J.P. Morgan 2022: Reaching New Heights, Looking to New Horizons

Reaching New Heights, Looking to New Horizons

Bobby Gaspar, M.D., Ph.D., chief executive officer

40th Annual J.P. Morgan Healthcare Conference

January 13, 2022

From Clinician to CEO: The Evolution of HSC Gene Therapy

Photo courtesy of Great Ormond Street Hospital

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We aspire to end the devastation caused by genetic and other severe diseases through the curative potential of HSC gene therapy.

2021 Progress and Achievements Lays Strong Foundation for 2022

MLD	✓	Libmeldy® (Europe): Initiated commercial launch activities and multiple NBS pilots
✓	OTL-200(U.S.): Received clarity from FDA on the expected clinical and CMC package for a BLA

OTL-203(MPS-IH):

• Presented one-yearproof-of concept data, including cognitive/motor function and growth

MPS ✓ Obtained regulatory guidance on design of a global registrational trial

OTL-201(MPS-IIIA):

• Completed enrollment of five patients in proof-of-concept trial; presented initial study data

R&D ✓ OTL-105(HAE): Signed a strategic collaboration with Pharming Group N.V.

Ended 2021 with approximately $220M of cash and investments; runway into 1H'23

4 |	Libmeldy is approved in the European Union, UK, Iceland, Liechtenstein and Norway. OTL-200 is an investigational therapy in the US.

2022: Reaching New Heights, Looking to New Horizons

Reaching New	Looking to New	The Journey
Heights	Horizons	Ahead

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