The Irides Weekly Update is our round-up of patent litigation news highlights from around the world. Taking its name from the plural of “iris” - a nod to the ability of Irides to see what’s happening around the world.

Our latest edition - 24 November 2023 - is below:

UK

MHRA approves the world’s first CRISPR-based gene therapy

On 16 November 2023 the MHRA approved a CRISPR-based gene therapy under the brand name CASGEVY. Developed by Vertex and CRISPR Therapeutics, CASGEVY, (which contains exagamglogene autotemcel, otherwise known as “exa-cel”), is the first therapy in the world to be authorised that uses the gene-editing tool CRISPR/Cas9. This approval comes just over a decade after the invention of CRISPR/Cas9, for which its inventors were awarded the Nobel Prize in 2020. This is also the first gene therapy for the treatment of sickle cell disease and transfusion-dependent beta thalassemia (TDT).

Both sickle cell disease and TDT are genetic conditions caused by errors in the gene responsible for the production of adult haemoglobin in red blood cells, meaning that tissues are not sufficiently oxygenated. Patients with these conditions have to have regular blood transfusions and take iron chelators such as EXJADE (deferasirox) to prevent iron overload. CASGEVY works by increasing the production of foetal haemoglobin to ensure adequate oxygenation. Foetal haemoglobin is typically expressed by foetuses during gestation, but is suppressed shortly after birth when a switch to adult haemoglobin production occurs. After the patient’s stem cells are removed from their bone marrow, CASGEVY inactivates the gene that suppresses foetal haemoglobin production (so that the cell is capable of producing foetal haemoglobin) via CRISPR gene-editing technology. This involves causing a double stranded cut in the DNA to inactivate the gene. The edited cells are then infused back into the patient, allowing the body to produce functioning foetal haemoglobin.

The UK authorisation for CASGEVY follows promising results from two global clinical trials. The first trial, for treatment of sickle cell disease, followed 29 participants for a long enough period to find that the therapy relieved 28 of those people of symptoms for at least one year after treatment. The second, for treatment of TDT, followed 42 participants for a long enough period to find that, among those, 39 did not need a red blood cell transfusion (the conventional treatment for this disease) for at least one year. The remaining three had their need for blood transfusions reduced by more than 70%.

CASGEVY is also under review by the FDA and the EMA, with the FDA expected to give a decision on the sickle cell disease indication by 8 December 2023 and the TDT indication by March 2024. An advisory panel to the FDA concluded last month that the benefits of the treatment for sickle cell patients far outweighed risks, such as CRISPR making unintended genetic modifications with unknown side effects. It is therefore widely expected that the 8 December decision will grant approval for the therapy to treat sickle cell disease. A decision from the EMA is expected in early 2024.

TAIWAN

Taiwan becomes eighth country added to the list of countries with a regulatory framework applicable to active substances exported to the European Union

On 10 November 2023, the Commission Implementing Decision amending the Directive 2001/83/EC was published. The Decision established a list of third countries with a regulatory framework applicable to active substances for medical products for human use and the respective control and enforcement activities ensuring a level of protection of public health equivalent to that in the EU, in accordance with Directive 2001/83/EC of the European Parliament and of the Council. In December 2021, Taiwan had made a request to be included in the list of third countries and based on the assessment of this request by the Commission, Taiwan was added to the list on 9 November 2023. Decision here. Taiwan joins Australia, Brazil, Canada, Israel, Japan, South Korea, Switzerland and the USA on the list.

UPC

Paris Central Division rules on the meaning of party under art.33(4)

On 13 November 2023, the Central Division of the Court of First Instance of the Unified Patent Court (UPC) rejected an application to postpone a hearing and reject a preliminary request in a revocation action between Edwards Lifesciences Corporation (the applicant) and Meril Italy srl (the respondent). Meril had filed a revocation action in the Central Division in Paris. Edwards, in response, filed a preliminary objection questioning the competence of the Division Seat, citing a pending infringement action on the same patent at the Munich Local Division. Order here.

The respondent provided written comments within the stipulated 14-day period following the notification of the Preliminary Objection and an oral hearing was scheduled, initially for October 19, 2023, but postponed to October 26, 2023, on the applicant's request. Both parties submitted additional written submissions before the rescheduled hearing. The respondent sought to postpone the oral hearing to adequately respond to the applicant's additional submission. Alternatively, they requested the exclusion of the applicant's new arguments and exhibits or the grant of leave to appeal in case of denial of these requests.

The Judge-rapporteur, Paolo Catallozzi, rejected the respondent's requests, emphasizing fairness and the right to a fair trial as per art. 6 of the European Convention on Human Rights (ECHR). The court held that the purpose of pre-hearing submissions was to facilitate a more efficient hearing, and any new evidence or arguments, unless occurring after the filing of the Preliminary Objection, were not admissible. The exhibits did not introduce new objections or relevant facts, thus not meriting a postponement or exclusion.

In relation to the substance of the hearing, the applicant argued that the respondent and Meril Life Sciences Ltd and Meril GmbH should be considered the "same parties" under art. 33 (4) UPCA, constituting an abuse of the UPC framework. However, the court found no sufficient evidence that the respondent and Meril were legally the "same party", maintaining separate legal personalities under Italian law. The court also rejected the "straw company" theory, finding insufficient evidence that the respondent's actions were attributable to its parent company.

The court ordered that the revocation action by the respondent did not inherently block or bifurcate the infringement action.

The applicant's request for the respondent to provide security for costs was considered but ultimately deemed unnecessary, given the respondent's corporate group backing and lack of evidence of financial difficulties.