Gene Therapies

Over 140 biopharma leaders share their views on developments to watch for in key therapeutic areas this year. Advances in multiple scientific fields are opening up new avenues for treatment.

Nippon Shinyaku has revised upward its US sales forecast for DMD drug Viltepso, which has overcome multiple challenges in building its position in the US.

Cancer retained its crown as 37 novel drugs were approved for pan-EU marketing last year; treatments for blood disorders followed closely behind in what was another slow year at the European Medicines Agency.

After ditching its clinical-stage ex vivo candidate in December, the CRISPR-based company is starting all over again with a focus on in vivo therapies and upregulating beneficial genes.

The Swiss giant’s blockbuster gene therapy is currently only administered intravenously into patients with spinal muscular atrophy under two years of age. Its intrathecal candidate, called OAV-101, has just passed a Phase III test which could expand its use in children and young adults.

China's Belief BioMed presented promising new Phase III results at ASH for its AAV gene therapy for hemophilia B, although the wider China CGT space continues to be dominated by oncology.

UniQure said discussions with the FDA resulted in a path forward for seeking accelerated approval of its gene therapy AMT-130 in Huntington’s disease.

The Swiss pharma acquired Kate Therapeutics for up to $1.1bn, gaining preclinical gene therapies for neuromuscular conditions and novel platform technology.

The company is trading below cash after admitting that an adverse event in a Rett syndrome trial left a patient critically ill.

Following ex-US approvals, PTC obtains a US OK for Kebilidi, branded Upstaza outside the US, as a gene therapy for ultra-rare AADC deficiency. The drug is administered via neurological surgery.

The biotech anticipates treating 74 patients with its gene therapies this year, with 30 more already scheduled for 2025.

Analysts also looked into how much differentiation there was between data for the gene therapy NGN-401 and what Taysha has shown with TSHA-102 in the same disease.

The otherwise encouraging data were marred by one patient death, but the company attributed it to the busulfan conditioning regimen and not BEAM-101.

The gene-editing therapy produced functional cures in eight of 11 patients receiving the go-forward 50mg dose, but the rate fell short of what some investors had hoped for.

Multiple Japanese ventures presented updates on their novel pipeline assets and modalities at BioJapan, including cell and gene therapies and combination treatments, despite lingering financing challenges.

Ocuphire will shift toward gene therapy for inherited retinal degeneration via a merger that will take Opus Genetics public. The firm will also be responsible for one of Ocuphire’s pipeline projects.

ARM’s Cell and Gene Meeting on the Mesa offered optimism that a biopharma financial market recovery is under way, but cell and gene therapies still are seen as risky investments.

Big pharma executives at ARM’s Cell and Gene Meeting on the Mesa shared some of the practical challenges of bringing one-time treatments to market in hemophilia, cancer and beyond.

With more advanced therapies gaining approval, companies at ARM’s Cell and Gene Meeting on the Mesa considered hurdles to making their products more broadly available and developing them for larger indications.

Purespring has just raised $105m from an investor syndicate who are betting it can develop the first gene therapy for kidney disease and learn from setbacks experienced in the space.