Drug Discovery World

DECEMBER 12, 2023

Exonate, an mRNA therapy company focused on treatments for diabetic complications, has announced that its lead ophthalmology asset, EXN407, has achieved its prespecified endpoints in a Phase Ib/IIa study. During the trial, EXN407 met all safety and pharmacokinetic parameters and displayed encouraging signals of biological activity.

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Drug Discovery World

MARCH 15, 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 Target Review

SEPTEMBER 25, 2024

Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.

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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. appeared first on Drug Discovery World (DDW).

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Covalent Modifiers

JANUARY 29, 2024

Moore Journal of Medicinal Chemistry 2024 DOI: 10.1021/acs.jmedchem.3c01835 3c01835 Building on recent advances in peptide science, medicinal chemists have developed a hybrid class of bioconjugates, called peptide–drug conjugates, that demonstrate improved efficacy compared to peptides and small molecules independently.

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Drug Discovery World

JANUARY 15, 2024

The NVIDIA DGX SuperPOD will be installed at Amgen’s deCODE genetics’ headquarters in Reykjavik, Iceland and will be used to build a human diversity atlas for drug target and disease-specific biomarker discovery. Chiesi and Oak Hill Bio intend to continue a Phase IIb clinical trial in 2024 in the United States, Europe, and Japan.

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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

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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Drug Discovery World

MAY 13, 2024

Key to this is the ability to rapidly determine protein structures alone and in combination with antibodies or small molecules. With that information, researchers can map sequence changes to the spike protein to assess whether there are obvious concerns with vaccine coverage or antibody therapies.

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Alta Sciences

AUGUST 29, 2024

Listen or read Issue 33 of The Altascientist on altasciences.com , or wherever you listen to podcasts: GROWING NEEDS FOR CNS THERAPY SOLUTIONS The different parts of the nervous system, including the brain and spinal cord and the peripheral nervous system, are important drug targets for many serious diseases affecting human health.

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Drug Target Review

JULY 18, 2024

Beyond the standard tumour-informed assay, we collaborated with Natera to create a more sensitive tumour-informed test using its Signatera molecular residual disease testing technology for research use in our AMPLIFY-201 clinical trial. Nat Med 30, 531–542 (2024). Reference 1 Pant, S., Wainberg, Z.A., Weekes, C.D.

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Drug Discovery World

FEBRUARY 5, 2024

DDW’s Megan Thomas spoke to experts from the drug discovery industry about their predictions on what 2024 holds for our sector. New innovations can attack the disease from multiple angles, and new data and machine-learning AI technology are helping to detect cancer earlier and optimise personalised care pathways.

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KIF1A

OCTOBER 3, 2024

Her leadership and vision have resulted in the establishment of crucial research initiatives, including a vision study and a speech study, alongside the development of gene therapy approaches and biomarkers specifically for KAND. We know that living with a rare disease can be an overwhelming and isolating journey.

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Sygnature Discovery

OCTOBER 10, 2023

Microglia are neuro-inflammatory cells that constitute ~10% of the brain’s cell population and are a key therapeutic target in the development of adequate treatments for Alzheimer’s Disease and other neurodegenerative conditions.

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Drug Discovery World

APRIL 30, 2024

Reece Armstrong explores the potential and future opportunities for mRNA-based therapies. For instance, consider how at the end of 2018, the mRNA therapies market was valued at $3.43 If the body fails to make the right protein, diseases such as cancer, type II diabetes, neurological conditions, and metabolic diseases can occur.

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Sygnature Discovery

NOVEMBER 29, 2023

They anticipate providing an update on safety and preliminary efficacy of the Phase 1a study in the first half of 2024. The initial work at UCL on this revolutionary approach to cancer therapy was made possible with backing especially from the UCL Technology Fund and the UK Pancreatic Cancer Research Fund. Qualigen Therapeutics, Inc.

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Drug Discovery World

JULY 17, 2024

Imperial College spinout NK:IO was awarded a £1.6m ($2m) Innovate UK New Cancer Therapeutics grant in 2023 to develop a pioneering NK cell therapy. DS: How can NK cell therapy tackle the limitations of cancer immunotherapy? Diana Spencer catches up with Co-Founder Mike Romanos to find out more. Six have been approved since 2017.

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Sygnature Discovery

NOVEMBER 29, 2023

They anticipate providing an update on safety and preliminary efficacy of the Phase 1a study in the first half of 2024. The initial work at UCL on this revolutionary approach to cancer therapy was made possible with backing especially from the UCL Technology Fund and the UK Pancreatic Cancer Research Fund. Qualigen Therapeutics, Inc.

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Drug Discovery World

SEPTEMBER 26, 2024

The Drug Discovery in Scotland Forum showcased how Scotland is shaping drug discovery using approaches including traditional small molecules, therapeutic proteins, CRISPR/ RNAi and cell therapies, as well as how these are being used in 2D and 3D models.

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Elrig

JUNE 4, 2024

The focus used to be more on small molecules, but we’re now starting to see antibody and cell therapeutics. All these ‘worlds’ can learn from each other – especially since the small molecule drug discovery process is much more mature than the antibody which is much more mature than the gene and cell therapy space.

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Drug Discovery World

JANUARY 24, 2024

DDW’s Megan Thomas caught up with Wigerinck to learn more about his work with small molecules for fibrosis research. MT: Your appointment comes as Fibrocor seeks to advance two drug candidates – exquisitely specific kinase inhibitor small molecules (FIB992 and FIB991) for rare indications into the clinic.

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Drug Target Review

MAY 31, 2024

I currently lead a talented team of multi-disciplinary scientists who are dedicated to discovering a treatment for Parkinson’s Disease, a debilitating condition with significant unmet medical need. As a woman in STEM, what unique perspectives or strengths do you believe you bring to your work?

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Drug Discovery World

SEPTEMBER 9, 2024

Vesper Bio has completed a Phase I study for its lead candidate, VES001, a potentially disease-modifying treatment for frontotemporal dementia patients with mutations in the progranulin gene (FTD(GRN)). Frontotemporal dementia is the most common cause of dementia in people under the age of 60.

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KIF1A

JANUARY 27, 2024

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. The study found improved spasticity and movement in mice treated with ARL61P1 gene therapy; it also restored abnormalities observed in the brains of ARL61P1-deficient mice.

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The Pharma Data

DECEMBER 14, 2020

It is considered an orphan disease for which there is no cure. billion by 2024, according to GlobalData, with the U.S. The effectiveness of standard therapy with TMZ is limited because the response of GBM to TMZ is dependent upon the expression of the DNA repair enzymatic protein, O 6 -alkylguanine DNA alkyltransferase (MGMT).

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Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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Conversations in Drug Development Trends

MAY 30, 2024

The peak number of trials in the last five years and the regulatory approvals of three additional disease-modifying treatments for ALS in the previous eight years signal cause for hope that, as a community, we have the potential to transform this progressive and ultimately terminal neurodegenerative disorder into a treatable condition.

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Agency IQ

APRIL 19, 2024

BY LAURA DIANGELO, MPH | APR 15, 2024 10:06 PM CDT FDA’s Center for Drug Evaluation and Research (CDER) has a new initiative to advance clinical trials Quick background: The push for “innovation” in clinical trial design. focused on a quality by design approach).” One key update compared to previous work: Moving from pilots to programs.

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Agency IQ

APRIL 19, 2024

FDA advisors endorse minimal residual disease (MRD) as accelerated approval endpoint for multiple myeloma Last week, FDA’s Oncologic Drugs Advisory Committee (ODAC) voted unanimously in favor of using minimal residual disease (MRD) as an accelerated approval endpoint for multiple myeloma.

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KIF1A

MAY 31, 2024

ASCEND Online and KOALA In-Person Natural History Study Updates Understanding KAND and the progression of the disease informs our path to treatment. Dr. Kaur now oversees 5 KAND research projects that utilize our patient-derived cell lines to screen for small molecule therapeutics and test potential gene therapies.

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Drug Discovery World

NOVEMBER 13, 2023

Ahead of protein and antibody engineering conference PEGS Europe 2024 in Lisbon, DDW’s Megan Thomas looks at what to expect from each track of the annual biologics technology meeting. Jürgen Kuball, PhD, Head, Hematology, University Medical Center Utrecht, on: ‘gdT cell inspired therapies’.

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KIF1A

AUGUST 20, 2024

To paraphrase two inspiring rare disease parents and advocates speaking about their children: “She still can’t do math, but improved speech, better attention – to us these are cognitive improvements.” The use of ellipses (…) represents skipped text that can be referenced in the original report. Will this _ KAND?

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New Drug Approvals

SEPTEMBER 13, 2024

19] As of 2016, aticaprant has reached phase II clinical trials as an augmentation to antidepressant therapy for treatment-resistant depression. [20] 19] As of 2016, aticaprant has reached phase II clinical trials as an augmentation to antidepressant therapy for treatment-resistant depression. [20] 2] Aticaprant is taken by mouth. [1]

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Codon

MARCH 19, 2024

Most people die from cardiovascular disease, while others succumb to Alzheimer’s and cancer. Take cardiovascular disease as an example. However, this approach is imperfect because it only addresses one manifestation of the disease, rather than its root. José Luis Ricón ( Nintil ) writes about aging for Issue 01.

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Codon

JUNE 11, 2024

But these little machines, small enough to fit inside a pocket, have become commonplace. Biologists use nanopores for everything from diagnosing diseases and monitoring changes within rainforest ecosystems to discovering proteins from microbes frozen in Icelandic glaciers. ” Asimov Press (2024). Cite: Stephen Malina.

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Agency IQ

FEBRUARY 23, 2024

BY CHELSEY MCINTYRE, PHARMD , KARI OAKES | FEB 21, 2024 7:48 PM CST Background: Dose-finding in oncology Traditionally, early development of oncology products has focused on identifying the maximum tolerated dose (MTD) of an investigational product. This has spawned a movement focused on optimizing dose selection in oncology drug development.

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The Pharma Data

DECEMBER 5, 2020

Taken together, these data support the potential of SY-1425 to become an important targeted therapy for RARA-positive AML patients, as well as for patients with a closely related hematologic malignancy known as higher-risk myelodysplastic syndrome.”. Syros expects to initiate the Phase 2 trial in the second half of 2021.

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