Aera Therapeutics has nominated AERA-109, a targeted in vivo CAR-T therapy designed to treat multiple B cell-mediated autoimmune diseases, as the company's first development candidate. The announcement follows the presentation of compelling preclinical data at the 10th Annual CAR-TCR Summit, demonstrating the therapy's potential to transform autoimmune disease treatment through innovative delivery technology.
Preclinical Data Shows Deep B Cell Depletion
The preclinical data presented at the summit demonstrated deep depletion of B cells in blood and tissue in humanized mouse models and non-human primates. AERA-109 showed an optimized delivery profile with highly specific and dose-dependent CAR-T cell generation, supporting its advancement as a development candidate.
"We are very encouraged by the deep B cell depletion we observed in non-human primates," said Akin Akinc, Ph.D., chief executive officer of Aera Therapeutics. The company plans to advance AERA-109 into clinical development in mid-2026.
Revolutionary In Vivo CAR-T Approach
AERA-109 leverages Aera's proprietary targeted lipid nanoparticle (tLNP) delivery platform and CAR-T technology to reprogram immune cells directly inside the body. This approach offers a potentially transformative method for treating serious autoimmune conditions with greater precision and improved safety compared to traditional ex vivo cell therapies.
"By enabling in vivo generation of CAR-T cells with precise tissue targeting, AERA-109 has the potential to address many of the logistical, safety, and scalability challenges of traditional ex vivo cell therapies," Akinc explained.
Addressing Manufacturing and Safety Challenges
The innovative approach eliminates several limitations of conventional CAR-T therapies. According to Bill Querbes, Ph.D., chief scientific officer of Aera, "This approach eliminates the need for lymphodepletion, complex manufacturing processes, or use of viral vectors that carry the risk of insertional mutagenesis."
The company has developed a formulation process that enables one-step targeted LNP generation without requiring a separate post-formulation conjugation step to incorporate a targeting ligand. This streamlined manufacturing process could significantly improve the accessibility and scalability of CAR-T therapy.
Company Background and Vision
Aera Therapeutics is a biotechnology company focused on harnessing next-generation enabling delivery technologies and precision payloads to unlock the full potential of genetic medicines. The company has multiple proprietary delivery platforms including targeted lipid nanoparticles (tLNPs), antibody oligonucleotide conjugates (AOCs), and protein nanoparticles (PNPs).
The Cambridge, Massachusetts-based company has raised $193 million to date from leading life sciences investors. Aera's vision is to expand the reach of genetic medicines to different tissues and applications to benefit more patients across more disease areas.
"The nomination of AERA-109 as our first development candidate represents a major step forward for Aera and is a compelling demonstration of our delivery first approach to significantly expand the reach of genetic-based medicines," Akinc stated.
