Gene therapy firm BioMarin stages haemophilia musical

The California biotech has a gene therapy for haemophilia A in late stage clinical development, and ahead of a potential filing next year it has taken a novel approach to show the wider public how the disease affects patients.

It also hopes to measure the impact on the young people performing the musical, who themselves have haemophilia, to see whether it helped them feel more confident, connected with each other, and better prepared to manage their condition.

BioMarin hopes to take on Roche’s Hemlibra (emicizumab), a prophylactic injection that has recently been approved in haemophilia A, as well as older enzyme replacement therapies from companies such as Shire and Novo Nordisk.

The market for these drugs is expected to be worth billions and BioMarin is hoping to win the hearts and minds of patients and doctors ahead of a potential new approval.

BioMarin’s stage production "Hemophilia: The Musical" is a first-of-its-kind theatrical production featuring 25 students affected by a bleeding disorder.

The musical performance in New York City follows the story of high school students who are working through the challenges of being perceived as "different" due to their medical conditions.

As the musical unfolds, the young adults learn to embrace who they are and follow their dreams, while living with a bleeding disorder.

The performance was managed by Patrick James Lynch, who runs the Los Angeles-based marketing and production agency Believe Ltd. that specialises in bleeding disorders.

Lynch, who has haemophilia himself, recruited the applicants from across the US and led the group to the performance in Times Square.

Preparation for the performance included a three-day intensive workshop that included coaching sessions on the impact of breathing and relaxation on pain management, as well as the psychosocial benefits and therapeutic value of self-expression in the arts.

Jeremy Nobel, Harvard Medical School faculty member and founder and president of the Foundation of Art & Healing’s UnLonely Project, said: "There is an important connection between creative expression and wellness for people living with chronic conditions, like haemophilia, which can be particularly isolating."

Porus Pavri, 14, one of the performers, told Reuters that the weekend represented a chance to get to know other teenagers who understood his daily challenges.

He said: "It is one of the only activities I can do where I can go all out without worrying about my health."

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