May 2021 Volume 17, Issue 5

Volume 17, Issue 5 | May 2021

May 2021

In this Issue

Clinical Trials

Encouraging results for tirzepatide in diabetes

Topline data from a recent Eli Lilly clinical trial indicate that tirzepatide led to superior A1C and body weight reductions from baseline compared to injectable semaglutide in adults with type 2 diabetes.

The outlook is BRIGHT for Fabry disease

Protalix and Chiesi share encouraging data from Phase 3 trial evaluating pegunigalsidase alfa in Fabry disease

Fighting fungal infections

Oral ibrexafungerp shows positive results in Phase 3 study against difficult fungal infections

A type 1 diabetes game-changer

Promising follow-up data from TN-10 teplizumab study published in Science Translational Medicine

Discovery

From culprit to ally

Research reveals that collagen, thought to aid cancer progression, actually fights disease spread

Meet a new alliance for neuroscience

Weill Neurohub joins forces with Genentech and Roche on neuroscience and CNS disorders

Tools against tumors

Grant from Innovate UK to Pathios seeks to advance cancer immunotherapy

Looking to mRNA for HIV and beyond

ConserV Bioscience and eTheRNA immunotherapies collaborate on mRNA vaccines for infectious diseases

Novel SARS-CoV-2 neutralizing antibodies

Therapeutic candidates retain binding to major COVID-19-causing “South African” variant

Spotlight on Liver & Kidney Disease

A look at some of the latest renal and hepatic R&D

New options for hepatocellular carcinoma

“A large number of pipeline agents with innovative approaches are under investigation for adjuvant, intermediate, and advanced therapy [for hepatocellular carcinoma].” Mandana Emamzadeh of GlobalData

Diagnostics

Information integration

Genomenon and BC Platforms seek to combine their tech platforms to boost genomic research

Molecular diagnostic and antibody research get a boost

Yurogen and Asuragen acquisitions expected to aid wide range of researchers

A new spinout for healthcare AI

The Quinten Group spins off Quinten Health, using AI for precision care and real-world data science

AI against Parkinson’s disease

New AI tool to be tested in NIH-funded study to improve diagnosis of Parkinson’s and related disorders

Algorithm could predict acute kidney injury

Findings showed that Previse could predict the onset of AKI up to 48 hours in advance of onset, sooner than the standard hospital systems like XGBoost AKI prediction model and the Sequential Organ Failure Assessment (SOFA).

Editor's Focus

Focus Feature: -Omics and Gene Therapy

Genomics, proteomics, and transcriptomics loom large in oncology

Twins and genomics: We are not the same

Different environmental exposures, different lifestyles, and so on. So they might have different weights or different styles or even different chronic diseases.

Genomics & Proteomics

The genomics of COVID

Advances in genomics research increased the speed with which vaccines came to the fore to mitigate infection by the SARS-CoV-2 virus, thanks to early sequencing of the virus. Here is a quick roundup of some recent genomics news related to COVID-19.

A potential gene therapy for spinal muscular atrophy

According to the company, the new data underscore the critical importance of identifying and treating SMA as early as possible.

Gene therapy center aims to cure the incurable

“Gene therapy has huge potential. It has already led to cures for conditions such as spinal muscular atrophy (SMA). In the future, we will be looking at conditions like motor neurone disease, Alzheimer’s and rare cancers,”

Feature

Out of order: Risks, relatively speaking

To be clear, any adverse event is always one too many. But in the real world, these things happen and often through no intrinsic fault of the therapeutic intervention

Business & Government Policy

Regenerative medicine market to reach $16.4B in 2026

“The COVID-19 pandemic could be an opportunity for the companies pursuing the development of gene therapies, as few products are in development to fight the viral illness.”
Alessio Brunello of GlobalData

A portal for regenerative medicine

“We have heard how difficult it has been to find standards and want to make it as easy as possible for the industry and suppliers to find the standards that can make product development and manufacturing processes more efficient.” Robert Shaw of SCB

Tools & Technology

A “cookie cutter” technique for COVID drug discovery

“Beyond COVID-19, we believe SMAs have huge potential to unblock constrictions in the drug pipeline.”

From astronomy to diagnostics?

Microcombs could be used in many ways, including detecting disease and finding new planets

Real-world data to the rescue

EVERSANA launches chronic disease real-world data solution to improving patient outcome

Preclinical

Alternating two-vector therapies prove promising

Data on HOOKIPAs cancer therapeutics have been published in Cell Reports Medicine

A double-barrel approach

Peptide drug targets two cancer proteins for increased specificity and uptake

Lifelike systems

Organ-on-chip and tissue-on-chip models continue to advance drug discovery

Two novel chronic kidney disease candidates arise

CKD is a slow and progressive loss of kidney function over several years, which can eventually cause permanent kidney failure. CKD often goes undetected and undiagnosed until the disease is well advanced, and the damage is irreversible resulting in poor survival rates. There are estimated to be 700 million cases of CKD worldwide, and currently there is no cure.
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Harnessing liver-on-a-chip models for drug safety

Discover how researchers leverage microphysiological systems in toxicology studies.  
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Explained: How are metabolite biomarkers improving drug discovery and development?

By offering a rich source of insights into disease and drugs, metabolite biomarkers are at the forefront of therapeutic exploration.
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