Remove Gene Remove Gene Sequencing Remove Genetics
article thumbnail

Ginkgo BioWorks gets grant for AAV vector for treating NPC1 with specific gene sequence

Pharmaceutical Technology

Discover the groundbreaking gene therapy patent by Ginkgo BioWorks Inc for treating NPC1 using AAV vectors. Learn how this innovative approach offers hope for rare genetic disorders like Neimann-Pick Disease Type C.

article thumbnail

Gene sequencing identifies rare pediatric disorders in Mexico

BioPharma Reporter

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.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

Ring 20: Could the rare disease get left behind by next-generation gene sequencing?

pharmaphorum

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. The post Ring 20: Could the rare disease get left behind by next-generation gene sequencing? appeared first on.

article thumbnail

Scenic Bio raises $31m for genetic modifier platform

pharmaphorum

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. The post Scenic Bio raises $31m for genetic modifier platform appeared first on.

article thumbnail

New CRISPR-Based Tool Called PASTE Gene Editing Inserts Large DNA Sequences at Desired Sites

XTalks

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 PASTE gene editing technique was recently published in Nature Biotechnology.

article thumbnail

A Reversal on Sequencing? Proposed Legislation Would Allow Patenting of Naturally Occurring Genes

FDA Law Blog

Rather, developers of diagnostic tests and, indeed of any product that relies on free access to gene sequence and other biomarker information, should pay also close attention, as PERA would overturn longstanding judicial precedent. By 2010, about 2000 isolated human genes had been patented in the U.S.

Gene 62
article thumbnail

Gene therapy specialist bluebird exits “untenable” European market

pharmaphorum

Europe market has proved so hostile to gene therapies when it comes to pricing and reimbursement for gene therapies that bluebird bio has decided to quit the market altogether, according to Andrew Obenshain, president of its severe genetic diseases unit. market and will wind down in Europe.