Drug Discovery World

JULY 19, 2024

TPD is a rapidly emerging field in drug development, exploiting a cell’s own destruction machinery to tackle disease-causing proteins that have historically been highly challenging to target with conventional therapies.

Targeted Protein Degradation 130

Targeted Protein Degradation Small Molecule Research Government 130

Drug Discovery World

JUNE 7, 2023

Biotech company Shasqi and Johnson & Johnson have entered into a research collaboration to develop new cancer therapies. We are excited to collaborate and apply click chemistry for potential new therapies in areas of significant unmet medical need.”

Therapies 130

Therapies Small Molecule Research Drugs 130

Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. It is imperative for the industry to communicate the precise composition and attributes of these therapies to regulatory bodies.

Therapies 162

Therapies Clinical Trials Small Molecule Production 162

DrugBank

JULY 24, 2024

These multifunctional small molecules are like tiny spies, hijacking the body’s natural protein degradation system to remove unwanted proteins. For example, PROTACs targeting STAT3, a protein crucial for tumor growth, have shown promise where conventional therapies have failed.

Small Molecule 84

Small Molecule Clinical Trials Drugs Therapies 84

Covalent Modifiers

FEBRUARY 6, 2024

The most common adverse effects are hyperphosphatemia caused by FGFR1 inhibition and diarrhea due to FGFR4 inhibition, as current therapies are not selective among the FGFRs.

Small Molecule 88

Small Molecule Treatment Science Therapies 88

Drug Discovery World

NOVEMBER 15, 2023

The small molecule, which binds to an inner region of a calcium channel to indirectly regulate it, outperformed gabapentin without troublesome side effects, providing a promising candidate for treating pain. A first-in-class small molecule Protein CRMP2 is a key regulator of the Cav2.2

Small Molecule 130

Small Molecule Treatment Regulations Clinical Trials 130

Drug Target Review

JUNE 19, 2024

The mission of Lineage Cell Therapeutics is to deliver on some of the early promises of cell therapy. Cell therapy as a concept is a wonderful idea, but many of the early efforts never generated the kind of clinical data that gets people excited and leads to new medicines. In some cases, cell therapy can be curative for the patient.

Therapies 84

Therapies Small Molecule Pharmaceutical Companies Molecular Biology 84

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.

Small Molecule 130

Small Molecule Trials Pharmacokinetics Treatment 130

Drug Discovery World

JULY 18, 2024

DSP-5336 is an investigational small molecule inhibitor of the menin and mixed-lineage leukaemia (MLL) protein interaction, which plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and haematopoiesis.

Small Molecule 130

Small Molecule FDA Drugs Therapies 130

The Pharma Data

NOVEMBER 17, 2020

Lead Pharma has entered into a collaboration and license agreement with Roche , hoping to lead the way in the development of oral small molecules for immune-mediated diseases like rheumatoid arthritis, psoriasis and inflammatory bowel diseases. . This includes therapies against the nuclear receptor ROR?t,

Small Molecule 52

Small Molecule Disease Immune Response Licensing 52

Drug Discovery World

JUNE 26, 2024

There remains a significant unmet need for novel therapies that address the underlying synaptic pathology central to AD and our first-in-class, synaptogenic small molecule has the potential to provide a meaningful clinical impact.

Small Molecule 189

Small Molecule Pharmacokinetics Clinical Trials Drugs 189

The Pharma Data

JANUARY 19, 2021

Precision medicine developer Kura Oncology is positioned to make a meaningful difference in the treatment of cancer in the next few years with its two, wholly-owned, small molecule compounds. As with any innovative therapy, “It takes multiple iterations and time to get things right,” he pointed out. “We Photo courtesy of Kura.

Small Molecule 52

Small Molecule Therapies FDA Approval Trials 52

Drug Discovery World

FEBRUARY 12, 2024

Cameron Ross, SVP, Generative AI, Elsevier “AI has the potential to dramatically shorten one of the most costly areas of early drug development – small molecule discovery. We’re already seeing biotechs making breakthroughs with AI in this area, with more than 150 small molecule drugs discovered and 15 already in clinical trials.

Small Molecule 349

Small Molecule Regulations Clinical Trials Therapies 349

Drug Discovery World

MAY 30, 2024

A new research paper has validated the mechanism of action of the Atrogi’s first-in-class, small molecule, ATR-127, in combating obesity and metabolic complications. Unlike existing anti-obesity therapies that compromise lean muscle mass, ATR-127 induces significant weight loss while preserving crucial lean body mass.

Small Molecule 147

Small Molecule Treatment International Production 147

Dark Matter Blog

AUGUST 27, 2020

A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. Using gene therapy, their approach was beautifully straightforward – “Missing an important gene? But we have not been the only people on this mission. We will replace it.”

Small Molecule 52

Small Molecule RNA Clinical Trials Drugs 52

Broad Institute

NOVEMBER 2, 2023

Bayer and Broad Institute extend cancer therapy research collaboration By Corie Lok November 2, 2023 Breadcrumb Home Bayer and Broad Institute extend cancer therapy research collaboration Long-standing industry-academia collaboration has already resulted in three clinical oncology candidates.

Therapies 103

Therapies Research Small Molecule Clinical Trials 103

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).

Drug Development 147

Drug Development Small Molecule Drugs Therapies 147

Drug Discovery World

MAY 24, 2023

million to develop the next generation of small molecule drugs against disease areas of high clinical need. The aim is to develop therapies with fewer side effects to treat diseases with a high clinical need. PharmEnable has closed a pre-series A investment round of $7.5 The post $7.5M

Small Molecule 130

Small Molecule Disease Therapies Treatment 130

Drug Target Review

JANUARY 4, 2024

What distinguishes Alder Therapeutics’ approach to regenerative cell therapy development from traditional methods, and how does it aim to reduce risks in the preclinical phase? Traditional approaches to regenerative cell therapy development are defined by several challenges. So, what makes these therapies unique?

Therapies 98

Therapies Small Molecule Production Disease 98

Drug Discovery World

OCTOBER 21, 2022

While advances in treatment over the last few years have resulted in good overall survival rates for patients with non-metastatic disease (90% five-year survival rate in the US 1 ), outcomes are still poor in certain cancers that do not respond to standard therapies. 3D bioprinted breast cancer tumours.

Therapies 130

Therapies Small Molecule Immune Response Engineering 130

Science Daily: Pharmacology News

NOVEMBER 30, 2023

Researchers have succeeded in restoring lost brain function in mouse models of stroke using small molecules that in the future could potentially be developed into a stroke recovery therapy.

Small Molecule 121

Small Molecule Therapies Research 121

Drug Discovery World

OCTOBER 19, 2022

Reece Armstrong speaks to Natalia Mateu, Co-Founder, Jelena Aleksic, Co-Founder and Peter Curran, Chemoinformatics Scientist, about utilising 3D chemistry and AI to get small molecule therapies from the clinic to the bedside. RA: How is Pharmenable aiming to replicate the specificity of biologics in small molecule drugs?

Small Molecule 130

Small Molecule Therapies Drugs 130

Drug Discovery World

JULY 19, 2024

Currently used animal models are poor predictors of drug outcomes, and many of the blood disease treatments on offer – such as mRNA drugs and human-specific small molecules – don’t test well in animal models.

Small Molecule 130

Small Molecule Drugs Engineering Disease 130

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.

Small Molecule 130

Small Molecule Clinical Trials Drugs Trials 130

Drug Discovery World

MARCH 12, 2024

Adoptive T-cell therapy (ACT), particularly T-cell-based transplantation, is attracting increasing attention and is advancing rapidly since the FDA approval of CAR-T therapy for the treatment of relapsed/refractory acute lymphoblastic leukaemia and B-cell malignancies.

Molecular Biology 204

Molecular Biology Small Molecule Therapies FDA Approval 204

Drug Discovery World

AUGUST 16, 2023

Over the next two decades, it is quite possible that most new drugs approved could be based on some form of RNA, whether it be through its delivery or targeting the molecule itself. The post Junk DNA: How the dark genome is changing RNA therapies appeared first on Drug Discovery World (DDW).

RNA 130

RNA DNA Therapies Small Molecule 130

Drug Discovery World

MAY 31, 2024

The selected news highlights this week report breakthrough findings from early phase research and development, mainly preclinical studies and Phase I clinical trials, including a drug that can prevent sepsis deaths, a microbiome based immuno-oncology candidate, and a plant-based therapy for diarrhoea.

Small Molecule 130

Small Molecule Clinical Trials Drugs Trials 130

Drug Discovery World

FEBRUARY 5, 2024

Opening keynote Following a change to the schedule, Juliet Williams of Kymera Therapeutics launched the event with a popular keynote talk ‘Solving Big Problems with Small Molecule Degraders’.

Small Molecule 162

Small Molecule Assay Development Production Science 162

Covalent Modifiers

JUNE 24, 2024

Rullo, Molecular Therapy: Oncology , 2024 , 200842, [link] Proximity-induction of cell-cell interactions via small molecules represents an emerging field in basic and translational sciences. Lake, Galina Denisova, Joanne A. Hammill, Jonathan L. Bramson, Anthony F.

Small Molecule 52

Small Molecule Engineering Science Therapies 52

Drug Discovery World

SEPTEMBER 6, 2022

DDW Editor Reece Armstrong looks at the cell and gene therapy landscape, examining the challenges facing developers and the trends we can expect to see throughout the year. . There’s no doubt that cell and gene therapies present some of the most exciting opportunities for emerging drugs. million price point per dose.

Therapies 130

Therapies Clinical Trials Vaccine Disease 130

Drug Discovery World

APRIL 5, 2024

The platform has a track record of identifying highly selective, brain penetrant small molecule drugs, a class that has the potential to revolutionise the treatment of neurological disorders. “We

Small Molecule 130

Small Molecule Therapies Treatment Science 130

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects.

Small Molecule 145

Small Molecule Drugs Clinical Pharmacology Trials 145

The Premier Consulting Blog

APRIL 18, 2024

Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. This blog will outline three characteristics of a successful nonclinical program to support entry into clinical trials for a cell or gene therapy product.

Therapies 52

Therapies Clinical Trials Small Molecule Immune Response 52

The Premier Consulting Blog

APRIL 18, 2024

Designing an adequate nonclinical program to support the safety of cell or gene therapy products is not always straightforward. The cell or gene therapy should be pharmacologically active in the animal model. Note that there is no strict requirement of toxicity studies in two species, which is often needed for small molecule programs.

Therapies 52

Therapies Small Molecule Production Clinical Trials 52

DrugBank

MAY 15, 2024

One approach is to look beyond the traditional drug molecule. Researchers are experimenting with biologics—larger biological molecules that can do things small molecules can't, like targeting larger, more complex structures on cell surfaces or even inside cells.

Small Molecule 83

Small Molecule Molecular Biology Drugs Disease 83

Drug Discovery World

JANUARY 17, 2023

The peptides that we have discovered can be used as targeting ligands directly conjugated to silencing RNAs, small molecules for therapeutics or as imaging probes,” Herrera-Barrera added. . Limitations of AAV gene therapy . Sahay and Ryals have received a $3.2

Small Molecule 246

Small Molecule Pharmacy RNA Packaging 246

Drug Discovery World

AUGUST 18, 2023

Genetic biomarkers could personalise therapy for TNBC A new study has shed light on the genetic messages encoded by genes within ‘triple negative’ breast cancers (TNBC), and shows they could predict response to chemotherapy.

Small Molecule 130

Small Molecule Virus Drugs Clinical Research 130