Pitch & Partner

 

Pitch and Partner, Wednesday 24 April 2024

Panel discussion
John Sholar
Pitch and Partner
12:25

A first-in-class revolutionary platform: jCell® Retinal Progenitor Cells (RPC) restoring vision in patients with blinding diseases

John Sholar, Chief Executive Officer, jCyte Inc
Carole Cramer
Pitch and Partner
12:35

Innovative biologics delivery for rare disease

Oskar Smrzka
Pitch and Partner
12:45

Adaptable decoys for the quick and selective removal of anti-drug and anti-vector antibodies from circulation

Otto Prohaska
Pitch and Partner
12:55

TransCytos' Non-Viral Ex-Vivo Slipstream™ Technology – a Transformation of Cell and Gene Therapy Delivery

XENIA PROTON DE LA CHAPELLE
Pitch and Partner
13:05

From patient organization to biotech

Shawn Ritchie
Pitch and Partner
13:15

Metabolic replacement therapy for the treatment of rhizomelic chondrodysplasia punctata

Sulagna Bhattacharya
Pitch and Partner
15:00

Revolutionizing vision restoration: Mutation-independent optogenetic therapy for inherited retinal diseases

Katheron Intson
Pitch and Partner
15:10

How to prepare for the genomic gold rush in pharma

Manohar Katakam
Pitch and Partner
15:20

ST-002: A promising therapy for the metabolic effects of hypercortisolemia and Cushing syndrome

Michael Weickert
Pitch and Partner
15:30

Zelenirstat: Safety and efficacy - ready to move to clinical investigation for Acute Myeloid Leukemia (AML)

Pawel Fludzinski
Pitch and Partner
15:40

Long acting glucagon analog for the treatment of CHI (Congenital Hyper Insulinism)

Frank Sorgi
Pitch and Partner
15:50

FLAG-003 for the Treatment of DIPG (Diffuse Intrinsic Pontine Glioma) in Children

Shane Hegarty
Pitch and Partner
16:00

First-in-class KCC2 potentiators as novel orphan drugs to treat CNS disinhibition and drug-resistance in rare neurological disorders

Sebastian Franzinger
Pitch and Partner
16:10

NPC, Galactosemia and Barth Syndrome: Developing modifier therapies for neurological and metabolic disorders

Joshua Card-Gowers
Pitch and Partner
16:20

Ensuring reliability in quantifying patient populations for orphan drugs: challenges and solutions

Colin Cook
Pitch and Partner
16:30

XDemics: Making tomorrow’s cell and gene therapies

Colin Cook, Technical Founder, XDemics Corporation
last published: 20/Apr/24 16:35

Pitch and Partner, Thursday 25 April 2024

Panel discussion
Pitch and Partner
12:00

Industry perspectives: Partnering in the rare disease space

Kathie Bishop, SVP, Head of Rare Disease and External Innovation, Acadia Pharmaceuticals Inc.
Shira Landskroner-Eiger, Director, External Innovation Rare Disease, Ipsen
Sean Ekins
Pitch and Partner
12:40

Development of an enzyme replacement therapy for batten disease CLN1 and other neurodegenerative diseases

Albert Wang
Pitch and Partner
13:00

Supercharging rare patient groups with technology

Sponsored by Comend

Donna Sullivan
Pitch and Partner
15:35

A candid conversation: What rare really needs

James Mongiardo
Pitch and Partner
15:55

A-Synaptic’s patent protected transdermal replacement for billion dollar oral childhood epilepsy treatment

last published: 20/Apr/24 16:35

 

Get Involved At World Orphan Drug Congress USA

 

To Sponsor Or Exhibit

 
 

Justin Franks
justin.franks@terrapinn.com
t/ +1 914 819 3506

 

 

 

To Speak

 
 

Gina McHugh
gina.mchugh@terrapinn.com
t/ +1 646 619 1775

 

 

 

Marketing & Press Opportunities

 
 

Kaylie Brogan
kaylie.brogan@terrapinn.com