Get the Daily Brief
Latest Biotech News
Clinical trial disruption for Aardvark’s Prader-Willi metabolic drug
Aardvark Therapeutics said the FDA placed a full clinical hold on ARD-101 for Prader-Willi syndrome after cardiovascular concerns emerged in a higher-dose healthy volunteer study. The hold halts...
Expansion of gene and RNA therapy momentum per ASGCT’s Q1 2026 landscape
ASGCT’s Q1 2026 landscape report points to continued growth in cell, gene, and RNA therapy development, including a 30% increase in startup funding versus Q1 2025. ASGCT CEO David Barrett said the...
Microbiome-derived therapeutics for liver fibrosis via immune modulation
Researchers reported that the gut bacterium Phocaeicola dorei can ease cholestatic liver fibrosis through efferocytosis-linked immune cell modulation. The work suggests a pharmabiotics approach...
Neuroscience biomarker genetics in ALS reveals peripheral-to-central immune sequence
A Northwestern University team mapped a “domino-like” immune cascade in ALS, linking early motor neuron changes to downstream inflammatory responses in blood and spinal cord. In the Nature...
Neurotechnology funding: ultrasound neuromodulation advances toward depression trials
Sonomind raised €20 million in a Series A round to advance its ultrasound-based neuromodulation device for depression. The company plans clinical trials using a non-invasive system with a custom...
In vivo CAR platform financing accelerates CREATE Medicines pipeline
CREATE Medicines closed a $122 million Series B to expand its in vivo immune programming platform across autoimmune disease and oncology. The company highlighted repeat-dose capabilities from its...
Regulatory and policy backdrop: EU Critical Medicines Act deal to tackle shortages
The EU reached agreement on the Critical Medicines Act aimed at reducing drug shortages by boosting European manufacturing capacity and enabling joint procurement. The package focuses on...
Drug discovery platform: DNA-guided CRISPR for cellular RNA targeting
Researchers at the University of Florida developed ΨDNA, a DNA-guided CRISPR–Cas12 system designed to target cellular RNA using DNA guide scaffolds rather than RNA guides. The approach aims to...
FDA greenlights ctDNA MRD-guided adjuvant immunotherapy in bladder cancer
The FDA approved Genentech’s Tecentriq (atezolizumab) and Tecentriq Hybreza (subcutaneous atezolizumab and hyaluronidase-tqjs) as adjuvant treatment for patients with muscle-invasive bladder...
Phase 3 readout for telitacicept in IgA nephropathy
RemeGen’s telitacicept delivered a strong interim efficacy signal in a prespecified Phase 3 analysis in high-risk IgA nephropathy, according to results presented at NEJM.org and associated meeting...
New gene therapy targeting liver for hemophilia A
Researchers reported efficacy improvements with two engineered AAV8 vectors—HMR-001 and its codon-optimized counterpart HMR-001z—in a hemophilia A gene therapy study focused on restoring...
Aardvark hits full FDA clinical hold after cardiovascular signal
FDA placed Aardvark Therapeutics’ Prader-Willi syndrome program under a full clinical hold, halting further testing of ARD-101 and related studies following a cardiovascular safety issue observed...
Gene therapy safety case raises AAV integration scrutiny
A CHOP-led team presented evidence of a brain tumor linked to rare adeno-associated virus (AAV) integration, highlighting a potential long-term risk to the AAV gene therapy field. The case,...
Telitacicept, Tecentriq, and beyond—drug pipeline momentum in gene and RNA therapy
ASGCT’s Q1 2026 Landscape Report pointed to accelerating regulatory and funding activity across gene and RNA therapies, with an increase in startup funding and a reported uptick in approval...
In-vivo CAR platform advances with $122M Series B funding
CREATE Medicines secured a $122 million Series B round to advance its in vivo CAR platform, with lead assets spanning autoimmune disease and oncology. The company’s proprietary mRNA-LNP platform...
Contract research and diagnostics: ARUP launches infectious disease testing dashboard
ARUP launched a public-facing infectious disease testing dashboard intended to provide near real-time signals on positivity rates across selected molecular assays. The reference lab said the...
Precision oncology: MYC linked to urothelial cancer mortality and microenvironment
Researchers tied urothelial carcinoma disease biology and outcomes to MYC overexpression, implicating a tumor-intrinsic mechanism for disease progression and treatment resistance. The work...
New in-orbit pharma manufacturing partnership
Varda Space Industries announced a partnership with United Therapeutics to test small-molecule drug development using Varda’s in-orbit experiment platform. Varda has been sending automated...
FDA precision oncology approval with ctDNA MRD testing
The FDA approved Genentech’s Tecentriq (atezolizumab) and Tecentriq Hybreza as adjuvant therapy for muscle-invasive bladder cancer patients who are ctDNA-positive after cystectomy, as determined...
Phase 3 trial results for telitacicept in IgA nephropathy
Telitacicept, a BAFF/APRIL-targeting fusion protein by RemeGen, delivered a significant reduction in proteinuria in an interim analysis of a Phase 3 trial in high-risk IgA nephropathy. The...