What I’m doing now: from Biomedical Science to Medicine | Biosciences at Sheffield

Mélina Li How Cheong student profile

I graduated from the Biomedical Science course at the university of Sheffield back in 2015 and am now in my 2nd year of Medicine at the University of Buckingham.

I know of a few graduates from my Sheffield cohort that are now also studying to become a doctor and similarly, there are quite a few other people on my medical course who have also already completed an undergraduate degree. In comparison to other biomedical science courses, I believe that Sheffield has given me a significant advantage in preparing me for my degree in medicine.

At the end of secondary school, I had initially applied to the University of Sheffield for medicine. Unfortunately I did not get in, but the university was willing to accept me for the biomedical science course.

This ended up being very beneficial in preparing me for my future studies in medicine. I really enjoyed the course because I thought that there was a good balance between molecular biology and physiology. Additionally, the lecturers often made sure to relate the lecture contents to how they applied in clinical medicine and in real life.

When starting the medical course, I quickly noticed that I had a strong foundation in human physiology compared to my fellow students. It seemed that a lot of other biomedical courses were more heavily focused on the molecular and cellular aspect of biology.

Another key advantage that the biomedical science course at Sheffield offered me was the anatomy module, which was by far my favourite part of the course.

Even though I had not studied anatomy for over 2 years before starting it in medical school, everything came back to me very quickly and made that part of the course very easy for me

Mélina Li How Cheong

BSc Biomedical Science

In second and third year, we got to do dissections on cadavers, an invaluable experience as not all universities can offer this. Even a lot of medical schools nowadays do not have the facilities to offer dissections as part of their anatomy course.

The quality of teaching was of a very high standard which gave me a thorough understanding of human anatomy. Therefore, even though I had not studied anatomy for over 2 years before starting it in medical school, everything came back to me very quickly and made that part of the course very easy for me.

Additionally, after graduating, I had the opportunity to work as an Anatomy Demonstrator for a semester at the University of Sheffield, which gave me valuable work experience in teaching, as well as allowing me to further consolidate my knowledge of human anatomy.

Soon I will be starting my clinical rotations. I do not yet know what specialty I will be going into as I want to keep an open mind and explore as I work in different departments. One thing I do know is that I want to get involved in teaching at some point in my career.

It is through my experience as an

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Ultragenyx and Solid Biosciences Announce Strategic Collaboration to Develop and Commercialize New Gene Therapies for Duchenne Muscular Dystrophy

Collaboration combines Solid’s differentiated microdystrophin construct and Ultragenyx’s HeLa PCL manufacturing platform for use with AAV8 and variants

Solid receives $40 million upfront via equity investment at a premium; up to $255 million in milestones plus royalty payments

Solid retains exclusive rights to all other uses of its microdystrophins, including its existing SGT-001 program

NOVATO, Calif. and CAMBRIDGE, Mass., Oct. 23, 2020 (GLOBE NEWSWIRE) — Ultragenyx Pharmaceutical Inc. (Nasdaq: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, and Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced a strategic collaboration and license agreement to focus on the development and commercialization of new gene therapies for Duchenne. The parties will collaborate to develop products that combine Solid’s differentiated microdystrophin construct, Ultragenyx’s HeLa producer cell line (PCL) manufacturing platform, and AAV8 variants. The collaboration also brings together Solid’s expertise in muscle biology and Ultragenyx’s expertise in bringing novel therapies to patients with rare diseases.

Under the terms of the collaboration, Solid granted Ultragenyx an exclusive license for any pharmaceutical product that expresses Solid’s proprietary microdystrophin construct from AAV8 and variants thereof in clade E for use in the treatment of Duchenne and other diseases resulting from lack of functional dystrophin, including Becker muscular dystrophy. Ultragenyx has made a $40 million investment in Solid and has agreed to pay up to $255 million in cumulative milestone payments per product upon achievement of specified milestone events, and tiered royalties on worldwide net sales at low double digit to mid-teens percentages. Upon achievement of proof-of-concept, Solid has the right to opt-in to co-fund collaboration programs in return for participation in a profit share or increased royalty payments.

“We believe that Solid’s microdystrophin is best-in-class with its unique neuronal nitric oxide synthase binding domain,” said Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx. “By using an AAV8 variant validated in prior human and other studies combined with our scalable, efficient HeLa producer cell line platform, we believe we can leverage our mutual strengths to develop a high-quality AAV-based treatment alternative for Duchenne.”

“Ultragenyx has a demonstrated track record of success in developing and commercializing innovative therapies for rare diseases,” said Ilan Ganot, Co-Founder, President and Chief Executive Officer at Solid Biosciences. “We believe it is the partner of choice for exploring new gene therapy opportunities for patients with Duchenne.”

Solid’s proprietary microdystrophin construct has exhibited functional benefit in preclinical models. In preclinical studies, animals expressing a microdystrophin capable of restoring neuronal nitric oxide synthase (nNOS) resisted fatigue better than those expressing a microdystrophin that does not. Patients dosed with Solid’s proprietary microdystrophin construct at the 2E14 vg/kg dose in Solid’s ongoing IGNITE DMD clinical trial have also preliminarily demonstrated nNOS activity and function, further validating these preclinical results. Solid expects to dose the next patient in the IGNITE DMD clinical

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