Gene therapy approval “game changer” in hemophilia B treatment
The approval of CSL's gene therapy Hemgenix has ushered in a new era for hemophilia…
November 24, 2022 - 7 minutes
Rare disease
Khondrion’s mitochondrial diseases treatment producing positive results
Clinical-stage pharmaceutical company, Khondrion, discovering and developing therapies targeting primary mitochondrial diseases announced yesterday (November…
November 23, 2022 - 5 minutes
A new treatment for tackling epidermolysis bullosa
By Jordi Casals, president, EMEA, Amryt Pharma Epidermolysis bullosa (EB), sometimes described as “the worst…
November 14, 2022 - 3 minutes
InfanDx tackles early detection of hypoxic ischemic encephalopathy
InfanDx AG, a privately held diagnostics company focusing on the development and commercialization of novel…
November 14, 2022 - 8 minutes
A biotech boom manifesting real results in rare pediatric diseases
By Bob Coughlin, managing director, life sciences, JLL For the last two years, the pandemic…
November 14, 2022 - 5 minutes
Centogene launches rare and neurodegenerative disease Biodata Network
German company, Centogene N.V., today (November 4) announced its launch of Biodata Network, a portfolio…
November 4, 2022 - 3 minutes
What’s on the horizon for gene editing-based therapies?
Gene editing has come far in the past decade. However, the field is still finding…
November 3, 2022 - 8 minutes
Servier and OSE Immunotherapeutics complete enrollment in Sjögren’s syndrome trial
Servier and French biotech company OSE Immunotherapeutics SA have completed patient enrollment in a phase…
November 3, 2022 - 2 minutes
Sponsored
by CENTOGENE
Improving rare disease diagnosis and treatment with next generation sequencing technology
Rare diseases refer to over 7,000 different known health conditions that together affect up to…
October 27, 2022 - 6 minutes
Syncona to acquire Applied Genetic Technologies Corporation
Healthcare company Syncona Limited says its newly established portfolio company has agreed to acquire, by…
October 24, 2022 - 2 minutes
Opportunities and challenges for cell and gene therapy investments
Companies in the cell and gene therapy space are struggling to raise investments amid adverse…
October 20, 2022 - 9 minutes
CMT Research Foundation funding boosts Samsara Therapeutics Charcot-Marie-Tooth treatment
The CMT Research Foundation (CMTRF), a non-profit focused solely on delivering treatments and cures for…
October 13, 2022 - 4 minutes
