New Medicines

Severe sickle cell disease


New molecular entity
Graphite Bio
Graphite Bio

Development and Regulatory status

Phase II Clinical Trials
Aug 22Graphite Bio expects to report initial proof-of-concept data from the CEDAR trial in mid-2023 [4].
May 22Granted fast track status in US for SCD [2].
Dec 21FDA grants orphan drug designation to GPH 101 for the treatment of severe SCD [2].


A gene edited autologous CD34+ haematopoietic stem cells therapy that directly targets the ß globin gene to decrease sickle haemoglobin production and restore normal adult haemoglobin expression. Uses clustered regularly interspaced short palindromic repeats (CRISPR) and natural DNA repair technology to cut the single globin gene and paste in the correct code. The defect will eventually be reversed after the correction. Given as a single dose following a myeloablative conditioning regimen.
SCD is thought to be the most common severe genetic disease in the UK and France, with 10,000-15,000 people affected. The prevalence of SCD is highest in sub-Saharan Africa. The sickle beta globin gene is spread widely throughout Africa, the Middle East, the Mediterranean and India (eg, sickle genes are present in 1 in every 50 Asians and 1 in every 100 Northern Greeks). The gene has spread through population movement to the Caribbean, North America and Northern Europe [1].
Severe sickle cell disease
Intravenous infusion

Trial or other data

Aug 22First patient has been dosed with GPH101 (nulabeglogene autogedtemcel; nula-cel) in the PI/II CEDAR trial. Collection of primary outcome data is due to complete May 26 [3,4].
Sep 21First-in-human single-arm PI/II CEDAR trial to assess the safety, efficacy and pharmacodynamics of GPH 101 IV infusion following myeloablative conditioning regimen in an autologous HSCT setting, in patients with severe sickle cell disease starts (GPH101-001; NCT04819841). The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data. The open-label, single-arm trial is enrolling 15 patients aged 12 to 40 years in the US [3].