Feliza Mirasol

FNIH CEO Dr Julie Gerberding will open the October 25-28 meeting with a talk on using AI, adaptive trials, and alternative methods in drug development.

Insilica's Dr Thomas Luechtefeld argues process traceability, not benchmark accuracy alone, determines whether AI toxicology decisions can be trusted.

Phase 1 HCXT-2001, an oral macrocyclic IL-23R peptide, could be paired with emvistegrast as a daily oral IBD treatment, according to Gilead.

Zylidac will test whether optogenetic cell lines that hit 21 g/L in small-scale CHO runs can deliver higher-yield complex biologics at GMP scale.

Revakinagene taroretcel (Encelto), the only FDA-approved MacTel therapy, slowed photoreceptor loss by up to 54.8% vs sham in phase 3 trials.

At PharmSci 360, Insilica CEO Thomas Luechtefeld will present on auditable agentic AI systems and how they are expanding computational toxicology beyond single predictive models.

Dr Julie Gerberding and Dr David Berry will headline the October 25-28 New Orleans meeting, which adds a USP standards workshop and AI, organoid tracks.

Backed by €400 million (US$449 million) in EU-cleared German aid, the new Frankfurt facility is designed to secure insulin supply for people with diabetes from 2032.

EA2251, the 7th registrational trial of ivonescimab, will test it plus chemotherapy vs pembrolizumab or durvalumab regimens in first-line BTC.

Inhaled EXO-CD24 kept 85% of patients with ARDS free of respiratory failure at day 7 vs 33.3% with placebo in a 90-patient phase 2b trial.

Nuravax will test AV-1959R in a 160-adult phase 2 trial to see whether the amyloid vaccine can prevent Alzheimer disease before symptoms begin.

BMS's Dr Nathan Pennell outlines how biomarker testing barriers and trial diversity affect patient access to targeted oncology drugs.

Guselkumab (Tremfya) sustained perianal fistula healing through 1 year in Crohn disease, with remission climbing to 35% in one FUZION dosing arm.

Cemiplimab's proposed EU regimen could cut Libtayo infusion visits from every 3 weeks to every 6 weeks, trimming infusion time by up to half.

EST-201 and EST-101 target the genetic root cause of ADPKD, a disease affecting more than 8 million people worldwide, with trials starting 2027-2028.

AVT29, a proposed aflibercept biosimilar to Eylea 8 mg, could extend retinal disease treatment intervals to 16 weeks, versus 8 weeks today.

Mirikizumab (Omvoh) achieved comprehensive disease control in 27.7% of UC patients and deep remission in 20.4% with Crohn disease at 1 year.

BNT113 plus pembrolizumab failed to significantly extend progression-free survival in a 285-patient HPV16-positive head and neck cancer trial.

Gilead will develop up to three PRO-XTEN T-cell engagers discovered by Vir Biotechnology for solid tumors in a deal worth up to $1.74 billion.

Johnson & Johnson's guselkumab achieved up to 29% fistula remission in hard-to-treat Crohn disease subgroups, including prior biologic failures.

Rona's ALK7 RNAi candidate, licensed to Roche for up to $1.06 billion in milestones, aims to cut visceral fat while sparing lean mass.

Clinical results reveal new opportunities and setbacks in rare disease and cancer care, shaping treatment strategies and future regulatory decisions.

Odyssey's 146-patient phase 2a trial will pair OD-001 with vedolizumab, targeting both innate and adaptive immune drivers of ulcerative colitis.

MT027, T-MAXIMUM's B7-H3 CAR-T therapy, earns a second FDA-cleared phase 2 trial, now testing lung and breast cancer brain metastases.

New clinical data highlight improved disease control, reduced eye bulging, and weight-loss benefits, potentially broadening options for patients with unmet needs.

Antisense oligonucleotides are reshaping drug design, from RNase H gapmers to inhaled delivery; this FAQ explains how they work and why trials still fail.

Bristol Myers Squibb's Nathan Pennell explains how the MRD endpoint supports accelerated approval in multiple myeloma without changing how clinicians monitor patients.

Teprotumumab N01 (Sycume) reduced eye bulging in 60.4% of patients with chronic, inactive thyroid eye disease in a phase 3 trial.

New clinical milestones, manufacturing investments, and regulatory incentives could help developers overcome barriers to delivering treatments for patients with rare diseases.

Genglycos, the gene therapy that earned the voucher, will fund new Ultragenyx therapies for other rare and ultra-rare diseases.