BIOMEET SESSIONS 2023 | Rare Diseases: Shaping the future with innovative breakthroughs

Date and Location

May 30, 2023, 17h00 (UTC+1). Streaming via Zoom.


This session of BIOMEET Sessions 2023 is dedicated to therapeutic innovation in the field of Rare Diseases. The session will feature a panel of experts from leading companies in the industry, working in different areas, who will share the latest therapeutic advances and innovative approaches in the research lines they are working on.

This session is organized by the P-BIO Rare Disease Working Group and will be held online, in English. Follow the streaming on Zoom.


Vasant Jadhav is a Senior Vice President of Research at Alnylam Pharmaceuticals, where he leads the Alnylam Technology group responsible for siRNA designs, new applications and delivery solutions that have led to 5 approved drugs and deep pipeline across multiple tissue targets.

Prior to Alnylam, Vasant was at Sirna/Merck for 12 years and worked on optimizing siRNA chemical modifications and leading the efforts on conjugate delivery approach. Dr. Jadhav obtained his PhD in Biotechnology from the University of Pune in 1998 and post-doctoral fellowship at University of Colorado Boulder, working on ribozymes.

He has authored >30 peer-reviewed publication in the field of nucleic acids therapeutics.

VASANT JADHAV | Senior Vice President of Research | Alnylam Pharmaceuticals

Pablo Rendo is Global Project Head (GPH) of Rare Disease and Red Blood Disorders Gene Therapy Programs, at Sanofi, since 2019. Prior to this position, he worked as Global Clinical Lead of the Hemophilia Program at Pfizer, from 2009 to 2019, having designed the gene therapy clinical plan for Hemophilia A and B.

Pablo has been working in the pharmaceutical industry for 20 years, as Head of Clinical Research at BIOSIDUS, from 1992 to 2003, as Medical Advisor at Aventis, from 2003 to 2005, and as RMM for Wyeth in Argentina, from 2005 to 2009.

Pablo obtained his MD from the University of Buenos Aires, Argentina in 1982 , with a specialization in hematology.

PABLO RENDO | GPH, Rare Disease and Red Blood Disorders Gene Therapy Programs | Sanofi

Sian joined PTC in 2015 and she is responsible for patient engagement and advocacy strategy for Europe, Middle East and Africa (EMEA).

Sian leads the EMEA patient advocacy team to ensure that patient insights inform PTC’s programs and initiatives.

Sian has over 20 years’ experience in the pharmaceutical industry, of which 16 have been dedicated to rare disease.

SIAN O’NEILL | Senior Director Patient Advocacy | PTC Therapeutics


Margarida Menezes Ferreira is a biologist by training, PhD in Medical Biochemistry (Université d’ Aix-Marseille II in France) and has a post-graduate fellowship at the National Institutes of Health, NIH, USA. She has been guest professor at Lisbon and Coimbra Universities.

Margarida worked for 23 years as a researcher at INFARMED – National Authority for Medicines and Health Products, Portugal, and was a member of the Biologics Working Party representing Portugal at the EMA – European Medicines Agency for 20 years. She was actively involved in the EU regulatory framework for ATMPs(Advanced Therapy Medicinal Products).

She is presently an independent consultant providing strategic orientation for ATMP quality development.

MARGARIDA MENEZES FERREIRA | Quality Expert for ATMPs and other Biological Medicinal Products


17h00 – 17h10


Filipa Sacadura, P-BIO

Margarida Menezes Ferreira

17h10 – 17h30

RNAi in the era of nucleic acid-based therapeutics

Vasant Jadhav, Alnylam Pharmaceuticals

17h30 – 17h50

Optimizing the clinical development strategies tp accelerate the timing of the AAV (Adeno associated virus) clinical plan

Pablo Rendo, Sanofi

17h50 – 18h10

Patient engagement at PTC Therapeutics: The importance of involving patients and caregivers in medicines research and development

Sian O’Neill, PTC Therapeutics

18h10 – 18h50


Moderator: Margarida Menezes Ferreira

Vasant Jadhav, Alnylam Pharmaceuticals

Pablo Rendo, Sanofi

Sian O’Neill, PTC Therapeutics

Note: The session will be online and in English.