Drug Development
As therapies for rare and neurological diseases earn accelerated approval, experts laud the program’s intent while remaining concerned about confirmatory trials and clinical efficacy, especially as products greenlit under this pathway are pulled from the market.
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Accurately diagnosing Alzheimer’s disease pathologies is becoming increasingly important, but the U.S. is facing imaging resource constraints.
The plethora of genes involved in obesity presents an intriguing opportunity for both gene silencing and ex vivo gene therapy approaches.
Given their seven-figure price tags, it’s not clear how accessible the would-be cures will be to U.S. patients on public or private insurance.
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With the late-stage win, Bayer announced Monday that it plans to talk to regulators about seeking approval in an indication that is central to its $3 billion-plus peak sales forecast.
As part of a pipeline realignment, Bristol Myers Squibb is returning the rights to Agenus for its proprietary TIGIT bispecific antibody program and terminating their 2021 license, development and commercialization agreement.
Longeveron and Lexeo Therapeutics are working on CGT therapies to treat Alzheimer’s disease, but it’s not clear whether they have a better chance of success than traditional approaches.
Active immune therapies hold promise for preventing or slowing disease onset, but some experts warn of potential safety risks.
Ultracompact CRISPR systems, which are in some cases one-third the size of Cas9, are being designed to be more specific and enable in vivo gene editing in difficult to reach tissues.
With an upfront $800 million payment and $325 million in potential milestones, Otsuka Pharmaceutical is acquiring Jnana Therapeutics’ drug discovery platform and rare disease candidate, the companies announced Thursday.
Eli Lilly on Thursday released late-stage data showing a 38% reduction in the risk of heart failure outcomes, as it plays catch-up with Novo Nordisk’s semaglutide which won the FDA’s cardio nod in March.
In this episode, Lori and guests discuss the pursuit of adequate representation and how clinical trials are being shaped by the legal environment they operate within.
Eisai presented a plethora of data on the drug at the Alzheimer’s Association International Conference, including a study showing the consequences of pausing treatment.
The FDA will have six months to review Vertex Pharmaceuticals’ suzetrigine, potentially the first new class of drugs for acute pain in more than 20 years, according to the company.