Cell therapy weekly: FDA authorizes CASGEVY for beta thalassemia
This week: The US Food and Drug Administration (FDA; MD, USA) expands the use of CASGEVY for beta thalassemia, Therapeutic Solutions International, Inc. (ID, USA) launches Epilepsy Bio, Inc. to deliver a clinical stem cell therapy for epilepsy and Alaya.bio (MA, USA) and Memorial Sloan Kettering Cancer Centre’s (MSK; NY, USA) Michel Sadelain Lab partner to advance CAR-T therapy delivery.
The news highlights:
- FDA approves the use of CASGEVY for transfusion-dependent beta thalassemia
- Therapeutics Solutions launches new company that leverages stem cells to treat epilepsy
- Alaya.bio and MSK researcher’s eye the advancement of in situ CAR-T therapy
FDA approves the use of CASGEVY for transfusion-dependent beta thalassemia
Last month, CASGEVY became the first CRISPR/Cas9 gene-edited cell therapy ever authorized by the US FDA. Initially authorized for sickle-cell disease it has now been extended to another hematological disorder – transfusion-dependent beta thalassemia (TDT) – for patients 12 years and older.
“On the heels of the historic FDA approval of CASGEVY for sickle-cell disease, it is exciting to now secure approval for TDT well ahead of the PDUFA date,” said Reshma Kewalramani, CEO and President of Vertex (MA, USA), the pharmaceutical company that produces CASGEVY. “TDT patients deserve new, potentially curative treatment options, and we look forward to bringing CASGEVY to eligible patients who are waiting.”
Therapeutics Solutions launches new company that leverages stem cells to treat epilepsy
Therapeutic Solutions has expanded its efforts to treat epilepsy by forming a subsidiary – Epilepsy Bio – dedicated to commercializing Therapeutic Solutions’ current intellectual property regarding the clinical and mechanistic findings of epilepsy-treating stem cell products.
“Therapeutic Solutions International has amassed a significant portfolio of intellectual property, trade secrets and collaborations in the area of applied immunology and regenerative medicine,” explained Timothy Dixon, President and CEO of Therapeutic Solutions and co-inventor of the intellectual property. “As these technologies pass the proof-of-concept phase, we believe that spinning them out as independent entities will create significantly more value for shareholders, as well as allow more flexibility for entry into licensing and [merger and acquisition] deals. This marks our ninth subsidiary.”
Alaya.bio and MSK researcher’s eye the advancement of in situ CAR-T therapy
MSK’s Sadelain lab, a pioneer of CAR-T technology, has signed a license and sponsored research agreement with Alaya.bio. This will see the Sadelain lab lead activities to advance Alaya.bio’s novel in situ polymeric delivery platform for CAR-T therapy into the clinic for a chosen hematologic cancer.
“The ingenuity of Alaya.bio’s targeting polymeric nanoparticles has the potential to simplify and refine the development, manufacturing and administration of CAR-T cell therapies in ways that expand applications and make them more accessible to patients,” commented CAR-T pioneer and lab leader, Michel Sadelain. “I am excited to leverage this technology and support its advancement on the frontiers of cell and gene therapy for patients.”
Last week in cell and gene therapy:
Advancing CAR-T therapies with decoy-resistant IL-18
Read how Simcha Therapeutics partnered with Janssen Biotech to enhance CAR T-cell therapies using interleukin-18 technology, alongside other stories from the week.
