Development

Cellectis is pivoting to in vivo gene editing to advancing its .HEAL-101 and .HEAL-201 candidates for lipid disorders while exiting its CAR T programs.

With 15 patients now dosed in its pivotal EMERALD trial, Resolution will build on the 70% 4-year transplant-free survival seen with RTX001 in earlier data.

Lundbeck's asedebart, which blocks ACTH signaling at its source, has won FDA orphan status for endogenous Cushingsyndrome.

Biora's BioJet capsule aims to deliver adalimumab without a needle, entering human testing after hitting 51.3% preclinical bioavailability.

Satralizumab cut MOGAD relapse risk 68% in a phase 3 trial, positioning it as the first potential treatment for this rare disease.

Ersodetug missed its phase 3 primary endpoint in congenital HI, but FDA is reviewing CGM data showing 50%+ hypoglycemia reductions.

Del-desiran missed its primary endpoint in a phase 3 DM1 trial, though secondary measures showed signs of clinical activity.

HHS announced four senior FDA leadership selections, including Karim Mikhail as permanent Director of the Center for Biologics Evaluation and Research and Michael Davis as Director of the Center for Drug Evaluation and Research, capping more than a year of turnover at both centers.

Aptar Pharma's Nanopharm business will lead inhaled formulation development and device assessment for ACT-101, Aceso Therapeutics' antisense oligonucleotide candidate designed to correct CFTR protein function in cystic fibrosis patients carrying the F508del mutation.

BrainChild Bio's BCB-276 extended survival to 19.8 months from diagnosis in phase 1, backing its $116 million-funded pivotal DIPG trial.

Moonwalk's $70 million new funding advances MW101, an adipose-targeted siRNA aiming to cut fat while preserving muscle in obesity patients.

Bristol Myers Squibb reported positive topline phase 2 results for arlocabtagene autoleucel, a potential first-in-class GPRC5D-directed CAR T cell therapy, in patients with quadruple-class exposed relapsed and refractory multiple myeloma who had already received a prior BCMA-targeted therapy.

9MW1911 cut severe COPD exacerbations by up to 100% at the highest dose, supporting Mabwell's push toward phase 3 development.

Acepodia's ACE723 delivers 2 cytotoxic payloads to GPC3-positive tumors, clearing FDA review to begin trials in liver cancer.

GMP cleanroom differential pressure control prevents FDA 483 citations and contamination failures through HVAC design and validation.

GSK's up to $1.3 billion deal centers on HUTCHMED's HMPL-A830, which pairs a KRAS inhibitor with an EGFR antibody.

Apazunersen missed its primary endpoint in a phase 3 Angelman syndrome trial, meaning that patient population remains without an approved treatment option.

ArsenalBio is halting all development of its ex vivo CAR T programs for solid tumors and laying off the majority of its workforce as it pivots the company entirely toward in vivo CAR T therapy, joining a fast-growing field that already includes Johnson & Johnson, Eli Lilly, and several other developers.

NewBiologix's Xcell platform aims to replace costly transfection with stable cell lines for Synastra's high-dose DMD gene therapy.

Teva's TEV '408 blocked gluten-induced gut damage in a phase 2a trial, supporting its use for celiac disease, which has no approved drug treatment.

SIM0660's dual CD79a/CD19 targeting aims to deepen B-cell depletion, supporting Roche's up-to-$1.53 billion bet on Simcere Zaiming's TCE platform.

AstraZeneca's Enhertu plus pertuzumab cut progression risk 44%, extending PFS past 3 years as the first new 1st-line HER2+ regimen in 10 years.

PharmaEssentia's ropeginterferon alfa-2b-njft (Besremi) cut ET response rates to 43% versus 6% for anagrelide, backing a first-in-decades FDA nod for a new rare blood cancer treatment.

LIlly reports that Taltz plus Zepbound helped 30.6% of psoriasis patients clear skin and lose 10%+ weight at 1 year, versus 4.4% on Taltz alone.

TSY-110 could become the first biosimilar rival to Roche's ado-trastuzumab emtansine (Kadcyla), a $2.5 billion HER2 breast cancer therapy.