ProQR

Intellia Therapeutics is restructuring, discontinuing its NTLA-3001 program for alpha-1 antitrypsin deficiency, laying off over 25% of staff, and focusing on NTLA-2002 and nex-z for hereditary angioedema and transthyretin amyloidosis. Laura Sepp-Lorenzino retires as CSO, succeeded by Birgit Schultes.

Nuvig Therapeutics closed $161M Series B to advance NVG-2089 for autoimmune dysregulation. Adcendo raised $135M Series B for ADC pipeline. Enveda secured $130M Series C for AI, natural compounds platform. Angitia Biopharmaceuticals closed $120M Series C for musculoskeletal disease treatments. Maze Therapeutics raised $115M Series D for kidney disease treatments. Citryll closed €85M Series B for NETs antibody. Antag Therapeutics secured €80M Series A for GLP-1 alternatives. Beta Bionics raised $60M Series E for insulin delivery system. ATB Therapeutics received €54M Series A for antibody platform. 35Pharma closed $53M Series C for TGF-beta and protein engineering. Allink Biotherapeutics raised $42M Series A for global expansion of bispecific antibody and ADC pipeline. KisoJi Biotechnology secured $41M for TROP2 antibody to treat solid tumors. AQEMIA closed $38M for teaching physics to generative AI platform. Noema Pharma raised CHF 27M Series B for trigeminal neuralgia, tuberous sclerosis complex. Valora closed seed funding for gluco-immune checkpoint platform. Biomemory raised $18M Series A for data storage using molecular technology. Celosia Therapeutics completed $16.7M Series A for ALS gene therapy. Qnovia secured $16M Series B for nicotine inhaler. Alessa Therapeutics closed $15M seed financing for prostate cancer treatment. Syncell raised $15M Series A for protein purification and spatial proteomics technology. Doron Therapeutics secured $11M Series A for regenerative osteoarthritis drug. DeepLife raised $10M Series A for AI-powered models of human cells. ProQR expanded partnership with Rett Syndrome Research Trust for $8.1M. Converge Bio secured $5.5M seed financing for GenAI in drug discovery. Alveron Pharma closed €5M Seed extension financing for intracranial hemorrhage.

Nuvig Therapeutics secured $161M Series B for NVG-2089 clinical trials. Adcendo raised $135M Series B for ADC pipeline. Enveda's $130M Series C funds AI-driven natural compounds research. Angitia Biopharmaceuticals' $120M Series C supports musculoskeletal treatments. Maze Therapeutics' $115M Series D advances kidney disease therapies. Citryll's €85M Series B funds CIT-013 development. Antag Therapeutics' €80M Series A supports GLP-1 alternatives. Beta Bionics' $60M Series E expands insulin delivery system. ATB Therapeutics' €54M Series A accelerates antibody platform. 35Pharma's $53M Series C advances TGF-beta inhibitors. Allink Biotherapeutics' $42M Series A funds global expansion. KisoJi Biotechnology raised $41M for solid tumor antibody. AQEMIA's $38M funding advances AI in drug discovery. Noema Pharma's CHF 27M Series B supports Phase 2 trials. Valora's seed funding develops AbLec platform. Biomemory's $18M Series A transforms data storage. Celosia Therapeutics' $16.7M Series A advances ALS gene therapy. Qnovia's $16M Series B funds nicotine inhaler. Alessa Therapeutics' $15M seed financing advances prostate cancer treatment. Syncell's $15M Series A expands protein purification tech. Doron Therapeutics' $11M Series A funds osteoarthritis drug trial. DeepLife's $10M Series A develops AI-powered cell models. ProQR's $8.1M funding supports Rett Syndrome research. Converge Bio's $5.5M seed financing advances GenAI in drug discovery.

WaVe Life Sciences Ltd. (NASDAQ:WVE) focuses on RNA editing technology for genetic diseases, with a promising DMD program showing 53% exon skipping. The company faces competition from firms like Sarepta Therapeutics and KRRO, but recent analyst price targets suggest growing confidence. WaVe's future hinges on clinical trial success and strategic partnerships, particularly in RNA editing, which is seen as a transformative technology in genetic medicine.

Wave Life Sciences' clinical trial results show RNA editing success in treating alpha-1 antitrypsin deficiency, with significant increases in AAT protein levels and no serious side effects, marking a milestone for RNA editing technology.