Development Gene Sequencing Genetics 
pharmaphorum
NOVEMBER 3, 2020
Ring Chromosome 20 Syndrome, or (R)20, is an ultra-rare form of epilepsy with a devastating impact – yet despite huge leaps forward in gene sequencing in recent years, diagnoses are going down instead of up. Unlike with many similar conditions, a child will develop normally until the seizures start to kick in,” said Allison.
pharmaphorum
MARCH 10, 2022
18 months after signing its first partnering deal with Genentech, Scenic Biotech has raised $31 million in first-round financing that it will use to take its own genetic modifier-targeted medicines into clinical trials. Scenic has developed a series of small-molecule drugs targeting QPTCL that block the expression of CD47 on cancer cells.
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BioPharma Reporter
JULY 11, 2023
A new genetic sequencing technology from Element Biosciences has helped researchers from the Translational Genomics Research Institute (TGen), part of the City of Hope, identify the likely genetic causes of disorders in six of nine children from Sonora, Mexico.
pharmaphorum
JUNE 23, 2022
billion in potential milestones if the project advances through development and onto the market. Systemic administration of gene-editing drugs would avoid that process with an “off-the-shelf” option for a one-shot, potentially curative treatment for diseases associated with genetic mutations.
XTalks
DECEMBER 14, 2022
Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The MIT research team turned to a family of enzymes called integrases to meet their goal.
pharmaphorum
FEBRUARY 23, 2022
Takeda has forged another alliance as it continues a push into gene therapy, agreeing a deal worth up to $2 billion with Code Biotherapeutics for opt-in rights to four candidates for rare diseases. The deal is mostly back-loaded, with “double-digit million” dollars in upfront payment, research funding and milestone payments.
pharmaphorum
APRIL 23, 2021
billion in milestone payments in the deal, which will focus on using small-molecule compounds to “fine tune” the effects of genetic medicines in the body. The approach relies on the use of drug responsive domains (DRDs) on the gene-editing medicine, which can be used to either increase or decrease protein expression.
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