Privately held Dyno Therapeutics Inc. has added another notch to its adeno-associated virus (AAV) vectors development portfolio in a deal with the Roche Group that includes $50 million up front and ultimately could top $1 billion. Dyno will help in developing next-generation AAV vectors, optimized by artificial intelligence, to target neurological diseases.
Privately held Dyno Therapeutics Inc. has added another notch to its adeno-associated virus (AAV) vectors development portfolio in a deal with the Roche Group that includes $50 million up front and ultimately could top $1 billion. Dyno will help in developing next-generation AAV vectors, optimized by artificial intelligence, to target neurological diseases.
Vironexis Biotherapeutics Inc. has come out of stealth mode, disclosing that it has more than 10 product candidates it’s been developing over the last three years. The therapies are built on the company’s AAV-based platform, Transjoin, which is designed to have patients' livers express bispecific antibodies that bind to both CD3 on T-cells and various targets on tumor cells.
Vironexis Biotherapeutics Inc. came out of stealth mode today, disclosing that it has more than 10 product candidates it’s been developing over the last three years. The therapies are built on the company’s AAV-based platform, Transjoin, which is designed to have patients' livers express bispecific antibodies that bind to both CD3 on T-cells and various targets on tumor cells.
Ascidian Therapeutics Inc. recently provided preclinical data for ACDN-01, an AAV-encoded RNA exon editor targeting ABCA4, being developed for the treatment of ABCA4-related retinopathies, including Stargardt disease.
Philadelphia-based Latus Bio Inc., co-founded by serial biotech entrepreneurs P. Peter Ghoroghchian and Beverly Davidson, launched on May 2 with two lead adeno-associated virus (AAV)-based gene therapy candidates and $54 million in a series A financing.
Pfizer Inc.’s Beqvez (fidanacogene elaparvovec) won FDA approval for use in adults with hemophilia B, making it the second adeno-associated viral (AAV) vector-based gene therapy available for patients in the U.S., following the late 2022 approval of CSL Behring’s Hemgenix (etranacogene dezaparvovec).
The hearing has returned for the first person who has received gene therapy for treating genetic hearing loss in the U.S. Initial results from Akouos Inc.’s phase I/II study showed that within 30 days of receiving AK-OTOF-101, pharmacologic hearing was restored to an 11-year-old who had profound hearing loss from birth.
After the initial approvals in monogenic inherited diseases, the scope of gene therapy is widening, with new delivery routes, novel vectors, cell-specific targeting and products aiming to treat chronic disorders, all making headway in 2023.
Takara Bio Inc. has announced the development of a novel adeno-associated virus (AAV) vector, Sonuaav, which exhibits high gene transfer efficiency into inner ear tissues, with a collaborator at Juntendo University School of Medicine.
