Drug Discovery

Arms’ Length: Bayer Sees Progress in Hands-Off R&D Approach

This month, Bayer tripled its peak sales forecast for the oral androgen receptor inhibitor, from €1 billion ($1.1 billion) to €3 billion ($3.4 billion), following positive clinical data presented February 17 at the 2022 American Society of Clinical Oncology (ASCO) GU Cancers Symposium. The data showed that Nubeqa plus androgen deprivation therapy (ADT) and docetaxel reduced the risk of death by more than 30% in patients with mHSPC compared to ADT plus docetaxel.

Efficacy of Polyfunctional GD3 CAR T Cells against Rare Tumors

In this Isoplexis sponsored GEN webinar, our distinguished presenter, Dr. I. Caroline Le Poole will discuss identifying benign TSC tumors expressing immunogens that can be targeted by CAR T therapy.

Advanced Therapies Lead to Supply Chain Complexity

Early advanced therapies that focused on orphan diseases are now being replaced by a generation of drugs designed to treat indications with larger patient populations, possibly in the tens or hundreds of patients at a time. Advanced therapy supply chains have always been more complex than earlier therapies. The supply chain is circular–beginning with a patient-specific starting material and passing through manufacturing before returning to the patient.

Successful Passage: Gene Therapy Shows Early Effectiveness in GM1

Philadelphia-based Passage Bio develops gene therapies for rare, monogenic CNS disorders through a strategic collaboration and licensing agreement with the University of Pennsylvania’s Gene Therapy Program (GTP) to conduct discovery and IND-enabling preclinical work. A year after it was launched in 2019, the company went public through an initial public offering that raised $227.5 million. Passage Bio’s co-founders include GTP Director and gene therapy pioneer James M. Wilson, MD, PhD.

Combination Therapy Found to Prolong Survival of Hormone-Sensitive Prostate Cancer

An international, randomized, double-blind, placebo-controlled, Phase III clinical trial led by scientists at Massachusetts General Hospital (MGH) demonstrated that adding the androgen-receptor inhibitor darolutamide to androgen-deprivation therapy and chemotherapy extended the survival of men with metastatic, hormone-sensitive prostate cancer.

Patient Biopsy Screening Platform Identifies Potential Drug Candidates against Brain Metastases

Researchers at the CNIO designed a screening platform, METPlatform, which uses patients’ own tumor tissue for testing drugs against brain metastases. Through an initial screen the researchers identified potential drug candidates from a collection of FDA-approved and clinical trials-stage drugs, as well as confirmed the validity of the therapeutic targets in brain metastases. The studies also identified biomarkers of poor prognosis that signify more aggressive forms of brain metastasis.

Efflux Pump’s Shape Shifting Sheds Light on Antibiotic Resistance

Chemists have discovered how the structure of the EmrE transporter changes as a compound moves through it. As the pH drops, the helices tilt so that the channel is more open toward the outside of the cell, guiding the compound out. Knowing the structure of the drug-binding pocket of this protein could allow for the development of blockers that would prevent the removal of antibiotics from the cell.

Get Me (DNA) Rewrite: Intellia’s First Acquisition Expands CRISPR Toolkit

Intellia concluded that Rewrite’s technology can provide safe and curative genome editing treatments like the ones it is working to develop. In announcing the acquisition, Intellia also said Rewrite offered it “the potential to target diseases beyond those currently being explored in our pipeline.” Intellia isn’t yet saying which diseases those might be: “The technology is still in early-research stage, and we’ll share more as appropriate,” president and CE) John Leonard said.

What COVID-19 Has Revealed about Science & Society

On this GEN Live, Dr. Hotez will join us to talk about his admirable work fighting science misinformation, the anti-vaccine movement, his own team’s progress in vaccine development, and the major lessons from his recent book, “Preventing the Next Pandemic”. Please join us!

BioNTech Moves Forward to Promote Scalable Vaccine Production in Africa

BioNTech is developing a turnkey solution to enable mRNA-based vaccine production in modular and scalable sites. The company plans to establish mRNA manufacturing facilities in Africa in partner countries Senegal, Rwanda, and potentially South Africa. Construction of the first facility is expected to start in mid-2022 in a partner country, with the others to follow. A new project in Ghana will support manufacturing with fill-and-finish capacities.