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Published on October 3, 2023
Genomics England has published an initial list of over 200 rare conditions that will be screened as a part of its new Generation Study, a program aimed at understanding whether sequencing babies’ genomes through newborn screening can help discover and treat rare genetic conditions earlier. The study will analyze the DNA of over 100,000…
Published on May 31, 2023
At the beginning of April, Vertex Pharmaceuticals and CRISPR Therapeutics announced they had applied for FDA approval for Exa-cel for treatment of sickle cell disease and beta thalassemia. If approved, this will be the first ever therapeutic approval for a gene edited therapy. With a number of similar gene editing…
Published on December 16, 2022
Research led by the University of Verona and the University of Harvard shows the importance of taking human genetic diversity into account when designing gene editing therapies for conditions such as sickle cell disease. A number of different CRISPR genome editing techniques are currently being developed to treat conditions such…
Published on December 15, 2022
Jim Geraghty Jim Geraghty, a seasoned biotech executive and a veteran of the orphan drug movement since its inception, took some time to talk to Damian Doherty about his new book, Inside the Orphan Drug Revolution. Jim gives an historical perspective of the leading protagonists who…
Published on August 16, 2022
Jonathan ThonFounder, CEO, STRM.BIO A serial biotech entrepreneur, Jonathan Thon, is the founder and CEO of STRM.BIO, a pre-clinical, VC-backed biotechnology company that is leveraging extracellular vesicles (EVs) to deliver gene therapies. Prior to launching STRM.BIO, he founded and served as CEO / CSO of PlateletBio…
Published on June 15, 2022
This year marks the 10-year anniversary of when CRISPR-Cas9 gene editing was first revealed to the world. Much has happened since then, including wide rollout in many sectors, development of CRISPR-based therapeutics and the discovery that the technology can also be used to develop highly accurate and affordable diagnostics. The…
Published on June 15, 2022
Deepak Asudani Deepak Asudani and his two children Ananya and Aarnav have published the first of a series of educational books on DNA and genetics aimed at schoolchildren who are curious to learn about what makes them unique whilst highlighting the similarities we share. Damian Doherty…
Published on June 10, 2022
A U.S. FDA advisory committee unanimously approved bluebird bio’s elivaldogene autotemcel (eli-cel) gene therapy for early active cerebral adrenoleukodystrophy (CALD) in certain patients. The committee’s recommendation is based on the Biologics License Application (BLA) currently under priority review by the FDA with a PDUFA goal date set for September 16,…
Published on May 11, 2022
54gene and its collaborators have published their first report on the project to sequence the genomes of 100,000 Nigerians spanning 300 ethnic groups. The Non-Communicable Diseases Genetic Heritage Study (NCD-GHS) led the work, which describes the team’s strategic vision and appears in Nature Genetics this week. This data will be used…
Published on March 8, 2022
Bluebird acknowledged Monday in a regulatory filing that Gina Consylman, the company’s CFO and treasurer, had given notice Friday of her resignation, effective April 3. Bluebird shares finished trading at $5 a share, down from $13.14 on November 15, when the post-spinoff Bluebird Bio started trading its shares. Shares of…
Published on February 16, 2022
Janice Chen is very inspiring. At the age of 30, she has not only completed a PhD. with recent Nobel prize winner Jennifer Doudna at the University of California, Berkeley, but co-founded Mammoth Biosciences together, where she serves as its CTO. Janice ChenCTO, Mammoth Biosciences Mammoth…
Published on December 10, 2021
Janice Chen is very inspiring. At the age of 30, she has not only completed a PhD with Nobel prize winner Jennifer Doudna at the University of California, Berkeley, but she has also helped co-found her own successful biotech company. Jumping straight from finishing her PhD in 2018 to co-founding…
Published on December 1, 2021
Cell-free (cf)DNA based non-invasive prenatal testing (NIPT) hit a milestone last August when the American College of Obstetricians and Gynecologists (ACOG) and the Society for Maternal-Fetal Medicine recommended it should be offered to all pregnant women regardless of their age or risk status. Since being introduced commercially 10 years ago,…
Published on November 9, 2021
A developer of gene-edited precision therapies co-founded by CRISPR pioneer Feng Zhang, Ph.D., has more than doubled its total capitalization after completing a $215 million Series B financing today. Arbor Biotechnologies said the financing brings its total capital raised to more than $300 million, and will enable it in part…
Published on July 22, 2021
Startup Orchid is launching the first preconception test that the company contends predicts a couple’s chances of conceiving a child with high risk of common illnesses, such as heart disease, stroke, schizophrenia, and several types of cancer. The test applies whole-genome sequencing (WGS) to a saliva sample from each partner,…