Perthes’ Disease is a rare condition that affects the developing femoral head (the “ball” of the “ball and socket” hip joint) of a child due to poor blood supply. This in turn can cause significant pain for children with Perthes’ disease and limit their activity levels significantly.

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Disease Doctors Treatment Trials 109

Drug Discovery World

JANUARY 29, 2024

In the same week that it was revealed that breast cancer was the most studied disease area in 2023 for the third year running, there were a number of promising developments in the treatment of colorectal, pancreatic and lung cancer and leukaemia. The post DDW Highlights: 29 January 2024 appeared first on Drug Discovery World (DDW).

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Treatment Disease Drugs 130

Drug Discovery World

MAY 21, 2024

The post New DDW Highlights podcast: 21 May 2024 appeared first on Drug Discovery World (DDW). You can listen below, or find The Drug Discovery World Podcast on Spotify , Google Play and Apple Podcasts.

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Virus Vaccine Therapies Drugs 147

Drug Discovery World

JULY 1, 2024

Following a trip to Copenhagen, Denmark, DDW’s Megan Thomas reflects on the Novo Nordisk Foundation’s efforts to tackle cardiometabolic diseases. The World Health Organization (WHO) reports that cardiovascular diseases (CVDs) are the leading cause of death globally, stating that an estimated 17.9

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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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Therapies Disease Research Drugs 130

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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Therapies Disease Research Drugs 147

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 post Speakers announced for Rejuvenation Startup Summit 2024 appeared first on Drug Discovery World (DDW).

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Perficient: Drug Development

MAY 28, 2024

On Sunday, May 19th, 2024, 21 individuals consisting of Perficient employees and their family members, participated in the 2024 Michigan Kidney Walk at the Detroit Zoo. This annual event aims to raise funds for the National Kidney Foundation, an organization that provides critical support to those affected by kidney disease.

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Drug Discovery World

JANUARY 29, 2024

In the same week that it was revealed that breast cancer was the most studied disease area in 2023 for the third year running, there were a number of promising developments in the treatment of colorectal, pancreatic and lung cancer and leukaemia.

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Treatment Disease Drugs Research 130

Drug Discovery World

MARCH 25, 2024

Allogene Therapeutics and Arbor Biotechnologies Biotechnology companies Allogene Therapeutics and Arbor Biotechnologies entered a non-exclusive, global gene editing licensing agreement for use of Arbor’s proprietary CRISPR gene-editing technology in Allogene’s AlloCAR T platform for the treatment of autoimmune disease (AID).

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Drug Discovery World

NOVEMBER 28, 2023

An experimental drug has shown potential as a disease-modifying therapy for Parkinson’s disease, according to a new study published in Nature Communications. Parkinson’s disease is highly associated with mitochondrial dysfunction. Mission is planning to initiate a MTX325 Phase I trial in humans in early 2024.

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KIF1A

APRIL 13, 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. One patient had the well-characterized E253K KIF1A mutation, highlighting the importance of increased screening for rare disease patients.

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Disease Research Science 111

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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Laboratories Cell Biology Disease Research 119

KIF1A

MAY 4, 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. By talking to groups with these mutations, we may identify other diseases whose patients have KIF1A variants. program in Chemistry in the fall of 2024).

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Science Medical Schools RNA Research 116

KIF1A

MARCH 16, 2024

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. Like hereditary spastic paraplegia, CMT is an umbrella disorder, associated with mutations in over 140 genes, but many diagnosed patients do not know their disease-causing mutation.

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Disease Clinical Development Research Treatment 105

LifeSciVC

JANUARY 16, 2024

recent announcements from Merck and Sanofi for obesity and broader metabolic disease). ongoing tirzepatide CVOT to assess head-to-head CV outcomes vs. Trulicity in T2D) will continue to build the case for weight loss (and incretins) in broader metabolic disease and suggest overall healthcare system cost benefits to anti-obesity therapies.

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Small Molecule Trials Therapies Disease 139

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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Therapies Clinical Trials FDA Disease 111

Drugs.com

JUNE 28, 2024

FRIDAY, June 28, 2024 -- Heart valve disease is much more common in aging adults that has been thought, a new study warns.More than a quarter of healthy and symptom-free people aged 60 and older had previously undetected heart valve disease.

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Disease 87

Drug Discovery World

FEBRUARY 19, 2024

The post New DDW Highlights podcast: 19 February 2024 appeared first on Drug Discovery World (DDW). With seasonal illnesses running amok in the western hemisphere at the moment, it is perhaps not surprising that this week’s news highlights focus on viruses and immunity.

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Virus Drugs Disease 130

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.

Disease 130

Disease Trials Small Molecule Pharmacokinetics 130

KIF1A

FEBRUARY 24, 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. Read the Article Rare Roundup Rare Disease Day is this Thursday, February 29th!

Disease 111

Disease Research Treatment Science 111

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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FDA Disease Therapies Clinical Trials 130

KIF1A

APRIL 20, 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. By looking at the players involved in cargo trafficking and the diseases they cause, we may be able to identify new therapeutic opportunities.

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Disease Research Treatment Science 105

Drug Discovery World

MARCH 8, 2024

Katie Abouzahr, Janssen Katie Abouzahr is the Vice President, Autoantibody Portfolio and Maternal Fetal Disease Area Leader at the Janssen Pharmaceutical Companies of Johnson & Johnson. The post IWD 2024: Eight women taking the lead in drug discovery appeared first on Drug Discovery World (DDW). Read the interview.

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Drugs International Pharmaceutical Companies Doctors 173

Drug Discovery World

APRIL 19, 2024

Askbio’s AB-1005 (also known as AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson’s disease, was well tolerated with no serious adverse events in a Phase Ib clinical trial. The data were presented at the American Academy of Neurology 2024 Annual Meeting in Denver, Colorado, USA.

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Therapies Disease Clinical Trials Trials 130

Drug Discovery World

FEBRUARY 16, 2024

in a full seed funding round in the second quarter of 2024. AMPLY works with a network of disease and biotech partners to help advance its programmes. to tackle complex diseases appeared first on Drug Discovery World (DDW). AMPLY raised its initial pre-seed funding in May 2022 and is planning to raise a further £1.4m

Disease 147

Disease Licensing Licensing Therapies 147

Drugs.com

MAY 21, 2024

TUESDAY, May 21, 2024 -- Matcha green tea has the potential to keep gum disease at bay, a new study finds.Lab experiments show that matcha can inhibit the growth of Porphyromonas gingivalis, one of the main bacterial culprits behind gum.

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Disease 98

Drugs.com

JUNE 10, 2024

MONDAY, June 10, 2024 -- An experimental 'supercharged' form of popular GLP-1 weight-loss meds could help ease fatty liver disease, a new trial suggests. The drug under development, survodutide, helped up to 83% of patients gain real improvements.

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Disease Trials Drugs 104

KIF1A

APRIL 6, 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. As our genetic analyses advance, studies like this highlight the value in reassessing established disease populations.

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DNA Medical Schools Therapies Research 111