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CRISPR–Cas3 deletes mutant TTR in vivo — large deletions lower serum TTR ~80%
Researchers evaluated CRISPR–Cas3 as an alternative genome‑editing strategy to induce long deletions at the transthyretin (TTR) locus and achieved near‑complete loss of expression in vitro and...
St. Jude’s CHANGE‑seq‑BE exposes base‑editor off‑targets — supports clinical safety cases
St. Jude researchers unveiled CHANGE‑seq‑BE, a genome‑wide, unbiased method to map off‑target activity induced by base editors. The approach prioritizes sensitivity and resource efficiency to...
Inhalable bispecific exosomes activate T cells — preclinical reversal of ICI resistance
An engineered inhalable exosome platform delivered a bispecific T‑cell activator to pembrolizumab‑resistant metastatic melanoma models and restored antitumor T‑cell activity. The work demonstrates...
CRISPR knockout screens map neuronal differentiation — PEDS1 linked to new disorder
A genome‑wide CRISPR knockout screen identified 331 genes essential for converting embryonic stem cells into neurons and implicated PEDS1 in a previously undescribed neurodevelopmental disorder....
Sanofi buys Dynavax for $2.2bn – bolsters adult immunization
Sanofi agreed to acquire Dynavax Technologies for about $2.2 billion in cash, adding the marketed hepatitis B vaccine HEPLISAV‑B and a phase I/II shingles candidate to its adult immunization...
Moderna seeks mRNA flu approvals: files dossiers across major markets
Moderna has submitted regulatory applications for mRNA‑1010, its seasonal influenza vaccine built on mRNA technology, to regulators in the U.S., Europe, Canada and Australia. The company reported...
Wegovy pill hits U.S. pharmacies: pricing and access move to center stage
Novo Nordisk has begun shipping the oral form of semaglutide branded as Wegovy to U.S. pharmacies, prompting immediate questions about pricing and access. The company said prescriptions could be...
Zenas' phase‑3 win sparks selloff – markets punish cross‑trial comparisons
Zenas Biopharma reported that obexelimab met the primary endpoint in the phase 3 Indigo trial for immunoglobulin G4‑related disease (IgG4‑RD), reducing flare risk by 56% versus placebo over 52...
Singapore’s Deep Intelligent Pharma raises $50M – aims to automate clinical trials
Deep Intelligent Pharma (DIP) closed a $50 million Series D round led by CDH Baifu to expand its autonomous clinical trial automation platform and global commercialization network. The company...
Insilico inks $888M pact with Servier – AI fuels oncology drug discovery
Insilico Medicine signed a multiyear R&D collaboration with Servier valued at up to $888 million to apply Insilico’s AI discovery platform to oncology targets. The agreement includes up‑front and...
Sanofi doubles down on Earendil: second AI biologics pact could top $2.5bn
Sanofi expanded its collaboration with AI‑driven biologics developer Earendil Labs with a follow‑on agreement worth up to $160 million in near‑term checks, supplementing an earlier potential $2.56...
CRISPR–Cas3 deletes TTR in vivo – long‑range edits cut serum TTR dramatically
Researchers reported use of CRISPR–Cas3, a mechanistically distinct genome‑editing system that produces long‑range deletions, to target the transthyretin (TTR) locus in mice. Delivered via lipid...
St. Jude unveils CHANGE‑seq‑BE: a rapid safety assay for base editors
Scientists at St. Jude Children’s Research Hospital developed CHANGE‑seq‑BE, an unbiased, sensitive method to map genome‑wide off‑target activity induced by base editors. Reported in Nature...
Inhalable bispecific exosomes reprogram T cells – activity in ICI‑resistant melanoma
A Nature Biotechnology paper described engineered inhalable exosomes carrying a bispecific T‑cell activator that, when delivered to the lung, activated antitumor T cells and suppressed...
Deep Intelligent Pharma Raises $50M — AI to automate clinical trials
Singapore-based Deep Intelligent Pharma (DIP) closed a $50 million Series D to scale an AI-driven platform that automates clinical trial design, execution and regulatory-readiness. The company...
Insilico and Servier Ink $888M Oncology Pact — AI discovery scales
AI drug-discovery company Insilico Medicine signed a multiyear R&D collaboration with French pharma Servier worth up to $888 million to jointly discover and develop oncology candidates using...
Sanofi doubles down on AI biologics — repeat pacts with Earendil
Sanofi expanded its relationship with AI-driven biologics developer Earendil Labs through a new agreement worth up to $160 million in near-term checks, adding to a prior Sanofi–Earendil pact that...
Wegovy pill launches in U.S. — pricing and access front and center
Novo Nordisk began shipping an oral formulation of semaglutide (Wegovy pill) in the U.S.; the company and pharmacies reported immediate dispensing for patients with prescriptions. The oral product...
Sanofi to buy Dynavax for $2.2B — adds HEPLISAV‑B and shingles candidate
Sanofi agreed to acquire Dynavax Technologies for approximately $2.2 billion in cash, a deal that adds the approved two-dose adult hepatitis B vaccine HEPLISAV‑B and a Phase I/II shingles vaccine...
Zenas’ phase III win in IgG4‑RD sinks stock — investors eye cross‑trial read
Zenas Biopharma reported positive Phase III Indigo results for obexelimab in immunoglobulin G4‑related disease (IgG4‑RD), meeting the primary endpoint with a 56% reduction in flare risk versus...