gene therapy

Ways to Pay

New gene therapies are coming with high price tags, the New York Times reports.

The New York Times reports that a number of companies and research institutes are pursuing gene therapies.

A US Food and Drug Administration panel recommends that the agency approve a gene therapy for pediatric leukemia, the New York Times reports.

To study the benefit of genetic testing data, the Foundation Fighting Blindness offered free clinical testing to 100 members of its My Retina Tracker patient registry.

NIH researchers got tantalizingly close to long-term engraftment at levels that could provide a clinical benefit in gene therapy applications.

The President's Council of Advisors on Science and Technology urges the adoption of a biodefense strategy, Tech Review reports.

A modified version of a commercially available magnetic bead cell sorting instrument from Miltenyi Biotec could be deployed to areas without cGMP facilities.

Some Cold Water

Even with all the current enthusiasm for gene therapies, Tech Review cautions that they are mostly in the early stages of development.

Scientists led by Eric Kmiec showed that their strategy of point mutation editing with single-stranded DNA oligos and double-strand breaks works with CRISPR/Cas9. 

Start It Themselves

Parents raise more than $1.8 million to start a clinical trial to help their daughter with Sanfilippo syndrome, Today reports.

Pages

A former Synthetic Genomics attorney alleges that the firm discriminated against her and other female employees, according to the San Diego Union-Tribune.

Due to privacy and lab certification questions, the planned giveaway of Orig3n testing kits at a Baltimore Ravens game was suspended.

Alnylam reports positive results from its phase 3 clinical trial of an RNAi-based drug, according to Stat News.

In Cell this week: adult mesenchymal cell populations in mouse lung, genetic diversity in HPV16 and cancer risk protection, and more.