3rd European ATTR Amyloidosis meeting for Patientsand Doctors

6-8 Sept. 2021

PRELIMINARY PROGRAMME

3rd European ATTR Amyloidosis meeting for Patients and Doctors I 2

Welcome to ATTR congressThe essential event for all of the Amyloidosis community!

ATTR will be hosted 100% virtually on September 6-8. 2 Channels, one dedicated to doctors one dedicated to patient associations across 3 days delivered a packed and high-quality scientific programme including cutting-edge lectures by international leaders in their field, sessions in collaboration with partnering companies, poster sessions and much more.

It’s the perfect place to keep you updated about Amyloidosis.

See you online on September 6-8.

Julian Gillmore

3rd European ATTR Amyloidosis meeting for Patients and Doctors I 3

Monday, 6 September 2021

Patients association Channel Doctors Channel

2:00 - 2:45 pmSponsor Symposium - PFIZER -

Addressing the multifaceted needs of your patients with ATTR amyloidosis

3:00 - 7:00 pm Benefit of having a positive lifestyle Pathogenesis and Diagnosis

Tuesday, 7 September 2021

Patients association Channel Doctors Channel

1:30 - 2:00 pmSummary of Day 1

Doctors presentation dedicated to patients

Poster Session

2:00 - 2:45 pmPharmaceutical companies

presentationsSponsor Symposium - ALNYLAM - Exploring

Patient-Centred Management of hATTR Amyloidosis

2:45 - 4:15 pm Disease-modifying treatment Disease-modifying treatment

4:15 - 5:05 pm Joint Session Roud Table - Part 1 & Part 2

5:05 - 5:50 pmSponsor Symposium - BRIDGEBIO/EIDOS

Role of the 6-minute walk test in ATTR amyloid cardiomyopathy

5:50 - 6:05 pm Innovative and Start up session - INTELLIA

Wednesday, 8 September 2021

Patients association Channel Doctors Channel

1:30 - 2:00 pm Poster Session

2:00 - 2:45 pm Pharmaceutical companies presentations

Sponsor Symposium - IONIS - ATTR Cardiac amyloidosis (ATTR-CM) -

The CARDIO-TTRansform Program

2:45 - 5:05 pm Symptomatic Management

5:05 - 6:00 pm Interactive joint Session - Part 1 & Part 2

6:00 - 6:30 pm Wrap up message and conclusion

3rd European ATTR Amyloidosis meeting for Patients and Doctors

Sponsored sessions Joint Session

PRELIMINARY PROGRAM

3rd European ATTR Amyloidosis meeting for Patients and Doctors I 4

PATIENTS ASSOCIATION CHANNEL

Monday, 6 September 2021

3:00 - 7:00 pm Benefit of having a positive lifestyle

Chairs: Jean-Christophe FIDALGO, France and David GREGORY , , United Kingdom

3:00 - 3:30 pm 3:30 - 3:50 pm 3:50 - 4:10 pm4:10 - 4:30 pm

4:30 - 4:35 pm 4:35 - 4:55 pm 4:55 - 5:15 pm 5:15 - 5:30 pm 5:30 - 6:00 pm

Holistic approach Laura OBICI, ItalyPhysical Activities Alan LEVI, United KingdomNutrition Tamer REZK, United KingdomCommunication with the family Dorien MÜLLER, The Netherlands and Paul VAN DER ZWAAG, The Netherlands Song of the caregiver Koenraad VERHAGEN, The NetherlandsMeditation / mindfulness Theodora WEISZETP - presentation pilote Origamy in France Agnes FARRUGIA, FranceRole and place of the psychologis Elodie CAMOIN, FranceQuestions & Discussion

Tuesday, 7 September 2021

1:30 - 2:00 pm Summary of Day 1 - Doctor’s presentation dedicated to patients Chairs: David ADAMS, France and Pablo GARCIA-PAVIA, Spain

2:00 - 2:45 pm Pharmaceutical companies presentations

2:00 - 2:15 pm IONIS presentation - Currently Enrolling Trial in Cardiac Amyloidosis: Details on the CARDIO-TTRansform Trial Gustavo BUCHELE, USA

PFIZER presentation

BRIDGEBIO/EIDOS presentation - Eidos clinical program update Jocelyn M. ASHFORD, USA

2:45 - 4:15 pm Disease-modifying treatment

Chairs: Jean-Christophe FIDALGO, France and Marco LUIGETTI, Italy

2:45 - 3:00 pm 3:00 - 3:15 pm 3:15 - 3:30 pm 3:30 - 3:45 pm 3:45 - 4:00 pm 4:00 - 4:15 pm

Trial Design and Endpoints in ATTR amyloidosis Mori GERTZ, USATTR Stabilisers Pablo GARCIA-PAVIA, SpainGene silencers in ATTR amyloidosis David ADAMS, FranceGene editing for ATTR amyloidosis Julian GILLMORE, United Kingdom Combinations Treatment in ATTR amyloidosis Mathew MAURER, USAThe future & Central neurological treatment possibilities Luísa SOUSA, Portugal

4:15 - 4:40 pm

4:40 - 5:05 pm

Joint Session Round Table: When to start, when to stop, when to switch - Part 1

Chairs: Jean-Christophe FIDALGO, France and Laura OBICI, Italy

Speakers: David ADAMS, France; Pablo GARCIA-PAVIA, Spain and Mathew MAURER, USA

Joint Session Round Table: When to start, when to stop, when to switch - Part 2

Chairs: Hazenberg BOUKE, The Netherlands and Marco LUIGETTI, Italy

Speakers: Mori GERTZ, USA; Julian GILLMORE, United Kingdom and Luisa SOUSA, Portugal

2:30 - 2:45 pm

2:15 - 2:30 pm

3rd European ATTR Amyloidosis meeting for Patients and Doctors I 5

PATIENTS ASSOCIATION CHANNEL

Wednesday, 8 September 2021

1:30 - 2:00 pm Poster Session

Chairs: Ute HEGENBART, Germany and Hartmut SCHMIDT, Germany

2:00 - 2:45 pm Pharmaceutical companies presentations

2:00 - 2:15 pm

2:15 - 2:30 pm

SOBI presentation - Shared-decision making for improved patient outcomes, perspectives of a patient and his doctor Carol WHELAN, United Kingdom and David GREGORY, United Kingdom

ALNYLAM presentation - You, me and the elephant in the room - Overcoming barriers to family dialogue in hATTR amyloidosis

- Welcome and Introduction Paul VAN DER ZWAAG, The Netherlands- Understanding the Family Dialogue Tool - Purpose and Objectives RosalineCALLAGHAN- The Family Dialogue Tool in Action Rosaline CALLAGHAN and PhilipVERHAGEN, The Netherlands- Q&A and Close Rosaline CALLAGHAN, Philip VERHAGEN, The Netherlands andPaul VAN DER ZWAAG, The Netherlands

2:45 - 5:05 pm Symptomatic ManagementChair Giovanni ds: ’ALESSIO, Italy and Pablo GARCIA-PAVIA, Spain

2:45 - 3:05 pm Heart failure Thibaud DAMY, France3:05 - 3:25 pm Other cardiac manifestations Esther GONZÁLEZ-LÓPEZ, Spain3:25 - 3:45 pm GI manifestations of autonomic neuropathy Jonas WIXNER, Sweden3:45 - 4:05 pm Peripheral neuropathic manifestations Alex ROSSOR, United Kingdom4:05 - 4:25 pm Central neurological manifestations Yoshiki SEKIJIMA, Japan4:25 - 4:45 pm Synovial manifestations Mazen HANNA, USA 4:45 - 5:05 pm Treatment of eye complications Antoine ROUSSEAU, France

5:05 - 5:30 pm

5:30 - 6:00 pm

6:00 - 6:30 pm

2:30 - 2:45 pm ALNYLAM presentation - Reach for the STAR - Empowering patients through symptom tracking

- Welcome and Introduction Catilena BIBILONI- STAR Consultation Carol WHELAN, United Kingdom and David GREGORY,United Kingdom- Q&A and Close Catilena BIBILONI, David GREGORY, United Kingdom and CarolWHELAN, United Kingdom

Interactive combined Session - Part 1

Chairs: Giovanni d'ALESSIO, Italy and Pablo GARCIA-PAVIA, Spain

Speakers: Antoine ROUSSEAU, France; Yoshiki SEKIJIMA, Japan and Jonas WIXNER, Sweden

Interactive combined Session - Part 2

Chair : Pablo GARCIA-PAVIA, SpainSpeakers: Thibaud DAMY, France; Esther GONZALEZ-LOPEZ, Spain; Mazen HANNA, USA and Alex ROSSOR, United Kingdom

Wrap up message and Conclusion

Chair s: David ADAMS, France and Julian GILLMORE, United Kingdom

3rd European ATTR Amyloidosis meeting for Patients and Doctors I 6

DOCTORS CHANNEL

Monday, 6 September 2021

2:00 - 2:45 pmSponsor Symposium - PFIZERAddressing the multifaceted needs of your patients with ATTR amyloidosis

Chair Fabian KNEBEL: , Germany

2:00 - 2:03 pm Welcome and Introduction2:03 - 2:08 pm ATTR amyloidosis as a systemic disease Lucía GALÁN, Spain2:08 - 2:18 pm Treating ATTR amyloidosis - what, why, how and when? Fabian KNEBEL, Germany2:18 - 2:38 pm Treating the ATTR amyloidosis patient - a patient-focused approach

to disease management - Case study 1: hATTR-PN patient with subsequent cardiac involvementLucía GALÁN, Spain- Case study 2: wtATTR-CM patient Esther GONZÁLEZ-LÓPEZ, Spain

2:38 - 2:43 pm Questions & Discussion2:43 - 2:45 pm Summary and close Fabian KNEBEL, Germany

3:00 - 7:00 pm Pathogenesis and Diagnosis

Chairs: Giampaolo MERLINI, Italy and Philip HAWKINS, United Kingdom

3:00 - 3:20 pm

3:20 - 3:40 pm

3:40 - 4:00 pm

4:00 - 4:20 pm

4:20 - 4:40 pm

4:40 - 5:00 pm

5:00 - 5:20 pm

5:20 - 5:40 pm

5:40 - 6:00 pm

6:00 - 6:20 pm 6:20 - 7:00 pm

Pathogenesis and mechanism of ATTR amyloidosis Vittorio BELLOTTI, United Kingdom

Organ involvement and Clinical Phenotype in ATTR amyloidosis Bouke HAZENBERG, The Netherlands

Genetic and epigenetic factors and susceptibility to ATTR amyloidosis Carolina LEMOS, Portugal

Red Flags for early diagnosis of wild-type ATTR amyloidosis Claudio RAPEZZI, Italy

Red Flags for early diagnosis of hereditary ATTR amyloidosis Marcia WADDINGTON-CRUZ, Brazil

Establish the diagnosis of ATTR amyloidosis Isabel CONCEICAO, Portugal

Counselling of family members & cascade testing in ATTR amyloidosis Catarina CAMPOS, Portugal

Diagnosis & monitoring of TTR mutation carriers Lucía GALÁN, Spain

Developement of Amyloidosis Networks Katrin HAHN, Germany

Cardiac Imaging in ATTR amyloidosis Marianna FONTANA, United Kingdom Questions & Discussion

Tuesday, 7 September 2021

1:30 - 2:00 pm Poster Session

Chair Vios: laine PLANTE BORDENEUVE, France and Julian GILLMORE, United Kingdom

2:00 - 2:45 pm Sponsor Symposium - ALNYLAM - Exploring Patient-Centred Management of hATTR Amyloidosis

2:45 - 4:15 pm Disease-modifying treatment

Chairs: Bouke HAZENBERG, The Netherlands and Laura OBICI, Italy

2:45 - 3:00 pm

3:00 - 3:15 pm

3:15 - 3:30 pm

3:30 - 3:45 pm

3:45 - 4:00 pm

4:00 - 4:15 pm

Gene silencers in ATTR amyloidosis David ADAMS, France

Gene editing for ATTR amyloidosis Julian GILLMORE, United Kingdom

Trial Design and Endpoints in ATTR amyloidosis Mori GERTZ, USA

TTR Stabilisers Pablo GARCIA-PAVIA, Spain

The future & Central neurological treatment possibilities Luísa SOUSA, Portugal

Combinations Treatment in ATTR amyloidosis Mathew MAURER, USA

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DOCTORS CHANNEL

4:15 - 4:40 pm

4:40 - 5:05 pm

Joint Session Round Table: When to start, when to stop, when to switch - Part 1

Speaker David ADs: AMS, France; Pablo GARCIA-PAVIA, Spain and Mathew MAURER, USA

Joint Session Round Table: When to start, when to stop, when to switch - Part 2

Speaker Mori GERs: TZ, USA; Julian GILLMORE, United Kingdom and Luísa SOUSA, Portugal

5:05 - 5:50 pm Sponsor Symposium - BRIDGEBIO/EIDOS

Chair Julian GILLMORE: , United Kingdom

5:50 - 6:05 pm Innovative and Start up session - INTELLIA

Chair Jeffr: ey CEHELSKY, USA

Wednesday, 8 September 2021

1:30 - 2:00 pm Poster Session

2:00 - 2:45 pmSponsor Symposium - IONIS ATTR Cardiac amyloidosis (ATTR-CM) - The CARDIO-TTRansform Program

Chair: Sam TSIMIKAS, USA

2:00 - 2:05 pm 2:05 - 2:15 pm 2:15 - 2:25 pm 2:25 - 2:35 pm 2:35 - 2:45 pm

IntroductionIonis ASO Platform and LICA Technology Sam TSIMIKAS, USAThe CARDIO-TTRansform Program Marianna FONTANA, United Kingdom Future landscape for ATTR-CM treatment Questions & Answers session with panelists

2:45 - 5:05 pm Symptomatic Management

Chairs: Giovanni d’ALESSIO, Italy and Pablo GARCIA-PAVIA, Spain

2:45 - 3:05 pm Heart failure Thibaud DAMY, France

3:05 - 3:25 pm Other cardiac manifestations Esther GONZÁLEZ-LÓPEZ, Spain

3:25 - 3:45 pm GI manifestations of autonomic neuropathy Jonas WIXNER, Sweden

3:45 - 4:05 pm Peripheral neuropathic manifestations Alex ROSSOR, United Kingdom

4:05 - 4:25 pm Central neurological manifestations Yoshiki SEKIJIMA, Japan

4:25 - 4:45 pm Synovial manifestations Mazen HANNA, USA

4:45 - 5:05 pm Treatment of eye complications Antoine ROUSSEAU, France

5:05 - 5:30 pm

5:30 - 6:00 pm

6:00 - 6:30 pm

Interactive combined Session - Part 1Chair Giovanni ds: ’ALESSIO, Italy and Pablo GARCIA-PAVIA, Spain

Speaker Antoine ROUSSEAs: U, France; Yoshiki SEKIJIMA, Japan and Jonas WIXNER, Sweden

Interactive combined Session - Part 2Chair Pablo G: ARCIA-PAVIA, Spain Speakers: Thibaud DAMY, France; Esther GONZÁLEZ-LÓPEZ, Spain; Mazen HANNA, USA and Alex ROSSOR, United Kingdom

Wrap up message and Conclusion

Chair David ADs: AMS, France and Julian GILLMORE, United Kingdom

5:05 - 5:35 pm

5:35 - 5:50 pm

Role of the 6-minute walk test in ATTR amyloid cardiomyopathy Julian GILLMORE, United KingdomQ&A

5:50 - 6:05 pm Safety considerations and methodology for genome editing Michael MAITLAND, USA