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BioWorld - Friday, December 5, 2025
Home » Newsletters » BioWorld Science

BioWorld Science

Jan. 8, 2025

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Celosia team

Australia’s Celosia heads toward clinic with gene therapy for ALS

After raising AU$16.75 million (US$10.4 million) in a series A round, Celosia Therapeutics Pty Ltd. is heading toward the clinic with its novel gene therapy that targets TDP-43, a protein directly linked to amyotrophic lateral sclerosis (ALS) pathology. Read More
Molecules and RNA enclosed by a lipid bilayer

Orna, Vertex next-gen sickle cell, thalassemia deal could reach $4.3B+

About four years after launch, Orna Therapeutics Inc. signed its second major deal, this time validating the lipid nanoparticle delivery technology it acquired through its Renagade Therapeutics Inc. buyout in May 2024, with Vertex Pharmaceuticals Inc. seeking next-generation approaches for hemoglobinopathies. Read More
Photo of older woman holding hand that's colored red

mPGES-1 inhibitor AGU-654 is anti-inflammatory and analgesic

Researchers from Gazi University and Friedrich-Schiller-Universität Jena presented the discovery and preclinical characterization of novel microsomal prostaglandin E2 synthase 1 (mPGES-1) inhibitors as potential anti-inflammatory and analgesic drug candidates. Read More
respiratory-red-lungs

SYH-2059 to enter trials in China for interstitial lung disease

CSPC Pharmaceutical Group Ltd. has obtained clearance from China’s National Medical Products Administration (NMPA) to conduct clinical trials in China of SYH-2059 tablets for interstitial lung disease, including idiopathic pulmonary fibrosis and progressive pulmonary fibrosis. Read More
2024 on teal background

2024 BioWorld Science Year in Review

Taking a look back at the achievements in research this year, including brain maps, pandemic breakthroughs and advances in pregnancy health. Read More
Euro-pig-bank.png

Series A at Neumirna supports RNA therapies for neurological disorders

A €20 million (US$20.6 million) series A financing round at Neumirna Therapeutics ApS is set to support the company’s development of RNA therapies for epilepsy, Parkinson’s disease and other neurological disorders, and enable the company to advance its lead asset into the clinic. Read More

Avenzo licenses ex-China rights to Duality’s EGFR/HER3 bispecific ADC

Avenzo Therapeutics Inc. and Duality Biotherapeutics (Suzhou) Co. Ltd. have entered into an exclusive license agreement. Read More

Nimbus Therapeutics patents new 15-hydroxyprostaglandin dehydrogenase inhibitors

Nimbus Therapeutics LLC has disclosed 15-hydroxyprostaglandin dehydrogenase inhibitors reported to be useful for the treatment of alopecia, muscle atrophy and more. Read More

SYS-6045 cleared for clinic in China for advanced solid tumors

CSPC Pharmaceutical Group Ltd.’s antibody-drug conjugate SYS-6045 has been granted clearance by China’s National Medical Products Administration (NMPA) to enter clinical trials. Read More

Interline Therapeutics divulges new RIPK2 inhibitors for IBD

Interline Therapeutics Inc. has synthesized receptor-interacting serine/threonine-protein kinase 2 (RIPK2; RIP-2) inhibitors reported to be useful for the treatment of inflammatory bowel disease (IBD). Read More
cardio-heart-attack.png

Cyclarity’s UDP-003 to enter clinic for acute coronary syndrome

Cyclarity Therapeutics Inc. has obtained regulatory approval to begin a first-in-human trial of UDP-003. 

Read More

Solid Biosciences gains IND clearance for Friedreich’s ataxia gene therapy

Solid Biosciences Inc. has gained IND clearance from the FDA for SGT-212 for the treatment of Friedreich’s ataxia (FA). Read More

Atai Life Sciences describes new monoamine transporter modulators

Atai Life Sciences has identified monoamine transporter modulators reported to be useful for the treatment of post-traumatic stress, eating disorders and obsessive-compulsive disorder. Read More
neurology-brain-protect.png

Novel NLRP3 inhibitor shows neuroprotective effect

Neuroinflammation is a key feature of Alzheimer’s disease (AD). The activation of the NLRP3 inflammasome has been associated with the pathogenesis of AD, therefore, targeting NLRP3 may help reduce neuroinflammation and slow down the progression of AD. Read More

New SIK inhibitors disclosed in Beigene patent

Scientists at Beigene Ltd. and Beigene Switzerland GmbH have divulged serine/threonine-protein kinase SIK inhibitors reported to be useful for the treatment of inflammatory and autoimmune diseases. Read More

By targeting HPA-1a, RLYB-212 prevents rare fetal blood disorder

Fetal/neonatal Alloimmune Thrombocytopenia (FNAIT) is a bleeding disorder caused by maternal alloantibodies against fetal platelets during pregnancy. Read More
Art concept for drug research

Novel PD-L1 degrader described in China

Immune checkpoint blockade is one of the major advances in cancer treatment in recent years. However, the majority of immune checkpoints are embedded in the cell membrane and this fact represents a limitation for targeted degradation using proteolysis-targeting chimeras (PROTACs). Read More

Yuhan discovers new HPK1 inhibitors

Yuhan Corp. has described pyrimidine/pyridine derivatives acting as mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1) inhibitors reported to be useful for the treatment of cancer. Read More

Other news to note for Jan. 8, 2025

Additional early-stage research and drug discovery news in brief, from: Biomea Fusion, Telomir Pharmaceuticals. Read More

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