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Healx and Bit Bio engage patients with rare diseases for public webinar

The Healx team pictured last year
The Healx team pictured last year

A rare disease webinar on August 13 will allow members of the public and patient groups to engage with Healx, the AI-powered, patient-inspired technology company, to learn how to decode the secrets of those rare diseases yet to be fully researched – the rare rare diseases.

Hills Road-based Healx – winner of AI Company of the Year at last year’s Cambridge Independent Science and Technology Awards –works closely with patient groups to accelerate the discovery and development of rare disease treatments.

The company was co-founded in 2014 by Dr Tim Guilliams and Dr David Brown, who is credited with the development of Viagra.

The event, which also involves Bit Bio, Charles River Laboratories and CureSHANK, highlights the commitment from Cambridge’s synthetic biology community to support the development of new data models for little-understood patient groups. It is the latest in a stream of patient-focused webinars, and will explore how to develop in vitro models for rare disease research.

Last year, Healx formed the Rare Treatment Accelerator, a partnering programme that gives patient groups and Healx the opportunity to work together to quickly discover and develop repurposed treatments for rare diseases using artificial intelligence.

Many patient groups are already participating but, as Karine Proulx, drug discovery alliance manager (patient group partnerships) at Healx, explains, there’s more work to be done.

“This webinar is for anyone – public and patient group – who wants to learn more about how to work with industry on rare disease research,” Dr Proulx told the Cambridge Independent. “We work with a lot of patient groups within our portfolio of rare disease projects, and this webinar is aimed at individuals and groups we don’t already work with.”

“The Rare Treatment Accelerator was launched last year to help us identify patient groups to collaborate with, but unfortunately we couldn’t work with every group that applied. The webinar series is an extension of the accelerator, and is an opportunity for patient groups and the public to gain tools and tips for working with industry partners in the future. We work closely with other companies and organisations in this space to show the different elements of industry-patient group partnerships.”

Dr Karine Proulx, drug discovery alliance manager (patient group partnerships) at Healx
Dr Karine Proulx, drug discovery alliance manager (patient group partnerships) at Healx

One of those other companies, Bit Bio, is also highly invested in the webinar. The synthetic biology enterprise based at the Babraham Research Campus, won Start-up of the Year 2018 Cambridge Independent Science and Technology Awards, back when it was named Elpis BioMed.

It applies the principles of computation to biology to decode cellular identity and provide human cells for research, drug discovery, and a new generation of medicines.

The current focus is to develop a scalable technology platform capable of producing consistent batches of every human cell.

Healx invited us to speak,” says Dr Farah Patell-Socha, VP product management at Bit Bio. “This is a very relevant topic. The lack of disease-specific human models is a big challenge for finding cures for rare diseases. Speaking with CureSHANK and Healx has only confirmed this shortage. Our principal investigator, Dr Tüzer Kalkan, will be speaking alongside Healx and our partner, Charles River Laboratories. Bit Bio is strengthening its focus on developing human disease models and this webinar will highlight this.”

“The event will discuss the importance of developing reliable in vitro human models for studying rare diseases,” says Dr Kalkan. “Human biology differs from the animal models and cell lines used for drug development. A reliable and scalable source of human cells is needed to improve the efficiency of drug development for rare diseases.

“Bit Bio’s cell type-specific transcription factor discovery platform and opti-ox cellular reprogramming technology can generate human cells with high consistency and functionality at scale from human induced pluripotent stem cells – iPSCs – including patient-derived iPSCs or those carrying rare disease-specific mutations.”

It’s an elegant sequence of speakers who will discuss if companies like Bit Bio can create reliable human models for rare diseases. Healx has the patient groups to think of. Charles River provides essential products and services to help pharmaceutical and biotechnology companies, government agencies and leading academic institutions around the globe accelerate their research and drug development efforts.

Bit Bio is now based at BioMed@Babraham
Bit Bio is now based at BioMed@Babraham

CureSHANK is a US-based patient-led organisation solely dedicated to accelerate the development of treatments for Phelan-McDermid Syndrome and SHANK-related disorders.

CureSHANK’s president and co-founder is Geraldine Bliss, whose son was diagnosed with Phelan-McDermid syndrome aged six. She joined the only family group dedicated to understanding the rare condition. Alarmed that there were no treatments coming along, she co-founded CureSHANKS to focus on developing therapies for the condition. Geraldine is the fourth speaker on the day. The one-hour event includes a Q&A.

Researching and building datasets for all rare diseases is vital, not least because there is less money to be made on treating rare diseases, so scalable and affordable human cell models are even more critical to support R&D.

Jo Balfour, operations manager at Cambridge Rare Disease Network, said: “I will definitely be tuning in for the Healx webinar on the 13th and we’ll promote this to our followers too.

“The beauty of online interacting is that those who wouldn’t ordinarily be able to attend can.”

Registration details here.

Check healx.io/news for webinar details
Check healx.io/news for webinar details

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