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CRISPR Just Graduated: First-Ever Phase 3 Win for a Gene-Editing Therapy Delivered Inside the Body
Intellia Therapeutics posted the first positive Phase 3 results ever for a CRISPR therapy delivered directly inside a patient, and the numbers are striking. A single IV infusion cut hereditary angioedema attacks by 87% versus placebo, with 62% of treated patients going completely attack-free for six months. Safety was clean, with no serious adverse events in the treatment arm. The FDA has already accepted a rolling filing with Priority Review, setting a March 2027 decision date. Shares jumped 23%.
Why it matters: This is to in vivo gene editing what the first SpaceX landing was to reusable rockets. It proves the model works, and it de-risks the entire field for every company building CRISPR therapies targeting cardiovascular disease, liver conditions, and beyond.
Read more →Oncology: Wins, Losses, and One Awkward Withdrawal
The 'Undruggable' Cancer Target Just Conquered Europe
European regulators recommended approval of Revolution Medicines' Rasonque for metastatic pancreatic cancer, less than two months after its FDA approval. The drug showed a 60% reduction in the risk of death versus chemo in Phase 3, nearly doubling survival in a disease where most patients live under a year. It's the first broad KRAS inhibitor to receive a positive regulatory recommendation in both the U.S. and Europe.
Read more →Daiichi Sankyo and Merck Pull Their Lung Cancer ADC Application Before the FDA Could Reject It
Rather than face a formal rejection, Daiichi Sankyo and Merck voluntarily withdrew their FDA application for ifinatamab deruxtecan in small cell lung cancer. The drug showed a 48% response rate, but tumor shrinkage lasted a median of just 5.3 months. The FDA's rising bar for accelerated approvals made a borderline data package untenable.
Read more →BioNTech's mRNA Cancer Vaccine Misses the Mark in Head and Neck Cancer
BioNTech's BNT113, an mRNA vaccine designed to supercharge immunotherapy in HPV-positive head and neck cancer, failed its Phase 3 progression-free survival endpoint. Adding the vaccine to pembrolizumab didn't beat pembrolizumab alone. Overall survival data could still rescue the trial, but analysts aren't holding their breath. BioNTech shares dropped 5-7%.
Read more →Verastem's KRAS G12D Inhibitor Posts a 50% Response Rate in Second-Line Pancreatic Cancer
Verastem's VS-7375 shrank tumors in half of second-line pancreatic cancer patients at the 900 mg dose, with a 41% response rate across later lines of treatment. The numbers are early and small (22 evaluable patients), but they compare favorably to competitors. A Phase 3 trial is planned by year-end.
Read more →Deals and M&A: Platform Fever Is Contagious
AstraZeneca Signs Its Second AI Deal in a Week, This Time for $247 Million
AstraZeneca is paying Absci up to $247 million to design a cancer antibody from scratch using generative AI. It's AZ's second major AI partnership in days (after a $1.5 billion insitro deal), signaling that AI-native drug discovery has moved from experiment to core sourcing strategy for top pharma companies.
Read more →BMS Drops $1.5 Billion on a Startup With Zero Clinical Data
Bristol Myers Squibb is acquiring Orbital Therapeutics, a preclinical-stage company building in vivo CAR-T therapies using circular RNA, for $1.5 billion in cash. Orbital has no approved drugs and no revenue. The bet: a programmable RNA platform that could make cell therapy work without a manufacturing plant.
Read more →Roche Bets Up to $1 Billion on Screening 300,000 Bispecific Antibody Combos
Roche signed a deal worth up to $1 billion with Dualitas Therapeutics, a startup barely a year out of stealth, to screen over 300,000 bispecific antibody combinations for immune diseases. The $36.5 million upfront payment buys access to Dualitas's DualScreen platform; the rest is milestones. It's a bet on discovery infrastructure, not a single molecule.
Read more →Approvals and Pipeline Moves
Novartis Quietly Kills Fabhalta's Biggest Expansion Play in Lupus
Novartis stopped its mid-stage Fabhalta trial in lupus nephritis, citing only "strategic considerations." The complement inhibitor, which generated $505 million in full-year 2025 revenue, is thriving in three approved indications with $3 billion-plus peak sales projected, but its push into the larger autoimmune kidney market just went blank. No data was released.
Read more →One Shot a Week Instead of 365: New Growth Therapy for Kids Nears European Approval
Europe's CHMP recommended approval of Ascendis Pharma's once-weekly TransCon CNP for children with achondroplasia, replacing the current standard of daily injections. In trials, treated kids grew 5.79 cm per year versus 4.02 cm for placebo, with safety comparable to placebo. A Germany launch is targeted for Q1 2027.
Read more →Teva's Monthly Schizophrenia Shot Drops the Three-Hour Monitoring Headache
The FDA approved Teva's Weltruza, a once-monthly olanzapine injection for schizophrenia that eliminates the three-hour post-injection observation window required by the older injectable version. Zero cases of post-injection sedation syndrome were reported across trials. For a disease where treatment adherence is half the battle, removing that clinic burden is a big deal.
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