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Maxcyte to publish positive data from Sickle Cell treatment reasearch

Doug Doerfler, MaxCyte’s chief executive, said: "We are excited by these results, and look forward to evaluating long-term data."

picture of blood being studied
Patients with sickle cell disease have abnormal haemoglobin,

MaxCyte Inc (LON:MXCT) has published positive pre-clinical study results from its gene editing technology in the treatment of sickle cell disease (SCD).

The data was presented Tuesday 25 April to the American Society of Gene and Cell Therapy Annual Meeting (ASGCT).

MaxCyte's platform (cGMP) was used by the National Heart, Lung and Blood Institute (NHLBI) and National Institute of Allergy and Infectious Diseases (NIAID) as part of a programme to develop potential curative therapy based on "correcting" the faulty gene in SCD.

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Using cGMP, there were corrections of the mutation behind SCD in more than 30% of patient-derived B cells.

Doug Doerfler, MaxCyte’s chief executive, said: "We are excited by these results, and look forward to evaluating long-term data."

The work follows on its attempts at achieving gene correction in X-linked chronic granulomatous disease.

This demonstrates 'the ability of our cGMP-compliant delivery platform to achieve robust, scalable, and a clinically relevant levels of gene correction in sickle cell disease patient-derived cells', he added.

Sickle Cell Disease

Patients with SCD have abnormal haemoglobin, called haemoglobin S or sickle haemoglobin, in their red blood cells. A life-long illness, its severity varies widely from person to person.

Currently, haematopoietic stem cell transplantation (HSCT) is the only cure for SCD, but this requires a well-matched donor. There are some treatments that can reduce symptoms and prolong life, MaxCyte added.

 

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AIM:MXCT
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