Digital model simulates the first fully functioning living cell
Entering a cell and watching its entire inner machinery at work, how DNA is copied, how proteins are assembled, or how it splits in two, has been, for decades, an impossible dream. Now, scientists at the University of Illinois have recreated everything that happens inside a cell at molecular scale in an unprecedented computational model. Syn3A is the first 4D digital cell, capable of combining time and space to simultaneously represent all the internal processes that drive the life cycle of a minimal prokaryotic organism.

Kyron-Servier deal focused on programmable glycoprotein platform
Newco Kyron Bio is showing its colors after sealing a research agreement with Servier SA, underpinning a claim that it has overcome one of the longest-running challenges of the biotech industry, and developing technology to precisely control the glycosylation of therapeutic proteins.

Digital model simulates the first fully functioning living cell
Entering a cell and watching its entire inner machinery at work, how DNA is copied, how proteins are assembled, or how it splits in two, has been, for decades, an impossible dream. Now, scientists at the University of Illinois have recreated everything that happens inside a cell at molecular scale in an unprecedented computational model. Syn3A is the first 4D digital cell, capable of combining time and space to simultaneously represent all the internal processes that drive the life cycle of a minimal prokaryotic organism.
Kyron-Servier deal focused on programmable glycoprotein platform
Newco Kyron Bio is showing its colors after sealing a research agreement with Servier SA, underpinning a claim that it has overcome one of the longest-running challenges of the biotech industry, and developing technology to precisely control the glycosylation of therapeutic proteins.
Unixell Biotech’s UX-GIP001 gains IND clearance for epilepsy
Shanghai Unixell Biotechnology Co. Ltd. has obtained IND clearance from the FDA for UX-GIP001, its iPSC-derived allogeneic cell therapy for focal epilepsy. A phase I study will evaluate UX-GIP001 in patients with drug-resistant epilepsy.
China NMPA clears IND for Akeso’s trispecific antibody
Akeso Inc.’s first-in-class trispecific antibody, AK-150, has received IND clearance from China’s National Medical Products Administration (NMPA) for clinical trials in patients with advanced solid tumors. Engineered using Akeso’s AI-driven drug discovery platform and its proprietary Tetrabody technology, AK-150 is a humanized anti-CSF-1R, ILT2 and ILT4 trispecific antibody that achieves multipathway blockade of both innate and adaptive immunity.
Roche identifies SIK inhibitors
An F. Hoffmann-la Roche Ltd. and Hoffmann-la Roche Inc. patent describes prodrugs of serine/threonine-protein salt-inducible kinases (SIK) inhibitors. They are reported to be useful for the treatment of atherosclerosis, type 2 diabetes, giant cell arteritis, glomerulonephritis, inflammatory bowel disease, metabolic dysfunction-associated steatohepatitis (MASH; NASH), primary sclerosing cholangitis and rheumatoid arthritis.

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Conferences

First-in-class POLG activator restores mtDNA across mutations
Genetic/congenital
Researchers from Pretzel Therapeutics Inc. presented preclinical data of PX-578, a first-in-class POLG activator aimed at restoring mtDNA replication and mitochondrial function independently of mitochondrial DNA depletion syndromes genotype.
Chemicare’s CIC-39 shows promise for DMD treatment
Genetic/congenital
Researchers from Chemicare Srl and the University of Piemonte Orientale have presented preclinical results regarding their (SOCE) negative regulator CIC-39. Researchers evaluated the dysregulation of SOCE in both ex vivo and in vivo models of Duchenne muscular dystrophy (DMD), as well as evaluated...
Enanta Pharmaceuticals presents data for KIT inhibitor EDP-978
Dermatologic
The activation of mast cells in the lungs and skin is involved in diseases such as asthma and chronic spontaneous urticaria, where the KIT receptor tyrosine kinase is a key regulator of mast cell function.
hATG SAB-142 shows favorable PK and safety data
Endocrine/metabolic
Researchers from Sab Biotherapeutics Inc. presented data generated in the development and validation of a pharmacokinetic (PK) assay for SAB-142 and the evaluation of its toxicology and immunologic effects in a juvenile cynomolgus monkey model.

Cancer

Enodia acquires Sec61 assets from Kezar Life Sciences
Deals and M&A
Enodia Therapeutics SAS has acquired Kezar Life Sciences Inc.’s assets from its Sec61-based discovery and development program, including clinical-stage KZR-261. Insights from Kezar’s Sec61-based programs will further strengthen Enodia’s core focus on Sec61-driven selectivity for targeting protein...

Infection

Aptar and Covirix Medical collaborate on inhaled antivirals
Collaboration
Aptar Pharma, part of Aptargroup Inc., has established a technical collaboration with Covirix Medical Pty Ltd. under a letter of intent to develop inhaled antiviral treatments.

Shionogi patents new agents for HIV-1 infection
HIV/AIDS
Shionogi & Co. Ltd. has disclosed heterocyclic derivatives characterized as reverse transcriptase/ribonuclease H (HIV-1) inhibitors for potential use in the treatment of HIV infection.
Atea Pharma presents data on compounds for hepatitis E
Conferences
Atea Pharmaceuticals Inc. recently presented preclinical data on two of its compounds, AT-2490 and AT-587, which have shown promising antiviral potential for treating hepatitis E virus infection.

Neurology/psychiatric

AAV-based gene therapy for GBA1-related diseases, including Parkinson’s and Gaucher
Genetic/congenital
Glucocerebrosidase (GCase), encoded by the gene GBA1, is a ubiquitous lysosomal enzyme that breaks down lipid substrates, glucosylceramide (GL-1) and glucosylsphingosine (Lyso-GL1), into glucose and ceramide. Loss-of-function mutations in GBA1 reduce GCase activity, resulting in lipid accumulation...
Keros reports beneficial effects of RKER-065 in ALS model
Conferences
Keros Therapeutics Inc. has presented data regarding their activin receptor ligand trap, RKER-065, for the inhibition of the activin/myostatin signaling axis.
MJFF grants support Casma’s TRPML1 agonist CSM-101
Financings
Casma Therapeutics Inc. has been awarded approximately $7.6 million in funding across two competitive grant programs from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to support Casma’s lead program, CSM-101.
Acurastem awarded grant to advance SYF2-targeted ALS therapeutics
Financings
Acurastem Inc. has received a two-year research grant from Target ALS to advance therapeutics targeting SYF2, a recently identified regulator of TDP-43 function. TDP-43 dysfunction is a central biological hallmark of amyotrophic lateral sclerosis (ALS).
SGT-212 restores FXN function in Friedreich’s ataxia models
Conferences
Friedreich’s ataxia (FA) is an inherited neurodegenerative disorder caused by GAA repeat expansions in the FXN gene, which produces a mitochondrial protein vital for iron-sulfur cluster assembly and energy metabolism. Researchers at Solid Biosciences Inc. presented preclinical data supporting the...

Endocrine/metabolic

Med14 essential for GLP-1 agonist response, study finds
Drugs that mimic GLP-1 are widely used to treat diabetes and obesity, but it is not fully understood exactly how they produce many of their beneficial effects. Investigators from The Salk Institute for Biological Studies published a paper in Proceedings of the National Academy of Sciences shedding...

Biomarkers

GWAS identifies variant associated with diabetic retinopathy and susceptibility to T2D
GWAS
Scientists at the University of Nottingham Ningbo China and collaborators aimed to identify genetic variants associated with diabetic retinopathy.

IL-22 and TL1A, a robust couple for diagnosing hidradenitis suppurativa
Conferences
Hidradenitis suppurativa (HS) is a chronic inflammatory skin disease with strong association with psoriasis and inflammatory bowel disease (IBD). While some signaling pathways are well defined in HS, the role of TNF-like ligand 1 (TL1A) is not well understood. A group of researchers has...
TL1A is overexpressed in hidradenitis suppurativa
Conferences
Despite the availability of advanced therapeutic options, about 40%-50% of patients with hidradenitis suppurativa do not achieve significant improvement in disease activity, thus there is a need for novel medications.

Gastrointestinal

KIST and Neocannbio report new NO production inhibitors
Patents
The Korea Institute of Science and Technology (KIST) and Neocannbio Co. Ltd. have jointly identified compounds acting as nitric oxide (NO) production inhibitors with potential for the treatment of inflammatory bowel disease (IBD).
GPR68 negative allosteric modulator ameliorates ulcerative colitis
Conferences
G-protein coupled receptor 68 (GPR68) is an acid-sensing protein receptor that has been implicated in inflammatory bowel disease (IBD) pathogenesis by its modulation of the inflammatory response and fibrosis.
SIK2 inhibitor mitigates pathology in ulcerative colitis models
Conferences
Salt-inducible kinase 2 (SIK2) is a serine/threonine kinase that regulates transcriptional programs in myeloid cells by phosphorylating transcriptional coregulators. In macrophages, SIK2 promotes the expression of proinflammatory cytokines and...
Targeting OGFOD1 improves hepatic ischemia-reperfusion injury
Cardiovascular
Hepatic ischemia-reperfusion injury (HIRI) is a severe pathologic condition associated with poor outcomes when individuals suffer from hemorrhagic shock, liver resection or transplantation surgery, leading to severe liver impairment and sometimes...
Spyre reports enhanced efficacy of anti-TL1A and anti-IL-23 combination
Conferences
Researchers from Spyre Therapeutics Inc. reported on the therapeutic efficacy of combining anti-TL1A and anti-IL-23 antibodies in preclinical models of colitis.
GS-8670 restores epithelial barrier function in IBD models
Conferences
The farnesoid X receptor (FXR) is a nuclear receptor that plays a central role in bile acid regulation, intestinal barrier integrity, immune modulation and microbiome balance, all key factors involved in the development of inflammatory bowel...

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