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New avenues for rare disease treatment

Drug Target Review

More than 7,000 rare diseases – conditions that affect fewer than 1 in 2,000 people – have been identified, and1 in 17 people will develop a rare disease in their lifetime, with children disproportionately affected. Most of these conditions are genetic in origin and the majority have no effective treatment.

Disease 110
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Taking a new approach to tackle neurodegenerative diseases

Drug Discovery World

New York-based Oligomerix is an emerging clinical-stage biotechnology company focused on developing disease-modifying therapeutics for neurodegenerative diseases characterised by aberrant tau protein ranging from rare tauopathies such as progressive supranuclear palsy (PSP) and frontotemporal dementia (FTD) to Alzheimer’s disease (AD).

Disease 130
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Three trends in the antibody-drug conjugate (ADC) market 

Drug Discovery World

Antibody-drug conjugates (ADCs) are biopharmaceutical products in which a monoclonal antibody (mAB) is linked to a small molecule drug with a stable linker 1. According to NCBI, most of the ADCs developed so far are for use in the treatment of cancer, but there is plenty of potential for using ADCs to treat other diseases 1.

Marketing 245
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Bayer acquires Noria and PSMA Therapeutics to expand portfolio in prostate cancer

The Pharma Data

Broadens company’s oncology platform of Targeted Alpha Therapies / Acquisition includes actinium-225 labeled differentiated PSMA small molecule for the treatment of prostate cancer. The pre-IND program focuses on the treatment of prostate cancer, the second most commonly diagnosed cancer in men.

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Knopp Biosciences Announces Positive Top-Line Results from the Phase 2 EXHALE Trial of Oral Dexpramipexole in Moderate-to-Severe Eosinophilic Asthma

The Pharma Data

These Phase 2 results strongly support the continued development of dexpramipexole as a potential first-in-class oral agent for the treatment of eosinophilic asthma,” said Michael Bozik, M.D., Knopp’s clinical-stage oral small molecule, dexpramipexole, is in Phase 2 clinical trials in moderate-to-severe eosinophilic asthma.

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AbbVie Acquires Syndesi Therapeutics, Strengthening Neuroscience Portfolio

The Pharma Data

This acquisition gives AbbVie access to Syndesi’s portfolio of novel modulators of the synaptic vesicle protein 2A (SV2A), including its lead molecule SDI-118. ” The lead molecule, SDI-118, is a small molecule currently in Phase 1b studies, which is being evaluated to target nerve terminals to enhance synaptic efficiency. .

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Metabolism of 2023 FDA Approved Small Molecules – PART 1

Metabolite Tales Blog

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. References [1] 2023 Novel Small Molecule FDA Drug Approvals.