24th ESGLD Workshop and Graduate Course
7th to 11th September 2022
SAVE THE DATE! As you may know it is the turn of the UK to hold the Bi-Annual ESGLD Workshop and Graduate Course. ESGLD Chair, Professor Brian Bigger and his University of Manchester Team are happy to announce that a date has been set for this event which will take place in Lancaster, North West of England. Please hold the 7th to 11th of September 2022 in your diaries!

24th ESGLD Workshop and Graduate Course
Full Scientific Programme
Please note ALL times are UK time (BST)
Day 1 Thursday 8th September
14:00 – 18:00 Registration – Management School Atrium
All sessions will take place in Management School Lecture Theatre 18
18:00 – 18:15 Opening Ceremony – Welcome Address
18:15 – 18:30 Flash talk awards
18:30 – 19:30 KEYNOTE SPEAKER 1 (45 minutes talk + 15 minutes questions)
Overview of Gene Therapy for Lysosomal Diseases
Professor Simon Jones, Manchester University Hospitals NHS Foundation Trust, UK
Chairs: Volkmar Gieselmann, Nicole Muschol
19:30 – 20:00 BREAK
20:00 onwards GET TOGETHER and DINNER – Barkers
Day 2 Friday 9th September – Lysosome Function
07:00 – 08:15 Breakfast – Marketplace or Hotel
ALL sessions will take place in Management School Lecture Theatre 18
Chairs: Markus Damme, Bruno Gasnier
08:30 – 09:30 KEYNOTE SPEAKER 2 (45 minutes talk + 15 minutes questions)
LYSET – A Key Protein in Lysosome Biogenesis
Professor Sabrina Jabs, University of Kiel, Germany
09:30 – 09:50 O01 Multiple lysosomal proteases knock-out models reveal new insights into their role in lysosomal proteolysis, autophagy, and mTORC1 activity (F. Bleibaum)
09:50 – 10:10 O02 The lysosomal enzyme trafficking factor LYSET enables nutrient generation from extracellular proteins (C. Pechincha)
10:10 – 10:30 O03 Phosphorylation-independent cytoplasmic retention of acetylated TFEB (A. Laudicina)
10:30 – 11:00 Coffee Break and Refreshments
11:00 – 11:20 O04 Role of the Cl-/H+ exchanger ClC-7 in maintaining lysosomal morphology and function (S. Bose)
11:20 – 11:40 O05 Crosstalk of class 3 PI3K signaling and lysosomal membrane damage response in LSDs (E. Le Guillou)
11:40 – 12:00 O06 Direct control of lysosomal catabolic activity by mTORC1 through regulation of V- ATPase assembly (E. Ratto)
12:00 – 13:00 ESGLD General Assembly (For ESGLD members only – Management School Lecture Theatre 18
13:00 – 14:00 Lunch Break – Management School Atrium
(13:00 – 14:00 ESGLD Board Meeting – lunch in a separate room) – Management School Ground Floor Meeting Room
14:15 – 18:00 Networking – (be at meeting point at 14:15, coaches depart at 14:30)
White Scar Cave coach will depart at 14:00

Scientific Programme Continues…
Management School Lecture Theatre 18
Chairs: Thomas Braulke, Mia Horowitz
18:30 – 19:30 KEYNOTE SPEAKER 3 (45 minutes talk + 15 minutes questions)
The interplay of autophagic processes and lysosomal homeostasis
Dr Oliver Florey, Babraham Institute, UK
20:00 onwards DINNER – Barkers
Day 3 Saturday 10th September – Therapies
07:00 – 08:15 Breakfast – Marketplace or Hotel
ALL sessions will take place in Management School Lecture Theatre 18
Chairs: Sandra Alves, Shaun Wood
Therapies – (15 minutes talks + 5 minutes questions)
08:30 – 08:50 O07 Improved enzyme replacement therapy of metachromatic leukodystrophy in mice using a superstable mutant of human arylsulfatase A (V. Gieselmann)
08:50 – 09:10 O08 A cell-based therapy for Pompe disease: transplantation of ex-vivo expanded muscle regenerative cells improves muscle function and lysosomal pathology (G. Schaaf)
09:10 – 09:30 O09 Development and validation of a GMP stem cell gene therapy manufacturing process for Mucopolysaccharidosis II (MPSII) (S. Ellison)
09:30 – 09:50 O10 A novel Adeno-associated viral gene therapy treatment protocol in sheep for use in MPSIIIC and other neurological disorders (B. Bigger)
09:50 – 10:10 O01 Fluoxetine treatment ameliorates the systemic pathology in a novel mouse model of Multiple Sulfatase Deficiency (MSD) (N.C. Sorrentino)
10:10 – 10:40 Coffee Break and Refreshments – Management School Atrium
10:40 – 11:00 O12 Brain targeting of GAA Promotes Full Correction of Murine Pompe Disease at a Clinically Relevant Dosage of Lentiviral Gene Therapy (F. Catalano)
11:00 – 11:20 O13 AAV-mediated homology-independent targeted integration leads to supraphysiological levels of lysosomal enzyme secretion from liver and correction of MPS VI in mice (F. Dell’Aquila)
11:20 – 11:40 O14 Ambroxol as a treatment for neurological manifestations in GD 2 (C. Aries)
11:40 – 12:00 O27 Endothelial dysfunction in Fabry disease in primary Endothelial Colony Forming Cells (S.Tokic)
12:00 – 13:30 Lunch Break – Management School Atrium
SESSION 2 (continued) Therapies – Management School Lecture Theatre 18
Chairs: Volkmar Gieselmann, Nicole Muschol
13:30 – 14:30 KEYNOTE CONFERENCE 4 (45 minutes talk + 15 minutes questions)
Transporting biotherapeutics across the blood-brain-barrier
A case example: Brain penetrant enzyme replacement therapy for MPSII
Dr. Annie Arguello
14:30 – 14:50 O16 Developing an iPSC-based neural gene therapy for Sanfilippo syndrome (O. Mandolfo)
14:50 – 15:10 O17 Drug repurposing approach in Mucopolysaccharidosis type IIIA: the case of Fluoxetine (A. Capuozzo)
15:10 – 15:40 Coffee Break and Refreshments – Management School Atrium
15:40 – 16:00 O18 Improving IDS.fusion peptides for BBB-crossing in Mucopolysaccharidosis type II (R. Searle)
16:00 – 16:20 O19 Fusion of RVG and gh625 peptide sequences to Iduronate-2-Sulfatase for the Treatment of Mucopolysaccharidosis Type II (S.Wood)
16:20 – 16:40 O20 An attenuated form of MPS plus due to deficiency of VPS33A and impaired HOPS complex (E. Pavlova)
16:40 – 17:00 O21 A precision medicine tool for high utilization and quality of individual treatment trials with immunomodulatory drugs in Mucopolysaccharidoses (A. M. Wiesinger)
17:00 – 19:00 Attended Poster Session and Refreshments – Management School Atrium
20:00 GALA Dinner – LICA Hall – Private Dining Room

Day 4 Sunday 11th September – Lysosomal Pathology
07:00 – 08:30 Breakfast – Marketplace or Hotel
ALL sessions will take place in Management School Lecture Theatre 18
Chairs: Stuart Ellison, Francisca Coutinho
Lysosomal Pathology – (15 minutes talks + 5 minutes questions)
08:50 – 09:10 O22 Secondary deficiency of neuraminidase 1 and its contribution to the CNS pathophysiology in neurological mucopolysaccharidoses (A. Pshezhetsky)
09:10 – 09:30 O23 Investigating the role of inflammation in MPS IIIA neuropathology (O. Mandolfo)
09:30 – 09:50 O24Mice lacking TRPML1 present kidney disease (G. Grieco)
09:50 – 10:20 Coffee Break and Refreshments – Management School Atrium
10:20 – 10:40 O25 Using Schizosaccharomyces pombe to understand the pathogenesis of CLN3 disease (J. Clemente-Ramos)
10:40 – 11:00 O26 Haplotypic and MicroRNA Profiling in Niemann-Pick type C Portuguese Patients (M. Encarnação)
11:00 – 11:20 O15 Brain-targeted lentiviral haematopoietic stem cell gene therapy for severe Mucopolysaccharidosis Type I: peptide tag optimisation for more efficient delivery across the blood-brain-barrier. (T. Andreou)
11:20 – 11:30 (Scientific committee to discuss prizes)
11:30 – 11:45 Prizes
11:45 – 12:00 Closing Remarks
12:00 onwards Lunch Box and Departure – Management School Atrium
12:30 onwards Coach Transfer to Manchester Airport /Lancaster Railway Station.
The coach to Manchester Airport will depart at 12:30 from Meeting Point (ETA 14:00) The shuttle bus to Lancaster Railway Station will depart at 12:30 from Meeting Point
*Alcoholic Beverages and networking activities are funded outside of the scientific programme

SPONSORS
The European Study Group on Lysosomal Diseases would like to thank all our sponsors for their support of the 24th ESGLD Workshop and Graduate Course.
PLATINUM SPONSORS


ESGLD 2022
For further information regarding 24th ESGLD Workshop and Graduate Course, 2022 Lancaster please contact:
Dr Tereza Andreou
School of Biological Sciences
Faculty of Biology, Medicine and Health
3.721 Stopford Building
The University of Manchester
tereza.andreou@manchester.ac.uk