Cell therapy weekly: in vivo CAR-T therapy for autoimmune disorders
This week: The FDA approved an Investigational New Drug application for a gene therapy to treat Parkinson’s disease associated with GBA mutations (PD-GBA), the first patient was dosed in a Phase I trial investigating an in vivo CAR-T candidate for autoimmune disorders, and SpliceBio (Barcelona, Spain) secured US$135 million in Series B funding to advance its gene therapy candidate for retinal disease.
The news highlights:
- IND clearance for Parkinson’s gene therapy
- In vivo CAR-T therapy for autoimmune disorders reaches clinical trials
- Digital solution for long-term follow-up studies
IND clearance for Parkinson’s gene therapy
The FDA has cleared Capsida Biotherapeutics’ (CA, USA) Investigational New Drug application for CAP-003, an intravenously administered gene therapy for PD-GBA.
“PD-GBA is an area of substantial unmet need given the lack of approved treatments that target GCase, which is the protein encoded by the GBA gene, and provide meaningful slowing or halting of disease progression,” said Swati Tole, Chief Medical Officer of Capsida Biotherapeutics. “We recognize the urgency for new treatment approaches, so we are working diligently to initiate the Phase I/II clinical trial for CAP-003 with the aim of dosing the first patient in the third quarter of this year.”
GBA mutations are the most common genetic risk factor for Parkinson disease and affect up to 15% of Parkinson’s patients. CAP-003 utilizes an engineered AAV capsid, which can cross the blood-brain barrier, to deliver a functional GBA1 gene and restore GCase activity.
In vivo CAR-T therapy for autoimmune disorders reaches clinical trials
The first patient has been dosed in Capstan Therapeutics’ (CA, USA) Phase I trial of CPTX2309, an anti-CD19 in vivo CAR-T therapy for B cell-mediated autoimmune disorders. The trial will assess safety, tolerability, pharmacokinetics and pharmacodynamic activity in healthy volunteers.
“With CPTX2309, our therapeutic goal is to achieve immune reset through rapid and profound B cell depletion using a transient, tunable and fully off-the-shelf in vivo CAR-T technology,” said Ramin Farzaneh-Far, Chief Medical Officer at Capstan. “The focus on a Phase I healthy volunteer population allows us to dose escalate efficiently, and importantly, underscores the potential safety advantages of an approach that does not involve lymphodepletion or permanent CAR integration into the genome. The objective of this trial is to determine a pharmacologically active dose (or doses) that can be advanced into Phase II studies to treat patients with autoimmune disease.”
Successful Series B funding for retinal disease gene therapy
SpliceBio has secured US$135 million in Series B financing to advance the clinical development of its gene therapy candidate SB-007 for Stargardt disease, an inherited retinal disorder caused by mutations in the ABCA4 gene. The funding was co-led by EQT Life Sciences (Amsterdam, Netherlands) and Sanofi Ventures (MA, USA), with Participation from Roche Venture Fund (Basel, Switzerland) and other existing investors.
The funding will support SpliceBio’s ongoing interventional Phase I/II ASTRA and observational POLARIS study and help to accelerate the company’s pipeline of AAV programs in other indications using its proprietary protein splicing platform.
The protein splicing approach works by dividing therapeutic genes into segments that are delivered using dual AAV vectors. Each fragment of the therapeutic gene is also flanked by engineered split intein sequences. Following cellular entry, each gene fragment is transcribed and translated, producing fragments of the therapeutic protein with attached split intein segments. These split inteins recognize their complementary partners, bind together, and catalyze a precise splicing reaction that removes the inteins while simultaneously joining the therapeutic protein segments to assemble the full-length, functional protein.
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