Mines D`or Orbec Inc

Beti-cel (ZYNTEGLO™) treatment for beta-thalassemia shows durable transfusion independence and normal hemoglobin levels up to 10 years post-treatment, with a favorable safety profile. 81% of participants have over 5 years of follow-up, and 90.2% achieved transfusion independence. Beti-cel is a one-time gene therapy approved for adult and pediatric patients requiring regular red blood cell transfusions.

AstraZeneca and Daiichi Sankyo's cancer drug dato-dxd showed tumor control for 6 months in EGFR-mutated lung cancer patients; Agenus plans 60% spending cut to focus on late-stage colorectal cancer immunotherapy; Amgen invests $1 billion to expand NC manufacturing; USPTO withdraws proposed rule on preventing 'patent thickets'; Bluebird bio to undergo reverse stock split; FDA grants accelerated approval to Merus' zenocutuzumab for NRG1-altered lung and pancreatic cancers.

CRISPR Therapeutics' stock has declined 46% since its 52-week high, despite FDA approval of Casgevy for sickle cell disease and beta thalassemia. Analysts suggest patient uptake has been slower than expected, partly due to treatment alternatives and high cost. ARK Investment Management has increased its stake in CRISPR Therapeutics, believing in the long-term value of gene editing cures. Despite recent dips, ARK funds have sold off shares, reflecting broader investor sentiment. Casgevy's commercial progress is seen as positive, with 45 of 75 treatment centers activated and initial revenue generated.

Gene therapies for sickle cell disease (SCD) face slow uptake due to high costs; Bluebird bio's Lyfgenia at $3.1 million and Vertex/CRISPR's Casgevy at $2.2 million. Both companies have entered agreements with CMS for an outcomes-based model to improve access and lower costs. The Cell and Gene Therapy Access Model aims to tie payments to therapy outcomes for Medicaid patients, launching in January 2025.

The Cell and Gene Therapy Access Model, initiated by the Biden-Harris Administration, aims to make advanced therapies for sickle cell disease more accessible by reducing costs. Bluebird Bio and Vertex Pharmaceuticals have agreed to participate, with the model set to launch in early 2025. This initiative seeks to improve patient outcomes and reduce long-term healthcare spending.

MEDIPOST, Inc. appoints five senior executives to lead its U.S. expansion of CARTISTEM, a regenerative therapy for knee osteoarthritis, aiming for IND approval and Phase III clinical trial initiation.

US FDA seeks to understand insertional mutagenesis in gene therapies like bluebird bio's Skysona, highlighting complexity and limitations of integration site analysis.

FDA investigates Skysona gene therapy after 7 of 67 CALD patients developed blood cancers. Despite risks, 94% showed no neurological decline 6 years post-treatment. Bluebird Bio links malignancies to lentiviral vector, emphasizing ongoing safety evaluations.

Novocure's electrical field-emitting device in Phase 3 trial showed extended survival in pancreatic cancer patients when used with chemo; Merus licensed U.S. rights of its cancer drug zenocutuzumab to Partner Therapeutics; FDA to decide on Cytokinetics' heart drug aficamten approval by Sept. 26, 2025; FDA investigates blood cancer cases post-Bluebird bio's Skysona treatment; Fate Therapeutics' CEO Scott Wolchko to retire, with Bob Valamehr taking over in 2025.

FDA investigates bluebird bio's Skysona gene therapy after reports of secondary blood cancers in treated patients, linked to clonal vector insertions and gene mutations. The FDA advises considering alternative therapies and is evaluating further regulatory action.