Manufacturing

Encoded Therapeutics closed a $275 million Series F financing to fund pivotal and expansion studies of ETX101, an AAV9-mediated gene regulation therapy for SCN1A-positive Dravet syndrome, alongside commercial-scale buildout of the company's in-house GMP manufacturing.

Amgen and AstraZeneca announced that the Phase 3 DeLLphi-305 trial met its primary endpoint, with Imdelltra (tarlatamab) plus Imfinzi (durvalumab) significantly improving overall survival versus durvalumab alone as first-line maintenance therapy for extensive-stage small cell lung cancer — the first phase 3 study of a bispecific T-cell engager to show a survival benefit in this earlier-line setting.

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

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.

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.

Cipla's US subsidiary secured exclusive rights to commercialize QL2107, Qilu Pharmaceutical's phase 3 biosimilar candidate to Keytruda, ahead of the reference biologic's anticipated 2028 patent expiration.

Medicus Pharma secured worldwide rights to PF-08046031 (CD228V), an antibody-drug conjugate targeting melanotransferrin that Pfizer discontinued earlier this year following its $43 billion Seagen acquisition, in a co-development and license agreement that could pay Pfizer more than $1 billion in milestones.

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

TScan Therapeutics is reorganizing to prioritize in vivo-engineered TCR-T therapy for solid tumors, pausing its phase 3 trial and autoimmune program and cutting about 75% of its workforce, even as new phase 1 data showed complete donor chimerism in all tracked heme malignancy patients.

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

uniQure submitted a Biologics License Application to the FDA and a Marketing Authorisation Application to the UK's MHRA for ifezuntirgene inilparvovec (AMT-130), an AAV5 gene therapy for Huntington's disease, based on three-year Phase I/II data — a potential first disease-modifying treatment for a condition with no approved therapies that slow progression.

Ultragenyx published 96-week phase 3 data for GENGLYCOS, its AAV8 gene therapy for glycogen storage disease type Ia, in The Journal of Inherited Metabolic Disease, showing sustained cornstarch-intake reductions and continued glycemic control roughly six weeks after the therapy's FDA accelerated approval.

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.

GSK will begin a phase 3 trial in September 2026 for its investigational mRNA seasonal flu vaccine after phase II data showed stronger immune responses than licensed comparators. The candidate is designed to target both hemagglutinin and neuraminidase, a dual-antigen approach not yet tested in a late-stage mRNA flu trial.

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.

Celularity has entered a US manufacturing collaboration with MuseCell Innovations to produce Dezawa MuseCells and related exosome and secretome products at Celularity's Florham Park, New Jersey facility, a relationship that could generate more than $300 million in aggregate purchases over five years.

HaemaLogiX has dosed the first patient in its Phase 1 KOALA trial of KMCAR T-cell therapy, a novel CAR-T designed to selectively target kappa-restricted multiple myeloma cells while sparing healthy immune cells, with the treatment reported as well tolerated and a second patient enrolling at a higher dose.

4basebio and Genezen have entered an expanded, non-exclusive collaboration giving drug developers access to 4basebio's hpDNA cell-free synthetic DNA technology within Genezen's viral vector development and manufacturing services, aiming to streamline gene and cell therapy programs from early development through commercial manufacturing.

AbbVie's bispecific antibody posted a 90% response rate in squamous NSCLC, headlining the company's expanding lung cancer pipeline data.

The FDA has approved Genglycos (pariglasgene brecaparvovec-opnr), a one-time AAV8 gene therapy, to reduce cornstarch dependence in patients eight and older with GSDIa, the first approved treatment to target the disease's underlying genetic cause.

AbelZeta Pharma has reclaimed global rights to prizloncabtagene autoleucel from Janssen and secured FDA clearance to begin US trials, positioning the anti-CD20/CD19 bispecific CAR-T for patients who relapse after existing CAR-T therapies.