History

In 2013, a team of scientists from the Institute of Bioorganic Chemistry of the Polish Academy of Sciences in Poznań, led by Professor Marta Olejniczak, filed the first patent protecting a technology for the selective silencing of the mutated gene responsible for Huntington’s disease and other CAG-repeat disorders, including SCA3 and DRPLA. The filing preceded a similar solution developed by the Dutch-American company uniQure by one year, which helped secure the uniqueness of the Polish know-how against global competition.

In 2019, the project – then at an early stage of in vitro research on cell lines – attracted the interest of Dr Kris Siemionow, a physician, entrepreneur and investor associated with the Biofund Capital Management fund. Biofund financed the first animal-model studies, opening the way for further development of technology.

In 2019–2020, the research team confirmed effective silencing of mutated genes in cells derived from patients with Huntington’s disease, SCA3 and DRPLA. In 2020, however, adverse side effects, including epileptic seizures, were observed in mouse studies, prompting the team to return to the molecule design stage.

In 2022, a new generation of the molecule – amiR136-A2 – was developed, eliminating the toxicity problem of the previous version. The drug candidate based on it met both safety and efficacy criteria.

In 2024, a further patent application was filed, extending the intellectual property protection of the project.

In 2024–2026, work continued on a drug candidate with an improved safety profile and a two-fold lower dose compared with the earlier version of the molecule.

From April 2026, the multiQure project began to be combined with Pure Biologics – a company listed on the Warsaw Stock Exchange since 2020. Biofund Capital Management entered into negotiations regarding the contribution of the multiQure RNAi platform to Pure Biologics in exchange for newly issued shares.

On 3 June 2026, Biofund signed a letter of intent with ACRX – until then the largest shareholder of Pure Biologics – concerning the contribution of the multiQure platform to the company as an in-kind contribution and the conversion of a loan previously granted by ACRX into shares. On 10 June 2026, the Extraordinary General Meeting of Pure Biologics approved the transaction: as a result, Biofund acquired 49.99% of the company’s shares, and independent experts from Baker Tilly valued the multiQure project at its current stage at over PLN 400 million (USD 111.3 million).

On 23 June 2026, Pure Biologics raised PLN 50 million from the issue of 50 million series S shares in an accelerated bookbuilding (ABB) process. The proceeds were allocated to the further development of the multiQure RNAi platform and the continuation of the PB003G and PB004 projects towards Phase I clinical trials. Following the transaction, the company adopted a plan to change its name to multiQure.

Key results of 12 years of work on the project

• An approximately 50% reduction in mutant HTT protein production achieved in humanised YAC128 mouse models.
• No adverse effects in safety studies in mice.
• Confirmed durable expression of the silencing RNA molecule – the silencing effect was maintained throughout the entire study period.
• Research by Professor Marta Olejniczak’s team published in globally recognised scientific journals.