The Whole Molecule, page-6

Sarepta has 11 drug molecules in their pipeline but its carrier is nowhere near as good as PYC's CPP. The recent FDA rejection of their Vyondys 53 is a casing point.

Think about a molecule owner as a goods manufacturer, it relies on efficient carrier like DHL or FedEx to deliver the goods to the end user. Who is making more money? Of course the carrier because it could deliver goods from many different manufacturers.

There are many many biotech companies in the race, some are using adeno-associated virus (AAV), and many have been researching for over 10 years. Most likely an effective carrier is the holy grail that they are looking for. Here is a list of the major ones:

• Spark Therapeutics - haemophilia, haemophilia B, Pompe disease, Batten disease and choroideremia
• Alnylam - haemophilia, hypercholesterolemia, rare liver disease and hypertension
• ArrowHead - Hepatitis B
• SQZ Biotechnologies - autoimmune diseases like type 1 diabetes
• AveXis - spinal muscular atrophy (SMA), Rett Syndrome and inherited forms of amyotrophic lateral sclerosis (ALS)
• Askepios BioPharmaceutical (AskBio) - untreatable genetic muscular and neurological diseases
• Cellectis - cancers including leukaemia and lymphoma
• Pluristem - inflammation, heart disease, muscle injuries, blood disorders and radiation damage
• RegenexBio - retina, neurodegenerative and metabolic diseases
• Bluerock Therapeutics - neuology, cardiology, immunology, hard to control degenerative disease such as Parkinson’s disease

PYC is in the box seat with a carrier which could deliver molecules with high efficiency and low toxicity.