Cell therapy weekly: Solid Biosciences faces lawsuits over Duchenne drug
This week: Solid Biosciences Inc. faces multiple filings over claims it misled investors and bone marrow-based cell therapy shows promise in Friedreich’s ataxia.
The news highlights:
[Developing story] Solid Biosciences faces lawsuits over Duchenne drug safety claims
UK group uses cell therapy to reverse Friedreich’s ataxia in mouse model
bluebird bio and Celgene to codevelop CAR-T therapy for multiple myeloma
[Developing story] Solid Biosciences faces lawsuits over Duchenne drug safety claims
Following the halting of Solid Bioscience Inc.’s (MA, USA) SGT-001 trial for Duchenne muscular dystrophy, a number of opportunistic law firms have indicated interested in filing complaints in federal courts. These firms, known for targeting companies whose stocks have dropped, typically argue that executives withheld information from investors. Since the FDA halted the trial, stocks in Solid Bioscience have dropped more than 60%.
The Boston Business Journal (MA, USA) reported that one firm has already filed in Boston federal court, with several others announcing the launch of similar cases soon after. It’s not yet known the likely outcome of these cases, which are likely to be amalgamated into one case, or how this could affect Solid Biosciences and future cell therapy developers.
UK group uses cell therapy to reverse Friedreich’s ataxia in mouse model
A Bristol (UK)-based team has demonstrated that a transplantation of bone marrow cells stimulate neural repair and start to reverse disease pathology in a mouse model of Friedreich’s ataxia (FA). FA is a neurodegenerative disease that leads to reduced levels of frataxin protein and mitochondrial dysfunction. FA patients suffer progressive impairment of movement and other functions as the disease advances.
In the paper, published in Annals of Neurology, the authors comment that “our data provide proofofconcept of gene replacement therapy, via allogeneic bone marrow transplantation, that reverses neurological features of Friedreich’s ataxia with the potential for rapid clinical translation.”
Transplantation of bone marrow cells that express frataxin upregulated native frataxin and other antioxidative proteins, and improved movement and coordination in the mice. The transplanted cells also integrated into damaged nervous system tissue and contributed genetic material to neurons and myelinating Schwann cells. With treatments for FA currently nonexistent, positive results such as these will give the community hope. However, many similar therapies have fallen at the first human hurdle.
bluebird bio and Celgene to codevelop CAR-T therapy for multiple myeloma
bluebird bio (MA, USA) and Celgene Corporation (NJ, USA) have entered into an agreement to co-develop and -promote bb2121 in the USA. bb2121 is an investigational anti-B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cell therapy for the potential treatment of patients with relapsed/refractory multiple myeloma.
“Entering into this codevelopment and co-promotion partnership with Celgene is a significant step forward in building a fully integrated oncology franchise for bluebird and together, we are committed to rapidly advancing development of bb2121 for patients,” said Joanne Smith-Farrell, oncology franchise leader and senior vice president, corporate development and strategy, bluebird bio.
“The collaboration builds upon our extensive research and development capabilities in oncology and is a testament to the strong partnership that exists between our two companies.”
For more weekly cell therapy news, read previous editions of the cell therapy weekly.