Novartis has decided to discontinue its ex vivo sickle cell disease (SCD) program, which was developed using Intellia Therapeutics’ CRISPR gene editing technology. This news comes as part of Intellia’s 2022 financial results, released on Thursday. The program aimed to provide a potential treatment for SCD, but unfortunately, the project has been halted.
Intellia is pioneering a groundbreaking in vivo editing approach to treat Sickle Cell Disease (SCD) that could potentially eliminate the need for bone marrow transplants. By developing this innovative approach, Intellia is paving the way for a new era of disease treatment.
Novartis is taking a proactive approach to tackle sickle cell disease (SCD) and beta-thalassemia. In 2022, the Swiss pharma announced a strategic partnership with Precision BioSciences to develop a curative gene therapy. This groundbreaking deal saw Novartis invest $75 million upfront in the project, with high hopes of providing a long-term solution to these illnesses.
Precision is creating a tailor-made ARCUS nuclease that Novartis will have the exclusive rights to license. With the potential to accrue up to a staggering $1.4 billion in milestone payments from its collaboration with Novartis, this could be a huge win for both parties.
Novartis’ Adakveo, an SCD therapeutic approved in 2019, has recently failed to reduce rates of vaso-occlusive crises in SCD patients in a Phase III study. Although the pharma is currently working with regulators to determine next steps, the findings of the study are providing insight into the complexity of treating this rare and serious condition.
Novartis suffered a blow this year after having to abandon its Huntington’s disease program due to the severity of the side effects uncovered in their benefit-risk assessment. However, this setback has not deterred them from further research, with over 150 projects still in clinical development.
A Race to the Finish
Gene therapy programs are making strides towards treating SCD, some with the finish line in sight and others less certain of success. Yet, this debilitating condition remains a prime target for these ambitious programs.
Graphite Bio, a leader in gene therapy, made a shocking announcement on Thursday that it would be discontinuing its gene therapy for Sickle Cell Disease (SCD) after a trial participant experienced prolonged periods of low blood cell counts. CEO Josh Lehrer said that, given the “evolving treatment landscape”, continuing nula-cel’s development would be an unwise business decision.
The gene therapy space is undergoing a radical transformation, as more and more treatments come closer to being approved. This means that the industry is evolving and opening up new possibilities for those seeking treatment. It’s an exciting time as we await the approval of these therapies, and look forward to the potential benefits they could bring.
Vertex Pharmaceuticals and CRISPR Therapeutics are on the brink of making a breakthrough with exa-cel, a revolutionary one-time treatment for sickle cell disease (SCD) and transfusion-dependent beta-thalassemia. This groundbreaking ex vivo gene therapy utilizes the CRISPR/Cas9 gene-editing technique to edit a patient’s own hematopoietic stem cells and produce high levels of fetal hemoglobin. This remarkable development has been granted a rolling review from the US Food and Drug Administration (FDA), paving the way for its official approval and launch.
In December, the FDA gave the green light to bluebird bio’s lovo-cel, a groundbreaking one-time gene therapy for sickle cell disease (SCD). After a successful review, the company is expected to submit a Biologics License Application (BLA) early this year, bringing hope to those suffering from this debilitating condition.
Editas Medicine has made history as the first to use the revolutionary gene-editing tool AsCas12a to edit human cells in a clinical trial. Their Phase I/II gene therapy has already demonstrated early success in treating sickle cell disease (SCD), offering a potential breakthrough in the fight against this debilitating disorder.