Exploring cell therapy’s potential in autoimmune disease
This week, as we anxiously await the FDA’s thumbs-up/thumbs-down on exa-cel, Switzerland-based CRISPR Therapeutics made other moves in the world of cell therapy.
Earlier this week, CRISPR shared that it is switching up its cell therapy plans, venturing into autoimmune disorders. Specifically, the biotech announced that it is expanding the evaluation of its allogeneic CAR-T candidate, CTX112, beyond oncology into autoimmune diseases. According to CRISPR, early clinical studies have shown that CD19-directed autologous CAR-T therapy can produce long-lasting remissions in multiple autoimmune indications.
Last fall, a paper in Nature made waves in the autoimmune space when it detailed how five patients with lupus enrolled in a compassionate-use CAR T-cell program achieved remission after three months.
CRISPR thinks CTX112 has the potential to provide similar results with several advantages, including greater scalability, lower cost of goods, and no patient apheresis. The biotech plans to initiate a clinical trial in systemic lupus erythematosus in the first half of 2024, with the potential to expand into additional autoimmune indications in the future.
The move could be lucrative for CRISPR from a business perspective. Whereas the patient population eligible for a gene-editing treatment for diseases such as sickle cell is limited, the autoimmunity sector includes more than 80 disorders affecting more than 20 million people in the U.S.
More importantly, if cell therapies prove to have a curative effect on autoimmune disease, it could represent a paradigm shift for cell therapy treatments. —Karen Langhauser
AbbVie on the hunt
This week, we reported that AbbVie is set to acquire Cerevel Therapeutics in a $8.7 billion deal, strengthening its position in the neuroscience sector with a focus on psychiatric and neurological disorders.
Cerevel's assets include emraclidine, a positive allosteric modulator in phase 2 trials for schizophrenia, and a pipeline featuring tavapadon for Parkinson's disease, CVL-354 for major depressive disorder, and darigabat for treatment-resistant epilepsy and panic disorder.
The acquisition is expected to be finalized by mid-2024, contingent on regulatory approvals and shareholder consent.
Unlike other drugs of its kind, emraclidine possesses M4 receptor subtypes that are selectively expressed in the striatum, a subcortical region of the brain located near the center. The activation of these receptors has been shown to indirectly regulate dopamine levels without blocking D2/D3 receptors. This differentiation is essential, as it helps prevent the undesirable motor side effects commonly associated with current antipsychotics.
This marks the second recent acquisition for AbbVie, after recently announcing a $10.1 billion purchase of ImmunoGen, marking an entry into the ovarian cancer space and giving AbbVie access to ImmunoGen's promising next-generation ADC pipeline with transformative potential across solid tumors and hematologic malignancies.
While the ink is still drying on these year-end transactions, 2024 is looking strong for AbbVie. — Andrea Corona