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Rare & Genetic Diseases
| 5 min read
A wave of FDA rejections reveals how CMC issues are becoming a leading barrier to approval in cell and gene therapy development.
| 4 min read
Another death linked to Sarepta’s gene therapy, a promising glioblastoma trial, a rejection for Replimune, and more led the news this week.
| 7 min read
Synthetic control arms are emerging as a valuable solution for clinical trials where using traditional placebo groups is difficult, unethical, or impractical.
| 2 min read
Qureight’s deep-learning image biomarkers and clinical data analytics platform used to analyse enrolled idiopathic pulmonary fibrosis (IPF) patient data, supporting efficacy outcome and expansion to larger global patient studies
| 4 min read
An injection of an antisense oligonucleotide in utero improved outcomes for mice with the neurogenetic disease.
| 2 min read
Enhanced Genomics’ 3D multi-omics platform harnessed to rapidly identify multiple novel therapeutic targets that were undetectable using conventional genomic approaches
| 3 min read
Boosting UBA5 gene production normalized neuronal activity in the first organoid model of the developmental and epileptic encephalopathy.
| 8 min read
Explore how single-dose gene therapy offers a potential long-term treatment for hemophilia, and discover the challenges contributing to its low adoption.
| 6 min read
Via a unique regulatory route in the UK, scientists found that a gene therapy restored vision in young kids with a very rare and severe retinal dystrophy.
| 6 min read
Rather than permanently cutting DNA, this new kind of gene therapy instead aims to regulate gene expression using epigenetic mechanisms.
| 4 min read
The therapy lengthened telomeres and improved immune function in people with telomere biology disorder, showing promise for treating bone marrow failure safely.
| 3 min read
A new compound, Vykat XR, addresses insatiable hunger in the rare genetic condition, offering relief for patients and hope for families.
