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Antibody/Gene Therapy Biotech Gains $138 Million in Early Funds

Adeno-associated virus

Adeno-associated virus diagram (Lawrence Berkeley National Lab)

13 Nov. 2023. A developer of synthetic antibodies for neurodegenerative disorders delivered like gene therapies is raising €129 million ($US 138 million) in its first venture funding round. VectorY Therapeutics BV in Amsterdam, The Netherlands plans to initially use the proceeds to advance its lead program, a treatment to delay progression of amyotrophic lateral sclerosis or ALS.

VectorY Therapeutics is a three year-old biotechnology enterprise founded by the Dutch life science venture investor Forbion to advance gene therapies for degenerative nervous system diseases such as ALS. The company designs and delivers synthetic antibodies aimed at clearing specific toxic proteins or lipids in brain cells or elsewhere in the central nervous system. VectorY says its antibodies are fused with degrons, amino acid tags identifying unique protein degradation sequences that provide precise targeting.

The company delivers these payloads with adeno-associated viruses or AAVs, benign viruses with single strands of DNA that allow for adding genetic materials. AAVs have become delivery workhorses for gene therapies. However, VectorY Therapeutics alters the capsid or outer shell of its AAVs to help penetrate the blood-brain barrier, a normally protective mechanism in the brain that also limits the reach of many medications. In addition, the company says it built a manufacturing platform based on baculoviruses that in nature infect insects, but are engineered in this case to reliably produce large-scale quantities of AAV vectors.

Targeting toxic proteins in brain cells

The lead product at VectorY Therapeutics, code-named VTx-002, applies the company process to address ALS, also known as Lou Gehrig’s disease that occurs when motor neurons in the central nervous system begin wasting away. People with ALS lose their ability to first control voluntary movements, such as standing and walking, then often progresses to walking, chewing, and swallowing, and eventually breathing. VTx-002 targets misfolded TAR DNA-binding protein 43 or TDP-43 in neurons associated with ALS, to clear accumulations of toxic misfolded TDP-43 and allow normal TDP-43 to form in its place. Company representatives presented preclinical lab evidence of this process at a scientific meeting in Dec. 2022.

VectorY Therapeutics is raising €129 million in its first venture financing round, called series A, led by life science investors Forbion in Narden, The Netherlands that founded the company and EQT Life Sciences in Amsterdam. Joining the round are MRL Ventures Fund, the corporate venture arm of drug maker Merck, ALS Investment Fund, BioGeneration Ventures, and others. According to Crunchbase, VectorY raised €31 million ($US 38 million) in seed funds in June 2021.

The company plans to first apply the proceeds for advancing VTx-002. “The investment,” says VectorY Therapeutics CEO Sander van Deventer in a company statement, “will enable us to advance our lead program VTx-002, a potentially disease-modifying therapy for ALS, into clinical development. Our program is uniquely positioned to address TDP-43 pathology, which underlies the disease in the vast majority of ALS patients. The series A will also support advancement of additional pipeline programs targeting proteinopathies in neurodegenerative diseases demonstrating the broad potential of our platform.”

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