Scientific Insights that Accelerate Drug Development
For the past couple of decades, leading American institutions, pharmaceutical and biotechnology companies, as well as the US FDA have been using quantitative tools (model-informed drug development; MIDD) to further their understanding of drug development assets. This integrated armamentarium has fueled industry consortiums, public-private partnerships, as well as several regulatory guidance documents. In this blog, we’ll discuss the FDA MIDD paired pilot program, and how it’s helping sponsors increase the efficiency of drug development.
Friedreich’s Ataxia (FA) is a rare, genetic, and progressive neurodegenerative disorder. A defect in the Frataxin (FXN) gene causes FA. FXN is essential for the proper functioning of the mitochondria, and its deficiency leads to mitochondrial dysfunction, oxidative stress, and neuroinflammation.
Your biotechnology start-up’s candidate asset has an intriguing proof of biology. Your investors are equally excited about it, and everything looks rosy regarding your future investments. You have a draft target product profile (TPP) that is all developed and squared away.
New biologic development and approval of novel biological medications have significantly moved the needle for addressing unmet diseases. Scores of biologics scientists and developers have supported this modern-day marvel.
A new class of complex biologics has emerged in recent years.
Recently, artificial intelligence (AI) has been front and center of newspapers, trade journals, and social media.
This analysis evaluated the PK, exposure–response, and exposure–PDrelationships of tremelimumab in patients with uHCC and supported the clinical utility of the STRIDE regimen.
This blog highlights 3 key areas for integrating pharmacometrics and clinical pharmacology to add value to a drug development program.
This blog discusses 3 critical formulation questions your drug development program should consider.
This blog discusses the clinical pharmacology strategy, dose selection, and safety considerations for cell therapy development programs.
In this blog, we will review and reflect on the scientific and strategic parts of the FDA guidance. We will also reflect on this guidance from a European Union (EU) perspective.
The ICH E11A draft Guideline on Paediatric Extrapolation was released on 4 April 2022 entering a public consultation phase. This guideline has several objectives. While aiming to harmonize methodologies and approaches for pediatric extrapolation in drug development, it balances access to pediatric indications and reduction of children’s exposure to experimental medications where warranted. The ICH E11A webpage outlines these considerations.
The new Clinical Pharmacology Considerations for Antibody-Drug Conjugates (ADC), Guidance for Industry, was issued by the US Food and Drug Administration (FDA) in February 2022. ADCs are targeted therapies that are designed to deliver cytotoxic payloads to cancer cells.
Bioequivalence is a commonly misunderstood term within biopharmaceutics. For starters, bioequivalence is a technical term baked in regulation.
Bioequivalence enables bridging safety and effectiveness of oral pharmaceutical dosage forms using pharmacokinetics.
Immunotherapy presents interesting and proximally viable therapeutic options in the growing…
Pandemics by nature are excellent fodders for disruptive innovation. They force you to think out of the box. The whole health care apparatus is on the COVID-19 frontlines with some more directly involved than others.
It is gratifying to see the continued uptake and adoption of tools for model-informed drug discovery and development since the early 2000s.
美国国家糖尿病、消化和肾脏疾病研究所认为，非酒精性脂肪肝（NAFLD）是一种肝脏储存过多脂肪的疾病，而非酒精性脂肪性肝炎（NASH）则是非酒精性脂肪肝的一种类型。
抗体-药物偶联物（Antibody-Drug Conjugate，ADC）是一种清除肿瘤细胞的独特方法，也是肿瘤学领域一种未得到充分利用的免疫疗法，无论是作为单一疗法还是联合疗法。
The era of gene therapy may have started a couple of decades ago, but approvals of agents based on the platform have been relatively recent. In 2017, Spark Therapeutics, Inc. received FDA approval of voretigene neparvovec-rzyl (Luxturna™), a recombinant adeno-associated virus serotype 2 (AAV2) vector expressing the gene for human retinal pigment epithelium 65 kDa 5 protein (hRPE65), for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy.
Alzheimer’s disease (AD) is one of the most common forms of neurodegenerative dementia in the United States. In fact, the Alzheimer’s Association predicts that by the year 2050, the number of people age 65 and older with Alzheimer’s dementia is expected to double to comprise 12.4 million patients.
by Rajesh Krishna and Abhijeeth Chandrasekaran
Written by Rajesh Krishna and James Bolognese
Strategic consulting within drug discovery and development is an exciting career choice for many. Unlike a biotechnology or pharmaceutical enterprise, where the assets one typically works on are within that ecosystem, consulting offers the possibility of working on many different innovation pipelines.
Global trials of therapeutic biologics have been historically absent from clinical
drug development.
传统的药物作用机制依赖于配体与其生物靶点之间可逆的、非共价的相互作用。On the other hand, a targeted covalent inhibitor (TCI) drug is designed to form a covalent bond between an electrophile on the ligand and a nucleophilic center in the target protein (Figure 1).
New therapeutics discovery and development for ocular diseases have been traditionally associated with a low probability of technical and regulatory success.
The safety of human participants in clinical trials, the intended patient population, and for the labeled indications at the point of introduction into the market is undoubtedly the paramount consideration of any drug hunting enterprise.
