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Integrated Biosciences unveils precision optogenetic drug screening platform targeting age-related diseases
Integrated Biosciences published a Cell paper detailing a novel optogenetic screening platform that enables millisecond precision activation of the integrated stress response (ISR) pathway,...
Genetic variability shapes tumor immunotherapy response in mouse models
Using the genetically diverse Collaborative Cross mouse model, researchers demonstrated that genetic variability influences tumor response to immune checkpoint inhibitors. This work, published in...
Ultragenyx Gene Therapy Rejection Hits Rare Disease Program
The U.S. Food and Drug Administration issued a complete response letter (CRL) to Ultragenyx Pharmaceutical, declining approval for its gene therapy UX111 targeting Sanfilippo syndrome Type A, a...
AstraZeneca's Hypertension Drug Baxdrostat Shows Phase III Success
AstraZeneca reported positive phase III data for baxdrostat, an aldosterone synthase inhibitor acquired through its 2023 purchase of CinCor Pharma. The drug met primary and secondary endpoints by...
Takeda Advances Narcolepsy Treatment with Phase III Wins for Oveporexton
Takeda Pharmaceutical announced successful results from two Phase III studies evaluating its oral orexin receptor 2-selective agonist, oveporexton, for narcolepsy type 1. The drug met all primary...
Mink Therapeutics’ NK T-Cell Therapy Yields Durable Cancer Remission
Mink Therapeutics reported a complete and durable remission in a testicular cancer patient treated with its allogeneic invariant natural killer T-cell therapy Agent-797 combined with nivolumab....
AI Uncovers Novel Antibiotics in Venoms to Combat Resistance
Researchers harnessed artificial intelligence to identify hundreds of potential antibiotics from snake, scorpion, and spider venoms, offering a new avenue to tackle the rising global threat of...
Viome, Scripps Collaborate on At-Home RNA Test for Colon Cancer Detection
Viome Life Sciences partnered with Scripps Research to develop and validate an at-home RNA-based screening test aimed at detecting precancerous colon polyps, facilitating early prevention of...
Cellular Origins-Led Consortium Targets Automated Cell Therapy Manufacturing
Cellular Origins, Resolution Therapeutics, and Cell and Gene Therapy Catapult formed a consortium awarded £1 million by Innovate UK to develop a fully automated, scalable manufacturing platform...
Private Equity Fuels Expansion at PCI Pharma Services in Drug Production
Private equity firms including Bain Capital and Kolberg notably invested in PCI Pharma Services, valuing the company at $10 billion. The contract development and manufacturing organization...
Circular Genomics and WashU Identify Circular RNA Biomarker for Alzheimer’s
Circular Genomics, collaborating with Washington University, discovered a circular RNA-based signature, including circHOMER1, associated with Alzheimer’s disease onset and progression. This...
Nutcracker and Elegen Pilot Cell-Free RNA Manufacturing for Personalized Cancer Therapies
Nutcracker Therapeutics and Elegen initiated a pilot for a fully synthetic, cell-free manufacturing platform to produce RNA-based personalized cancer therapeutics (PCTs). The platform addresses...
AstraZeneca’s blood pressure breakthrough validates $1.3B CinCor acquisition
AstraZeneca’s experimental drug baxdrostat demonstrated statistically significant reductions in systolic blood pressure among patients with uncontrolled or resistant hypertension in a pivotal...
Takeda’s orexin agonist posts twin phase 3 wins – Narcolepsy filing imminent
Takeda’s novel orexin receptor 2 selective agonist, oveporexton, achieved all primary and secondary endpoints in two pivotal Phase 3 narcolepsy studies. The oral drug demonstrated clinically...
FDA rejects Ultragenyx gene therapy – Manufacturing flaws halt Sanfilippo treatment
The FDA issued a complete response letter declining approval of Ultragenyx’s gene therapy UX111 for Sanfilippo syndrome Type A, citing manufacturing process issues. The regulatory setback arises...
Private equity fuels PCI Pharma’s global drug production expansion at $10B valuation
PCI Pharma Services secured new investments from Bain Capital, Kolberg, and Mubadala, valuing the contract development and manufacturing organization (CDMO) at approximately $10 billion. The funds...
Viome and Scripps partner on at-home RNA colon cancer screening – 1000-patient trial set
Personalized medicine firm Viome and Scripps Research launched a collaboration to develop and clinically validate a non-invasive RNA-based test for early detection of precancerous colon polyps....
Integrated Biosciences unveils optogenetic platform – New path for targeted drug discovery
Biotech startup Integrated Biosciences introduced an innovative optogenetic screening platform integrating chemistry and AI to discover small molecule modulators with precision. Demonstrated...
Genomic AI breakthrough: DeepMind’s AlphaGenome decodes noncoding DNA impact
DeepMind’s AlphaGenome AI system can analyze up to one million DNA base pairs to predict functional effects of noncoding genetic variations linked to diseases like cancer and rare disorders. This...
Cancer immunotherapy response shaped by tumor genetics – Mouse model study reveals
Researchers using genetically diverse Collaborative Cross mice demonstrated that inherited genetic variability significantly influences tumor response to immune checkpoint inhibitors. This...