Uniqure, Inc.

Kurma Partners raised EUR 140 million in the first closing of Biofund IV, targeting EUR 250 million total. The fund aims for 16-20 new investments, focusing on innovative therapeutics. Three initial investments have been made, and the fund leverages Kurma's networks for success.

Kurma Partners raised €140m for Biofund IV, targeting €250m by 2025. Focused on innovative therapeutics, it aims for 16-20 investments, including SciRhom, Memo Therapeutics, and Avidicure. Kurma's model, proven by Amolyt Pharma, Emergence Therapeutics, and Corlieve Therapeutics acquisitions, attracted investors like Eurazeo, Bpifrance, and CSL.

Pfizer withdraws Oxbryta, its sickle cell disease treatment, from global markets due to increased risks of vaso-occlusive crises and fatal events. The decision follows recent FDA approvals of two gene therapies for SCD, Casgevy and Lyfgenia, which offer potential cures but come with high costs.

uniQure's AMT-191, a gene therapy for Fabry disease, received Orphan Drug Designation from the FDA. The Phase I/IIa trial, initiated in August 2024, aims to assess safety, tolerability, and efficacy through a one-time intravenous infusion. Initial data is expected in 2025.

FDA grants Orphan Drug Designation to uniQure's AMT-191 gene therapy for Fabry disease, with a Phase I/IIa trial underway in the U.S.

The U.S. Translational Regenerative Medicine Market is projected to grow from USD xx.x Billion in 2023 to USD xx.x Billion by 2031, with a CAGR of xx.x%. Key applications include orthopedics, cardiovascular, oncology, wound healing, and others. Major manufacturers include Athersys, Avita Medical, AxoGen, and more. The market's future is promising yet complex, with regional variations and emerging trends.

Recent advancements in neurology include positive phase 1/2 study results for tividenofusp alfa in Hunter syndrome, Delphi-MD for nerve stimulation, VG-3927 for Alzheimer's, and tirzepatide for sleep apnea. Ongoing trials for PGN-EDO51, tolebrutinib, and NS-050/NCNP-03 show promise. FDA discussions for CNM-Au8 and AMT-130, and approvals for Nerivio and STS101 highlight progress in treatment options.