Mines D`or Orbec Inc

Real-world data from University Hospital Heidelberg show beti-cel's efficacy in eliminating transfusion dependence in transfusion-dependent β-thalassemia patients, with all 8 patients achieving transfusion independence. Safety concerns include pituitary-gonadal endocrine dysfunction and other adverse events, similar to clinical trial findings. The study highlights the need for less toxic conditioning regimens.

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The Gene Switch Market is projected to grow from USD 0.78 Bn in 2024 to USD 1.78 Bn by 2031, with a CAGR of 11.6%. CoherentMI's report provides a comprehensive analysis of market trends, competitive landscape, and key players like Novartis, Pfizer, and Gilead Sciences.

The global cancer gene therapy market is projected to grow from $2.3B in 2023 to $8.8B by 2030, driven by advances in gene editing, personalized medicine, and rising cancer incidence. Key technologies like CRISPR and next-generation sequencing are shaping the market, while challenges include high costs and regulatory hurdles.

Halozyme offers to buy Evotec for €2B; Bluebird records first revenue from Lyfgenia; BeiGene rebrands as BeOne Medicines; Adaptimmune to lay off 33% of staff; Leerink Partners adds senior M&A directors; Abeona awaits FDA decision on pz-cel.

10% of Skysona-treated CALD patients developed hematological cancers, linked to lentiviral vector insertions in proto-oncogenes. Six cases in ALD-104 involved MECOM/PRDM16, possibly exacerbated by myeloablative conditioning. Despite one death, CALD treatment success is noted, though cancer risks remain.

Acadia Pharmaceuticals sold its rare pediatric disease priority review voucher (PRV) for $150 million, with Neuren Pharmaceuticals set to receive $50 million. PRVs, awarded by the FDA for developing drugs for rare pediatric diseases, can be sold to accelerate drug reviews, highlighting their value in licensing deals.

A recent analysis of 50 US drug launches found only 12% accurately met sales forecasts, leading to wasted resources and limited patient access. High-profile failures like Novartis' Beovu and Bluebird Bio's Zynteglo highlight the importance of pre-launch management. The article outlines five key determinants for a successful launch.

Two new gene editing medications for sickle cell disease, including the first FDA-approved CRISPR-based drug, face barriers like high costs and complex delivery processes. Pfizer withdrew its sickle cell drug Oxbryta due to safety concerns, and patient uptake for new gene therapies has been slow. Despite these challenges, the sickle cell treatment market is expected to grow, with over 40 companies developing more than 50 treatments. Cellarity is developing an oral drug to induce fetal hemoglobin, aiming to provide a more accessible treatment option.