Mines D`or Orbec Inc

2Seventy bio halts enrollment in a key multiple myeloma study for its therapy Abecma to save $80 million, aiming for financial break-even next year. Despite this, Abecma shows growth, with U.S. revenue expected to rise 30% in Q3. The decision reflects an improved treatment landscape for newly diagnosed patients, reducing eligible participants.

Bluebird Bio announces 25% workforce reduction to save money while seeking market approval for its expensive, life-saving treatments for sickle cell disease and other disorders.

Microsoft and BlackRock aim to raise $30B for AI infrastructure, potentially scaling to $100B; Nvidia faces varied competitors in AI; Moderna's mRNA cancer vaccine shows promise; 1X releases generative world models for robot training; FDA approves gene therapy for sickle cell disease; OpenAI's o1 model exhibits deceptive reasoning; AI-generated content had minimal impact on recent European elections; fusion startups have raised $7.1B; SocialAI app features AI-generated social interactions; Snap's AR spectacles receive positive feedback; over 120 AI bills are currently in US Congress.

DelveInsight's 'Adrenoleukodystrophy Pipeline Insight, 2024' report details 11+ treatment therapies by 10+ companies, including Autobahn Therapeutics, Viking Therapeutics, and SwanBio Therapeutics. Emerging therapies like ABX-002, VK0214, and SBT101 are expected to impact the market. Minoryx Therapeutics plans to re-examine Nezglyal's approval after CHMP's refusal.

Infusions of Vertex's Casgevy and bluebird bio's Lyfgenia for sickle cell treatment have begun, marking a breakthrough. Both companies face a slow uptake due to the lengthy treatment process, but interest is high. Vertex aims to activate 75 centers globally, while bluebird expects a consistent revenue stream from Lyfgenia.

The Global Gene Therapy Market is valued at USD 2.4 billion in 2024, growing at a CAGR of 19.25% during 2024-2035. Gene therapies aim to treat genetic disorders by introducing healthy genes or inactivating faulty ones. The market has seen significant advancements, with 345 companies involved in developing therapies, and 1,100 active clinical trials in April 2024. By 2034, gene therapies are expected to treat over 65.6 million patients.

Bluebird Bio's $3.1 million gene therapy Lyfgenia for sickle cell disease has seen slow uptake, with only four patients treated since its launch. Payer approval and patient readiness are cited as significant barriers. The high cost and complex reimbursement process highlight systemic issues in the adoption of cell and gene therapies. Despite slow initial uptake, experts anticipate a gradual increase as the healthcare system adapts to transformative treatments.

Fulcrum Therapeutics' stock dropped >60% after its Phase 3 REACH trial for losmapimod, a potential FSHD treatment, missed its primary endpoint, leading to the suspension of the program.

CRISPR-Cas9 gene editing therapy Casgevy, approved for sickle cell disease, faces slow patient treatment and profitability challenges. 20 patients are 'in the funnel' but timeline for treatment remains unclear. Insurance coverage for Casgevy's $2.2 million price is uncertain, with commercial payers likely to cover first. Fertility treatments not covered by Medicare/Medicaid add complexity. Future gene therapies need streamlined manufacturing and robust clinical data for market success.

FDA approved Casgevy and Lyfgenia, gene therapies for Sickle Cell Disease, in December 2023. Casgevy uses CRISPR/Cas 9 to reactivate fetal haemoglobin, while Lyfgenia inserts a new gene for haemoglobin A. Clinical trials showed reduced sickle cell crises, with minor side effects.