Deepmind’s AI model predicts the effect of variants in dark genome
Google Deepmind is shedding light on the dark genome with its latest AI model, which is trained to decipher the 98% of DNA that does not code for proteins. Alphagenome is designed to predict how variants in the regulatory genome exert their effects on the expression of the genes they control.

Deepmind’s AI model predicts the effect of variants in dark genome
Google Deepmind is shedding light on the dark genome with its latest AI model, which is trained to decipher the 98% of DNA that does not code for proteins. Alphagenome is designed to predict how variants in the regulatory genome exert their effects on the expression of the genes they control.
Seamless seals $1.12B gene editing deal with Lilly on hearing loss
Seamless Therapeutics has received big pharma endorsement of its proprietary recombinase gene editing platform, sealing a potential $1.12 billion deal with Eli Lilly and Co. to apply the technology in hearing loss.
Siren Biotechnology’s glioma program gains IND clearance
Siren Biotechnology Inc. has obtained IND approval from the FDA enabling the initiation of its first-in-human trial for its lead investigational program SRN-101 in adult patients with recurrent high-grade glioma.
Therapy restores cognition after symptom onset in tauopathy mice
Several neurodegenerative diseases, including Alzheimer’s disease, frontotemporal dementia and progressive supranuclear palsy, are classified as tauopathies due to the pathological accumulation of tau protein in specific brain nuclei. Researchers in Argentina have proposed the use of RNA interference (RNAi)-mediated therapies using viral vectors to target tau.
Chia Tai Tianqing Pharmaceutical Group patents ERα-targeting PROTACs
Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has disclosed proteolysis targeting chimeric (PROTAC) compounds comprising a E3 ubiquitin ligase-binding moiety covalently linked to an estrogen receptor-α (ERα)-targeting moiety. They are reported to be useful for the treatment of breast cancer.
SCN2A Foundation and Unravel Biosciences collaborate
The SCN2A Foundation has entered into a research collaboration with Unravel Biosciences Inc. to advance preclinical research for SCN2A-related disorders caused by loss-of-function mutations, a subset of SCN2A conditions driven by insufficient functional protein.

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Conferences

KLF15 shows potential as marker, target for cholangiocarcinoma
EASL
Researchers at the Biodonostia Health Research Institute reported on the role of KLF15 in cholangiocarcinogenesis and its potential as a therapeutic target in this disease.
Serotonin receptor modulators for the treatment of OUD
Substance use and poisoning
Opioid use disorder (OUD) causes high morbidity and mortality rates, with fentanyl driving unprecedented overdose rates. Researchers from the University of California have used an AI-based drug discovery platform with the aim of identifying preclinical drug candidates for OUD.
Study identifies CAND1 as therapeutic target in hepatoblastoma
EASL
Hepatoblastoma is the most common liver cancer during childhood, with limited therapeutic options in aggressive or relapsed cases. NEDDylation is a post-translational modification that modulates cullin-RING ligases and has arisen as a crucial regulator of protein turnover in cancer.
5-HT2R allosteric ligands attenuate cocaine use and seeking behavior
Substance use and poisoning
Serotonergic G-protein-coupled receptors (GPCRs), including the 5-HT2A receptor (5-HT2AR) and 5-HT2CR, are key regulators of cortical signaling pathways and promising targets for neurotherapeutic drug discovery. Researchers from the University of Texas System have developed AB-0124, a 5-HT2AR...

Cancer

Selective CAR T targeting mutant CALR shows preclinical activity against MPNs
CAR T
Despite the successful application of adoptive T-cell transfer with chimeric antigen receptor (CAR)-engineered T cells for the treatment of various hematologic malignancies, several other hematologic disorders, such as BCR::ABL1-negative myeloproliferative neoplasms (MPNs), still lack effective...

Infection

New cross-reactive human mAb protects against RSV and hMPV challenge in mice
Monoclonal antibody
Human respiratory syncytial virus (RSV) and human metapneumovirus (hMPV) can cause severe and lethal bronchiolitis and pneumonia among particularly vulnerable populations, mostly infants, the elderly and immunocompromised patients. Researchers from Vanderbilt University Medical Center have isolated...

Age impacts disease tolerance to sepsis, study shows
The immune system is a critical factor of host survival, allowing resistance to infections and maintaining tissue integrity. The activation of immune responses requires precise regulation to assure a balance between the benefits and costs of these responses. Moreover, the theory of antagonistic...
Centre Hospitalier Regional Universitaire de Lille divulges new 3CLpro inhibitors
Coronavirus
Centre Hospitalier Regional Universitaire de Lille has patented new 3C-like proteinase (3CLpro; Mpro; nsp5) (SARS-CoV-2; COVID-19 virus) inhibitors reported to be useful for the treatment of SARS-CoV-2 infection (COVID-19).

Neurology/psychiatric

O-Alkylated piperine derivative inhibits Nav1.7, shows antinociceptive effects
Voltage-gated sodium channels, particularly Nav1.7, are highly expressed in peripheral sensory neurons and are crucial for pain signal conduction and signal transmission in the spinal dorsal horn. Because of their role in triggering pain, these channels are considered critical targets for...
Harmine-based compounds emerge as brain-penetrant GSK-3β inhibitors
The serine/threonine kinase glycogen synthase kinase-3β (GSK-3β) plays a multifunctional role through its involvement in multiple signaling pathways. Because of its relevant role in Alzheimer’s disease (AD) pathogenesis, regulating GSK-3β activity has been proposed as a potential approach to target...
Mair Therapeutics and Radboud collaborate in Parkinson’s
Collaboration
Mair Therapeutics BV has established a scientific collaboration with Radboud University to accelerate the discovery of small-molecule agonists of TMEM175, a lysosomal ion channel genetically linked to Parkinson’s disease.
US Dept. of HHS synthesizes new dopamine D2 antagonists
Patents
U.S. Department of Health and Human Services (HHS) has discovered dopamine D2 receptor antagonists potentially useful for the treatment of Tourette disease, bipolar disorder, tardive dyskinesia, Huntington’s disease, schizophrenia, depression, postoperative nausea and vomiting and gastroesophageal...
REST emerges as biomarker in ALS, knockdown improves ALS symptoms
Biomarkers
About 10% of amyotrophic lateral sclerosis (ALS) cases result from inherited genetic mutations, with about 20% of them attributed to mutations in the gene encoding the ubiquitous cytoplasmic copper/zinc superoxide dismutase 1 (SOD1).

Endocrine/metabolic

Tetrapharm advances TPC-026 for chronic use in obesity
Tetrapharm (Tetra Pharm Technologies ApS) has announced promising preclinical data for TPC-026 for the chronic treatment and long-term management of metabolic disorders, including obesity. The preclinical findings support TPC-026 as a differentiated therapeutic candidate, designed to address...

Biomarkers

MicroRNAs in circulating extracellular vesicles as IPF biomarkers
Inflammatory
MicroRNAs (miRNAs) are small noncoding RNAs gaining increasing attention due to their crucial role in gene expression regulation and influence in various cellular processes and diseases. miRNAs can be encapsulated in extracellular vesicles (EVs), which are released by most cell types to modulate...

Brain-derived tau in blood predicts stroke severity and outcome
Cardiovascular
Brain-derived tau, a protein that is exclusive to the brain and detectable in the blood, could serve as an indicator of brain damage after an ischemic stroke. The analysis of this special form of tau has revealed a relationship between high levels of the protein and extensive brain injury, a higher...
Biallelic variants in COX18 identified as cause of Charcot-Marie-Tooth disease
Genetic/congenital
Charcot-Marie-Tooth (CMT) disease is a group of clinically and genetically heterogeneous sensorimotor peripheral neuropathies. It is the most frequent inherited neuromuscular disorder affecting 9.7-82.3 patients per 100,000 individuals. Over 100 genes with all patterns of inheritance have been...

Gastrointestinal

Boehringer Ingelheim and Simcere partner on SIM-0709
Deals and M&A
Boehringer Ingelheim International GmbH and Simcere Pharmaceutical Group Ltd. have entered into a license and collaboration agreement to develop SIM-0709 for the treatment of inflammatory bowel disease.
RIPK1 arises as target in primary sclerosing cholangitis
Inflammatory
Increasing evidence exists regarding receptor-interacting protein kinase 1 (RIPK1), a necroptosis regulator, being involved in inflammation and fibrosis in chronic liver disorders. The relationship between necroptosis and the inhibition of RIPK1...
Nanjing Chia Tai Tianqing Pharmaceutical identifies new somatostatin SST2 receptor agonists
Patents
Nanjing Chia Tai Tianqing Pharmaceutical Co. Ltd. has discovered somatostatin SST2 receptor agonists described as potentially useful for the treatment of acute pancreatitis and peptic ulcer bleeding.
First circRNA-based protein replacement therapy for liver fibrosis
Researchers from Shanghai Institute of Nutrition and Health and Guangming Advanced Research Institute (China) proposed a new strategy based on a circular RNA (circRNA) encoding human relaxin-2 (cRLN2). circRNAs, thanks to their high stability and...
SLIT2 shows promise as a druggable target in liver injury
About 10% of acute hepatitis cases and 50% of acute liver failure are caused by drug induction, where treatment remains largely limited. The SLIT/ROBO signaling axis is composed of the secretory SLIT proteins (SLIT1, SLIT2 and SLIT3) and their...
Fat tissue subtyping gives functional insights
Science
Once it was considered to be more or less a passive energy-storing device that could double as a cushion. But increasingly, fat is conceptualized as an endocrine organ as much as a tissue type. Now, separate research groups have reported new...

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