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New Treatment For Genetic CHAPLE Disease David Orchard-Webb
September 28, 2023
There are fewer than 100 individuals worldwide known to have the disease. In the U.S., fewer than 10 patients with CHAPLE disease have been identified.
Indo-Irish Biotech Startup CyGenica Secures Funding from SOSV to Accelerate Cancer and Rare Genetic Disease Therapy b3cnewswire
June 09, 2021
CyGenica has announced that it has raised USD $1.4 million in a seed fund investment round, led by global venture capital investor SOSV.
BridgeBio Pharma LLC Signs Joint Collaboration Agreement with Cincinnati Children's to Accelerate Development of Genetic Disease Therapeutics pharmafocusasia
November 13, 2018
"BridgeBio is privileged to partner with leading experts at Cincinnati Children's, an institution whose impact on science and patients we admire. We hope we can help advance the mission by partnering to move therapies forward to patients," said Neil Kumar
FDA approves RNA therapy for rare genetic disease pharmafile
August 14, 2018
The US Food and Drug Administration has approved the RNA-based therapy Onpattro, for the treatment of the rare genetic disease polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients.
FDA approves Palynziq for PKU, a rare and serious genetic disease europeanpharmaceuticalreview
May 29, 2018
Novel enzyme therapy for adults with PKU who have uncontrolled blood phenylalanine concentrations with current treatment…
BridgeBio Pharma gets $135M for genetic disease work biospectrumasia
September 18, 2017
The company will use the cash to boost the work on its early-stage discovery and development programs, with the goal of creating genetically targeted therapeutics
What's next after CRISPR succeed in quest to transplant pig organs into humans? fiercebiotech
August 14, 2017
Transplanting animal organs into humans is an attractive approach, but the risk of immune rejection or catching a virus from the organ has been a significant roadblock. eGenesis used CRISPR to address the latter concern.