May 2021- Volume 17, Issue 5
In this Issue
Editor's Focus
Twins and genomics: We are not the same
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.
Focus Feature: -Omics and Gene Therapy
Focus Feature: -Omics and Gene Therapy
Genomics, proteomics, and transcriptomics loom large in oncology
Business & Government Policy
Regenerative medicine market to reach $16.4B in 2026
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
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
Feature
Out of order: Risks, relatively speaking
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
Genomics & Proteomics
Gene therapy center aims to cure the incurable
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,”
A potential gene therapy for spinal muscular atrophy
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.
The genomics of COVID
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.
Discovery
Spotlight on Liver & Kidney Disease
Spotlight on Liver & Kidney Disease
A look at some of the latest renal and hepatic R&D
New options for hepatocellular carcinoma
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
From culprit to ally
From culprit to ally
Research reveals that collagen, thought to aid cancer progression, actually fights disease spread
Meet a new alliance for neuroscience
Meet a new alliance for neuroscience
Weill Neurohub joins forces with Genentech and Roche on neuroscience and CNS disorders
Looking to mRNA for HIV and beyond
Looking to mRNA for HIV and beyond
ConserV Bioscience and eTheRNA immunotherapies collaborate on mRNA vaccines for infectious diseases
Tools against tumors
Tools against tumors
Grant from Innovate UK to Pathios seeks to advance cancer immunotherapy
Novel SARS-CoV-2 neutralizing antibodies
Novel SARS-CoV-2 neutralizing antibodies
Therapeutic candidates retain binding to major COVID-19-causing “South African” variant
Preclinical
Two novel chronic kidney disease candidates arise
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.
Alternating two-vector therapies prove promising
Alternating two-vector therapies prove promising
Data on HOOKIPA’s cancer therapeutics have been published in Cell Reports Medicine
A double-barrel approach
A double-barrel approach
Peptide drug targets two cancer proteins for increased specificity and uptake
Lifelike systems
Lifelike systems
Organ-on-chip and tissue-on-chip models continue to advance drug discovery
Diagnostics
Algorithm could predict acute kidney injury
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).
Information integration
Information integration
Genomenon and BC Platforms seek to combine their tech platforms to boost genomic research
Molecular diagnostic and antibody research get a boost
Molecular diagnostic and antibody research get a boost
Yurogen and Asuragen acquisitions expected to aid wide range of researchers
AI against Parkinson’s disease
AI against Parkinson’s disease
New AI tool to be tested in NIH-funded study to improve diagnosis of Parkinson’s and related disorders
A new spinout for healthcare AI
A new spinout for healthcare AI
The Quinten Group spins off Quinten Health, using AI for precision care and real-world data science
Clinical Trials
Encouraging results for tirzepatide in diabetes
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
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
Fighting fungal infections
Oral ibrexafungerp shows positive results in Phase 3 study against difficult fungal infections
A type 1 diabetes game-changer
A type 1 diabetes game-changer
Promising follow-up data from TN-10 teplizumab study published in Science Translational Medicine
Tools & Technology
From astronomy to diagnostics?
From astronomy to diagnostics?
Microcombs could be used in many ways, including detecting disease and finding new planets
Real-world data to the rescue
Real-world data to the rescue
EVERSANA launches chronic disease real-world data solution to improving patient outcome
A “cookie cutter” technique for COVID drug discovery
A “cookie cutter” technique for COVID drug discovery
“Beyond COVID-19, we believe SMAs have huge potential to unblock constrictions in the drug pipeline.”
