Nucleic Acid Therapeutics: From Bench to Bedside Symposium
17-18 February 2027, Bilbao (Spain)

 

PRELIMINARY PROGRAM 

Wednesday, February 17 
 

Nucleic acid therapeutics - From bench to bedside (part 1) 

Bridging the gap between preclinical antisense oligonucleotide (ASO) research and its clinical application. Lectures by renowned experts on the translation process, regulatory pathways, and the sharing of both successful and unsuccessful case studies in clinical trials. 

 

09:00 – 09:05 Welcome – Institutional welcome TBC 

09.05 – 09:10  Welcome - Virginia Arechavala Gomeza, Biobizkaia HRI 

 

09:15 – 12:45 Session 1: The drug 

09:15 – 09:45 Talk 1: Drug development – TBC 

09:45 – 10:15  Talk 2: Delivery challenges – Prasad Wakchaure, AstraZeneca 

10:15 – 10:45   Talk 3: How to define the human dose: PK/PD – Peter Gennemark, AstraZeneca 

10:45 – 11:00   Q&A session 
 

11:00 – 11:30 Coffee break 
 

11:30 – 12:00  Talk 4: AI in drug development – Roxanna Redis, Charles River 

12:00 – 12:30 Talk 5: The role of CROs – Roxanna Redis, Charles River 

12:30 – 12:45  Q&A session 

 

12:45 – 14:00 Lunch break 

 

14:00 – 17:30 Session 2: The patient   

14:00 – 14:30 Talk 1: Patient engagement from Pharma perspective– Florence Kheime, Servier 

14:30 – 15:00 Talk 2: How to choose BOMs, COMs and PROMs – TBA 

15:00 – 15:15  Q&A session 
 

15:15 – 15:45  Coffee break 
 

15:45 – 17:30  Roundtable with patients  

Short presentations followed by a roundtable discussion – moderator: TBA 

• Henri Thélu – Epilepsy syndromes linked to KCNT1 

• Fundación Noelia – Collagen VI Congenital Muscular Dystrophy 

• Albert Carbonell – Glutaric Aciduria Type I TBC 

• SOD1 patient - Amyotrophic Lateral Sclerosis (ALS) TBC 

 

Thursday, February 18 

 

Research Training 3: From bench to bedside (part 2) 

 

09:00 – 12:30 Session 3: Trial design  

09:00 – 09:30 Talk 1: IND Filling– TBA 

09:30 – 10:00  Talk 2: Clinical trial design – Florence Kheime, Servier  

10:00 – 10:30   Talk 3: Regulatory pathways – Marjon Pasmooij, Medicines Evaluation Board  

10:30 – 10:45    Q&A session 
 

10:45 – 11:15     Coffee break 
 

11:15 – 12:00    Talk 4: AntimiR23 for DM1 – Beatriz Llamusí, ARTHEx Biotech 

12:00 – 12:45  Talk 5: TBA - Matthis Synofzic
12:45 – 13:00 Q&A session 

 

13:00 – 14:00 Lunch break 

 

14:00 – 17:30 Session 4: Stories  

14:00 – 14:40 Talk 1: N-of-1 Stargardt disease – Rob Collin, Radboudumc 

14:40 – 15:20 Talk 2: Silence ALS program – Miguel Angel Rubio, Hospital del Mar Research Institute. 

15:20 – 15:50 Coffee break 
 

15:50 – 16:50 Keynote Lecture: TBA 
 

17:00 – Closing