Drug Discovery World

MARCH 25, 2024

Cardior is focused on the discovery and development of therapies that target RNA to prevent, repair and reverse diseases of the heart. The company seeks to advance a novel class of antisense oligonucleotides (ASOs) targeting non-coding RNAs (ncRNAs) that act on several key disease pathways simultaneously.

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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

FEBRUARY 1, 2024

Vivet is a clinical stage biotech company developing gene therapies for rare inherited metabolic disorders. These deposits can cause progressive damage to the brain and other areas of the body. Disease symptomatology is characterised by chronic diarrhea during infancy and cataracts in late childhood.

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PPD

SEPTEMBER 16, 2024

Real-world data paired with machine learning is a game changer in drug development RWD is becoming increasingly important in influencing the drug development landscape, particularly when used to develop the natural history and patient journey through disease.

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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

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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Drug Discovery World

JANUARY 5, 2023

CANbridge Pharmaceuticals has exercised its option to secure the exclusive global rights to develop, manufacture and commercialise a novel gene therapy to treat spinal muscular atrophy (SMA) from UMass Chan Medical School. . The benchmark therapy uses a vector similar to that used in the only gene therapy approved for SMA.

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Drug Discovery World

FEBRUARY 10, 2023

Breast cancer retained its position as the most-studied disease area in clinical trials in 2022, despite a drop in activity, according to analysis from analytics company Phesi. The post Breast cancer still top disease area for clinical studies appeared first on Drug Discovery World (DDW). Download the full report.

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Drug Discovery World

DECEMBER 4, 2023

Qnovia has announced positive results from its first in-human study of QN-01, an inhaled smoking cessation therapy. Smoking continues to be the leading cause of preventable death and disease, leading to almost eight million direct and indirect deaths annually.

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Drug Discovery World

MARCH 14, 2023

Teitur Trophics, a biotech company developing new treatments for patients suffering from neurodegenerative diseases, has complete a €28M Series A financing. The financing was co-led by Sunstone Life Science Ventures and Sound Bioventures, with participation from new investors, Industrifonden, Innovestor’s Life Science Fund and P53 Invest.

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Drug Discovery World

JUNE 19, 2024

DDW Editor Reece Armstrong speaks to Jasminka Taleska , Director, RLT HCS Readiness and Policy at Novartis, about the opportunities radioligand therapies (RLTs) represent in treating cancer patients. The post How ready are we for radioligand therapies? RA: What are the potentials of RLTs across cancer? JT: This is very important 6.

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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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Drug Discovery World

APRIL 6, 2023

Granular Therapeutics, a UK-based biotech company specialising in precision biologic therapies for treating mast cell driven disorders, announced clinical candidate selection for its lead programme which has demonstrated promising results in initial preclinical studies. It has developed a pipeline that selectively targets mast cells.

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Drug Discovery World

SEPTEMBER 4, 2024

EVO756 is a highly selective small molecule antagonist of mas-related G-protein coupled receptor X2 (MRGPRX2) and could offer the potential for once-daily oral administration without the side effects observed with other therapies.

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Drug Discovery World

SEPTEMBER 6, 2022

In recent years the advanced therapy industry has been the most significant disruptor in medicine. Cell and gene therapies are not only transforming the way diseases are treated but are also aiming to cure entirely new patient populations. Record breaking investments in 2021 fuel an industry on fire . billion raised.

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Drug Discovery World

DECEMBER 20, 2022

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP). . The therapy was also recently approved by the FDA for patients in the US. .

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Drug Discovery World

SEPTEMBER 20, 2022

SKYSONA has become the first FDA approved therapy to slow the progression of neurologic dysfunction in boys with cerebral adrenoleukodystrophy (CALD). CALD is a rare, progressive, neurodegenerative disease that primarily affects young boys and causes irreversible, devastating neurologic decline.

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Drug Discovery World

AUGUST 28, 2023

DDW’s Diana Spencer speaks to Peter Hamley , Chief Scientific Officer of Samsara Therapeutics, to understand the role autophagy plays in neurodegenerative diseases and how boosting this process could increase healthspan. DS: Samsara’s autophagy inducing candidate SAM001 will be the first agent of its class to enter clinical trials.

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Drug Discovery World

JULY 12, 2024

The analysis of 66,935 clinical trials reveals that the top five most studied diseases are now breast cancer, solid tumours, stroke, prostate cancer and type 2 diabetes. The mid-year analysis indicates breast cancer could be the most researched disease area for four-years running, after topping the table last year.

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Drug Discovery World

OCTOBER 4, 2022

William Erhardt , President, Head of Development & Operations of biotech Oligomerix, tells Lu Rahman about the company’s differentiated approach to developing disease-modifying therapeutics for neurodegenerative diseases. . Overcoming sector challenges .

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Drug Discovery World

DECEMBER 14, 2023

“We are thrilled to see this level of clinical activity in such a heavily pre-treated patient population,” said John Houston, Chairperson, Chief Executive Officer and President at Arvinas. Vepdegestrant is the only PROTAC ER degrader in late-stage clinical development. months of additional follow-up and 78.3%

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Drug Discovery World

MAY 16, 2023

What are the global innovations in cell and gene therapy? What opportunities and challenges are emerging and can therapies get to market faster? The deeper the understanding of product and process from a characterisation standpoint, the smoother the regulatory conversations and development pathway will be.” The solution?

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Drug Discovery World

DECEMBER 21, 2022

Biopharmaceutical company Rezolute has initiated a Phase II proof-of-concept study for RZ402, a plasma kallikrein inhibitor (PKI) being developed as an oral therapy for the treatment of diabetic macular oedema (DMO). .

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Drug Target Review

JULY 31, 2023

Our inaugural report is a groundbreaking exploration of the remarkable advancements in cell and gene therapy that are revolutionising the field of drug discovery. Expert Insights We are honoured to have collaborated with renowned experts in the field of cell and gene therapy, who have generously shared their invaluable insights.

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Drug Discovery World

APRIL 3, 2024

million from the Spanish Research Agency (AEI), under the call for grants for Public-Private Collaboration Projects, to accelerate the development of an innovative treatment aimed at controlling disabling and lethal haemorrhage.

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Drug Target Review

OCTOBER 11, 2023

Almost every one of us will know someone with a neurodegenerative disease such as Alzheimer’s or Parkinson’s. It is less common to know someone with a rare genetic disease; but while individually rare, collectively they have an enormous impact. This will, in turn, slow the progression of, or even halt, the disease.

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Drug Target Review

FEBRUARY 1, 2024

What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? But commitment to neuroscience drug development is critically important given the significant unmet medical needs and the ways in which patients, caregivers, and families suffer.

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Conversations in Drug Development Trends

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment.

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FDA Law Blog: Drug Discovery

JUNE 12, 2024

Sasinowski — Hyman, Phelps & McNamara (HPM) would like to congratulate the 7 rare disease programs selected for the inaugural class of the FDA’s “Support for clinical Trials Advancing Rare disease Treatment” (START) pilot program. HPM is proud to aid 5 of the 7 selected participants for the START pilot program.

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Drug Discovery World

JULY 11, 2022

David Lewandowski, Business Director of Cell & Gene Therapy at Azenta Life Sciences, explains how efficient sample management can help to work efficiently and bring therapies to market faster. The global cell and gene therapy (CGT) market reached a value of ~$4 billion in 2020 and is expected to grow to ~$34 billion by 2030 1.

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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.

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The Pharma Data

FEBRUARY 24, 2022

Bayer announced today the initiation of the CONFIDENCE study, a Phase II, three-arm study that will investigate simultaneous initial combination therapy with finerenone and the SGLT2 inhibitor empagliflozin, compared with finerenone alone and empagliflozin alone respectively in patients with chronic kidney disease (CKD) and type 2 diabetes (T2D).

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Drug Discovery World

JANUARY 11, 2024

Vice President, Clinical Development, Infectious Diseases at Moderna examines the data on cytomegalovirus (CMV), why attempts at vaccines for CMV have so far proved elusive and the emerging options to address this unmet need. In the first article, Allison August, M.D.,

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Drug Target Review

MAY 7, 2024

Why are biomarkers particularly critical in the context of rare diseases? Developing novel therapeutic approaches for rare diseases poses inherent challenges due to the limited populations available for testing. How do AI-enhanced approaches accelerate the drug development process for rare diseases?

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Cytel

APRIL 10, 2024

Orphan drug designation is a regulatory status granted to pharmaceuticals developed for the treatment of rare diseases. It provides incentives to encourage the research, development, and approval of therapies targeting small patient populations.

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Drug Discovery World

JULY 17, 2023

Analysis of 13,490 clinical trials finds almost a third have been cancelled during Phase II amidst an overall decline in clinical development productivity. Data analytics company Phesi has released the results of its mid-year global analysis of all clinical trials conducted in 2023 to date.

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Drug Discovery World

FEBRUARY 26, 2024

The US Food and Drug Administration (FDA) has granted Fast Track Designation for Certa Therapeutics’ investigational therapy FT011 for the treatment of systemic sclerosis (scleroderma).

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