Cell therapy weekly: Orphan Drug Designation for Friedreich’s ataxia therapy
This week: ScaleReady (MN, USA) awarded US$150,000 to Verismo Therapeutics (PA, USA) as part of the US$20 million G-Rex Grant initiative, Asimov (MA, USA) launched a suite of tools for gene therapy development and The US Food and Drug Administration (FDA; MD, USA) granted Orphan Drug Designation to an experimental therapy for Friedreich’s ataxia.
The news highlights:
- US$150,000 grant awarded to advance KIR-CAR
- Suite of AAV tools launched
- Orphan Drug Designation for Friedreich’s ataxia therapy
US$150,000 grant awarded to advance KIR-CAR
Verismo Therapeutics, a company developing novel CARs that more accurately reflect the design of killer immunoglobulin-like receptors, has been awarded a US$150,000 G-Rex Grant by ScaleReady. The grant will support Verismo in advancing the development and production of its killer immunoglobulin-like receptor-CAR platform with the use of G-Rex technology.
The G-Rex Grant Program is a US$20 million initiative that was launched this May in partnership with Wilson Wolf Manufacturing, Bio-Techne Corporation (both MN, USA) and CellReady (TX, USA) to accelerate the progress of cell and gene therapies.
“We are grateful to have been chosen for the G-Rex Grant at Verismo Therapeutics,” stated Raymond Luke, Director of Manufacturing Science and Technology at Verismo Therapeutics. “This funding will help us further optimize our cell therapy manufacturing processes by transitioning our active and future pipeline to G-Rex, ensuring we can deliver life-saving treatments to patients more efficiently and cost-effectively. We are excited to work with ScaleReady to drive forward innovative solutions in cell therapy production.”
Suite of AAV tools launched
Asimov has introduced the AAV Edge System, a toolkit designed to support the development and production of AAV gene therapies. This platform aims to streamline the gene therapy process by offering access to various tools in a single solution, addressing challenges related to safety, efficacy, manufacturability and cost within the field. Key features include AI-designed, tissue-specific promoters, DNA sequence optimization and tools to reduce toxicity during production.
Alec Nielsen, co-founder and CEO of Asimov, said, “Our team has been on a roll – AAV Edge is our third launch in cell and gene therapy this year. The cost and safety of gene therapies is top of mind for many in the field, and we’re driven to help our partners on both design and production to enable more of these powerful medicines to reach patients. This is Asimov’s latest application in programming biology, made possible by leveraging AI, synthetic biology, and bioprocess engineering. There’s more to come, and we’re excited to keep pushing the envelope.”
Orphan Drug Designation for Friedreich’s ataxia therapy
The FDA has granted Orphan Drug Designation to Papillon Therapeutics’ (CA, USA) PPL-001, an experimental gene-corrected CD34+ hematopoietic stem and progenitor cell (HSPC) therapy for Friedreich’s ataxia. This therapy employs a targeted excision method to address the GAA repeat expansion in Intron 1 of the FXN gene.
The Orphan Drug Designation program recognizes investigational drugs and biologics aimed at treating rare diseases or conditions that impact fewer than 200,000 individuals in the US. This designation provides several benefits, such as tax credits for clinical trial expenses and exemptions from prescription drug user fees.
“We are pleased that the FDA has granted Orphan Drug Designation to our program PPL-001,” said Carter Cliff, CEO of Papillon Therapeutics. “This significant milestone underscores our commitment to developing groundbreaking therapies for patients with rare, debilitating diseases such as Friedreich’s ataxia. We believe this therapy has the potential to treat multiple symptoms and address the unmet treatment needs of patients and their families.”
