Capsida Biotherapeutics, a gene therapy startup that has quietly amassed a network of Big Pharma partnerships, announced in January that its partner AbbVie will exercise its option on their first neurodegenerative disease program, triggering a $40 million license payment. The five-year-old company, which has built its war chest primarily through pharma collaborations rather than traditional…
How Dandelion Health’s multimodal dataset promises to shed light on GLP-1 research
Dandelion Health, the maker of a real-world data and clinical AI platform, has launched the first multimodal real-world clinical dataset focused on uncovering insights and opportunities related to GLP-1 receptor agonist drugs. The company describes the GLP-1 data library as “the first multimodal real-world clinical dataset” related to GLP-1 receptor agonists. The library is sourced…
Quantum-inspired power could brighten drug discovery
In the realm of drug discovery, the Harvard-educated chemist Nick Paras, Ph.D. compares the early stages of identifying new compounds to a game of Zelda, in which a player at times must navigate through dark rooms and solve puzzles to progress. Similarly, medicinal chemists and biochemists too must traverse the vast, often obscure landscape of…
Capsida Biotherapeutics to present preliminary findings on engineered AAV capsids at ASGCT annual meeting
The gene therapy platform company Capsida Biotherapeutics (Thousand Oaks, California) is gearing up to unveil initial data on its engineered adeno-associated virus (AAV) capsids at the upcoming American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, held May 16–20 in Los Angeles. In developing these capsids, Capsida Chief Scientific Officer Susan Catalano explained…
Brain breakthroughs: Aprinoia Therapeutics’ harnesses AI and strategic partnerships to propel neurodegenerative disease research
Neurodegenerative disease research is witnessing significant advances. To that end, Hong Kong-headquartered Aprinoia Therapeutics is embracing a ‘precision neuroscience’ approach to neurodegeneration diagnostics. The company’s lead program, APN-1607, represents a new generation of advanced tau positron emission tomography (PET) tracers, which play a crucial role in effective and efficient diagnosis of patients suffering from neurodegenerative…
Lonza and ABL Bio partner on bispecific antibody development for immuno-oncology and neurodegenerative diseases
Lonza (LON:0QNO) and South Korea-based ABL Bio (KOSDAQ:298380) have joined forces to accelerate bispecific antibody development for immuno-oncology and neurodegenerative diseases. Over email, Basel, Switzerland–headquartered Lonza noted that its experience with novel molecular formats enables the company to handle ABL Bio’s unique challenges. In the partnership, Lonza will provide submission-ready chemistry, manufacturing and controls (CMC) data…
QRL-201, a novel therapy targeting STMN2 expression, enters phase 1 ALS trial
Cambridge, Massachusetts-based QurAlis has dosed the first patient with the biotherapeutic QRL-201 in a Phase 1 clinical trial (ANQUR) in Canada. QRL-201 is a novel therapy targeting the restoration of STATHMIN-2 (STMN2) expression in amyotrophic lateral sclerosis (ALS) patients. QurAlis believes ANQUR the first study to evaluate such a treatment. Stathmin-2 (STMN2), also known as…
QurAlis targets UNC13A RNA mis-splicing in ALS and FTD with novel FlexASO platform
QurAlis, a privately-held company specializing in precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, has debuted its latest program. This initiative targets UNC13A RNA mis-splicing, a critical gene alteration present in ALS and frontotemporal dementia (FTD). FTD has recently gained renewed attention following the disclosure of Bruce Willis’ diagnosis of the condition.…
FDA launches 5-year initiative for rare neurodegenerative diseases
FDA has unveiled its Action Plan for Rare Neurodegenerative Diseases, including amyotrophic lateral sclerosis (ALS). The plan will involve the use of public-private partnerships and incorporate feedback from patients. The initiative will encompass regulatory science initiatives, changes to existing programs and new policy initiatives. Milestones of the initiative include the creation of an FDA rare neurodegenerative…
Scientists find new potential drug targets for neurodegenerative diseases
Researchers at Oregon State University (OSU) have discovered a new class of potential drug targets for diseases like Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis (ALS). The scientists are working to identify the best method to attack the targets — oxidized proteins. The most potent oxidant of the bunch is peroxynitrite, which is produced in conditions involving inflammation.…
What is needed to develop disease-modifying therapies for Parkinson’s and Alzheimer’s
Developing disease-modifying therapies for neurodegenerative diseases remains a pressing need. The incidence of neurodegenerative disease is ramping up in the U.S. and elsewhere as much of the global population ages. One out of three seniors dies with Alzheimer’s disease or another form of dementia, according to the Alzheimer’s Association. Parkinson’s disease is also becoming more…