Pitch & Partner
Pitch and Partner, Wednesday 24 April 2024
Pitch and Partner
11:25Venture capital panel: Charting the path for rare disease therapies innovation: Unveiling opportunities, trends, and challenges
Moderator: Ken Kengatharan, Managing General Partner, Atheneos Ventures
Devin Rosenthal, Vice President & Head of Due Diligence, NovaQuest Capital Management
Geeta Vemuri, Managing Partner, Agent Capital
Mary Schaheen, President, Prevail Partners Llc
Laura Tadvalkar, Managing Director, RA Capital Management
Pitch and Partner
12:25A first-in-class revolutionary platform: jCell® Retinal Progenitor Cells (RPC) restoring vision in patients with blinding diseases
John Sholar, Chief Executive Officer, jCyte Inc
Pitch and Partner
12:35Innovative biologics delivery for rare disease
Carole Cramer, President & Founder, BioStrategies
Pitch and Partner
12:45Adaptable decoys for the quick and selective removal of anti-drug and anti-vector antibodies from circulation
Oskar Smrzka, Chief Executive Officer/CSO, Ablevia
Pitch and Partner
12:55TransCytos' Non-Viral Ex-Vivo Slipstream™ Technology – a Transformation of Cell and Gene Therapy Delivery
Otto Prohaska, Founder and Chief Executive Officer, Transcytos
Pitch and Partner
13:05From patient organization to biotech
XENIA PROTON DE LA CHAPELLE, Chief Executive Officer and Patient Representative, AtmosR
Pitch and Partner
13:15Metabolic replacement therapy for the treatment of rhizomelic chondrodysplasia punctata
Shawn Ritchie, Chief Executive Officer, Med-Life Discoveries
Pitch and Partner
15:00Revolutionizing vision restoration: Mutation-independent optogenetic therapy for inherited retinal diseases
Sulagna Bhattacharya, Chief Executive Officer, Nanoscope Therapeutics
Pitch and Partner
15:10How to prepare for the genomic gold rush in pharma
Katheron Intson, Chief Executive Officer, Varient
Pitch and Partner
15:20ST-002: A promising therapy for the metabolic effects of hypercortisolemia and Cushing syndrome
Manohar Katakam, Chief Executive Officer, SteroTherapeutics
Pitch and Partner
15:30Zelenirstat: Safety and efficacy - ready to move to clinical investigation for Acute Myeloid Leukemia (AML)
Michael Weickert, Chief Executive Officer, Pacylex Pharmaceuticals
Pitch and Partner
15:40Long acting glucagon analog for the treatment of CHI (Congenital Hyper Insulinism)
Pawel Fludzinski, Chief Executive Officer, AmideBio
Pitch and Partner
15:50FLAG-003 for the Treatment of DIPG (Diffuse Intrinsic Pontine Glioma) in Children
Frank Sorgi, President and Chief Executive Officer, FLAG Therapeutics
Pitch and Partner
16:00First-in-class KCC2 potentiators as novel orphan drugs to treat CNS disinhibition and drug-resistance in rare neurological disorders
Shane Hegarty, Chief Scientific Officer & Co-Founder, AXONIS Therapeutics
Pitch and Partner
16:10NPC, Galactosemia and Barth Syndrome: Developing modifier therapies for neurological and metabolic disorders
Sebastian Franzinger, Head of Business Development, Scenic Biotech
Pitch and Partner
16:20Ensuring reliability in quantifying patient populations for orphan drugs: challenges and solutions
Joshua Card-Gowers, Senior Evidence Lead, HealthLumen
Pitch and Partner
16:30XDemics: Making tomorrow’s cell and gene therapies
Colin Cook, Technical Founder, XDemics Corporation
last published: 19/Apr/24 23:05
Pitch and Partner, Thursday 25 April 2024
Pitch and Partner
12:00Industry perspectives: Partnering in the rare disease space
Moderator: Maria Törnsén, President, North America, Calliditas Therapeutics
Sean Ekins, Chief Executive Officer, Collaborations Pharmaceuticals
Kathie Bishop, SVP, Head of Rare Disease and External Innovation, Acadia Pharmaceuticals Inc.
Jason Tardio, Chief Operating Officer, Ovid Therapeutics
Shira Landskroner-Eiger, Director, External Innovation Rare Disease, Ipsen
Pitch and Partner
12:40Development of an enzyme replacement therapy for batten disease CLN1 and other neurodegenerative diseases
Sean Ekins, Chief Executive Officer, Collaborations Pharmaceuticals
Pitch and Partner
12:50To be announced
Patrick Lu, President and Chief Executive Officer, Sirnaomics
Pitch and Partner
13:00To be announced
Sponsored by Comend
Pitch and Partner
13:20To be announced
James Fink, Director, Epilepsy Therapeutic Lead, Quiver Bioscience
Pitch and Partner
15:35A candid conversation: What rare really needs
Donna Sullivan, Director Of Patient Advocacy, Pathways To Trust
Pitch and Partner
15:55A-Synaptic’s patent protected transdermal replacement for billion dollar oral childhood epilepsy treatment
James Mongiardo, Chief Executive Officer, A-Synaptic Corp.
last published: 19/Apr/24 23:05
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