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☆ Experimental gene therapy restores some vision in patients with inherited blindness
▼ + stars: | 2024-05-06 | by ( Jacqueline Howard | ) edition.cnn.com time to read: +16 min

“Now, post-surgery and post recovery, I am able to see in dimmer lighting with my left eye,” Cook said. A treatment that used CRISPR was found to be safe and efficacious in improving vision among a small sample of patients with inherited blindness in the Phase 1/2 clinical trial that Cook participated in. Months following the treatment, Cook was sitting with friends on a balcony that had Christmas lights wrapped around the railing. Courtesy Olivia CookBefore the treatment, Cook said that she sometimes could conceal the vision challenges she has had. Mass Eye and EarKalberer described the CRISPR treatment as “groundbreaking,” but warned it’s not a cure.

Persons: Olivia Cook, Cook, ”, ” Cook, “, I’ve, “ I’d, you’d, CRISPR, Eric Pierce, Brigham, “ We’re, ” Pierce, Jason Comander, ‘, Michael Kalberer, Kalberer, it’s, “ It’s, It’s, Pierce, Editas, We’re, Art Caplan, ” Caplan, —, Vlad Diaconita, ” Diaconita, Dr, Sanjay Gupta, Aliaa Abdelhakim Organizations: CNN, Missouri State University, New England, of Medicine, Mass, Harvard Medical School, Editas Medicine, Perelman School of Medicine, University of Pennsylvania, University of Michigan, University of Miami, Oregon Health & Science University, US Food and Drug Administration, CEP290, pharma, NYU Grossman School, Medicine’s Department of Population Health, Columbia University Vagelos College of Physicians and Surgeons, CNN Health Locations: Springfield, United States, CEP290

Scientists are setting out to collect genetic material from 500,000 people of African ancestry to create what they believe will be the world’s largest database of genomic information from the population. The initiative was launched Wednesday by Meharry Medical College in Nashville, Tennessee, as well as Regeneron Genetics Center, AstraZeneca, Novo Nordisk and Roche. Organizers said there’s a clear need for the project, pointing to research showing that less than 2% of genetic information being studied today comes from people of African ancestry. Each of the pharmaceutical companies involved intends to contribute $20 million toward the genetic and educational parts of the effort. ___The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Science and Educational Media Group.

Persons: Meharry, ”, Anil Shanker, Lyndon Mitnaul Organizations: Meharry Medical College, Genetics Center, AstraZeneca, Novo Nordisk, Roche, Genomics Institute, Organizers, Regeneron Genetics, University of Zambia, Project, Genetics, Corporate, Associated Press Health, Science Department, Howard Hughes Medical Institute’s Science, Educational Media Group, AP Locations: Nashville , Tennessee, Americas, Asia, Europe, Nashville, U.S, Africa, Meharry

☆ Scientists updated the human genome to make it more inclusive
▼ + stars: | 2023-05-10 | by ( Katie Hunt | ) edition.cnn.com time to read: +6 min

CNN —Scientists have pieced together a new draft of the human genome that better captures humanity’s genetic diversity. The scientists involved say it will improve our ability to diagnose disease, discover new drugs and understand the genetic variants that lead to ill health or a particular physical trait. The pangenome, a digital amalgamation of sequences that can be used to compare, construct and study other human genome sequences, is still a draft. The first draft of the human genome was released in 2001 and was only fully completed in 2022. The new pangenome reference is an amalgamation of different genomes from 47 people with ancestry from around the world.

☆ A drug company abandoned a treatment for ‘bubble boy disease.’ After a 5-year fight, this little girl is about to get it
▼ + stars: | 2023-04-27 | by ( Elizabeth Cohen Lauren Mascarenhas | Elizabeth Cohen | Lauren Mascarenhas | ) edition.cnn.com time to read: +29 min

The Sulacks weighed their options: Have a transplant with a match that was less than ideal – far less – or wait for gene therapy to become available. The news release didn’t say anything else about the SCID gene therapy. Or was the company abandoning its plans for SCID gene therapy altogether? In February, 2021, the parents of more than 20 children who were waiting for the gene therapy treatment, including the Sulacks, wrote a letter to Gaspar. Insurance companies have sometimes balked at paying for gene therapy, which is typically given in one treatment.

☆ Gene-editing companies are growing thanks to fast-paced science, even as biotech faces a downturn
▼ + stars: | 2022-10-12 | by ( Andrew Dunn | ) www.businessinsider.com time to read: +8 min

CRISPR companies are faring better by producing faster and more dramatic clinical results. A layoff tracker from Fierce Biotech counts at least 90 drug companies that have laid off staff in 2022. Gene-editing biotechs have avoided the brunt of the downturn because of fast clinical successIntellia Therapeutics is developing CRISPR-based gene-editing medicines that could cure diseases. Just in the past month, CRISPR Therapeutics moved into a swanky new headquarters in Boston that can house up to 1,000 people. BeamTo be sure, 2022 hasn't been smooth sailing for the CRISPR companies.

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