Myriam Mirza PhD MBA

- Working with Founder to create strategies to grow awareness of the disease - Managing research teams working on oligonucleotide technologies - Evaluating potential startups partners - Consulting Founder on strategies for the foundation

- Identifying new researchers and start-ups to work with - Managing research teams working on CLN1 Batten Disease - Working directly with the Founders to create strategies to grow the foundation

• Identified and partnered with clinical and CRO partners to meet project milestones and ensure FDA compliance • Created a budget plan and fundraising/partnering strategy to raise the $4 million required to fund a phase-I clinical trial • Facilitated conversations with commercial partner interested in acquiring IP rights • Managed the coordination of 6 large stakeholder organizations working on research

• Built a gene therapy project from ground-up, including identifying and partnering with preclinical, manufacturing and clinical institutions • Facilitated partnership agreements and set-up for a one-patient exon-skipping IND, now in clinical trials and hailed as a break-through approach by Science Magazine • Co-created a plan with Founder to diversify the foundation and pursue a new direction involving personalized medicine

- Managed over 100 projects, including teams, budgets and clients - Consulted on 400 medical device, Pharma and life-science projects - Analyzed data from secondary and primary sources to create evidence-based strategic reports, recommendations and implementation - Presented reports to clients and board-meetings - Authored a comprehensive report on medical device pricing and reimbursement in the EU sent to clients on behalf of firm

• Organized an international meeting of 40 KOLs stemming from both academia and pharma to brainstorm future direction of CLN3 Disease research • Researched and co-wrote a comprehensive CLN3 dossier resulting in a first-authorship publication set to come out in 2018.

Working as a team in an institute Managing my own projects as well as those of students (in English and German) Teaching techniques to students Communicating my work to scientists and non-scientists at 12 international meetings (in English and German) Writing scientific papers Critically analyzing data and results Making decisions as to how to the project should proceed Making important scientific collaborations

Dr. Katherine Borden is a leading scientist in the field of leukemia and is running a nationwide clinical trial on M4/M5 acute myeloid leukemia. I assisted in doing real time PCR on tissue samples collected from patients in the clinical trial as well as western blots by looking for changes in specific markers in these patients.

Working on Cystic Fibrosis

Under the supervision of Dr. Andrew Peden, I studied the role of VAMPs and SNAREs in post-golgi trafficking. The main objective was to learn how vesicles trafficked to the plasma membrane and back through the lysosomes to the Trans-Golgi Network. This was done by creating mutant cell lines and studying the effects by immunofluorescence.

The cystic fibrosis laboratory of Dr. Frederic Becq works on better understanding CF and finding drugs which can correct the trafficking of the mutated protein CFTR. My project consisted of testing the drug Miglustat on different cell lines as well as improving and teaching the western blotting technique to the mostly patch-clamp laboratory.

Dr. David Y Thomas had recently started working on CF when I joined the group and started undertaking a major compound screen in search for compounds which could push mutated CFTR to the plasma membrane. I assisted in the screen by western blotting the positive hits and observing effects through confocal microscopy.

• Working in collaboration with the Toronto’s Sick Kids Hospital on The Gene Modifier project • Conducting thorough research on the medical history of patients with Cystic Fibrosis • Combining data on Excel databases

Cystic Fibrosis specialist Dr. Larry Lands studies the relationship between CF, chemokines and oxidation in cells. As a learning student I was given a simple project of determining which was the best way to normalize CF assays in papers, either using cell number or protein content. Through protein assays and cell counting it was concluded that cell number was the best form of normalization. The published abstract is mentioned below.

Entrepreneurship and New Venture Capital Grade: Distinction

Characterization of the retinal degeneration and glial activation of neuronal ceroid lipofuscinosis mouse models Cln3Δex7-8 and Cln6nclf and the beneficial effects of dietary supplementation Grade: Magna Cum Laude

Characterization of New CFTR Monoclonal Antibodies