As you may have read earlier, PYC, a pharmaceutical company from Australia, has been given permission to start a clinical trial with a potential treatment for people with ADOA. The PYC study consists of two studies: a clinical trial testing treatment PYC-001 (SUNDEW) and a study of the natural history of ADOA (BANKSIA).
To get new medicines on the market and have them reimbursed by health insurance, clinical research is needed. This research can only succeed if there are enough participants. At the request of PYC, we share their invitation to participate in their SUNDEW and BANKSIA studies with you.
SUNDEW: Clinical Study of PYC-001
SUNDEW is investigating a potential treatment for OPA1-associated ADOA: PYC-001. This drug is the first to target the cause of the genetic mutation. The treatment involves administering a single, ascending dose of the drug directly into the eye, to see how safe it is and how well people tolerate it.
Treatment PYC-001
In the classic form of ADOA, in short, a deficiency of OPA1 leads to damage and in some cases death of mitochondria in the retinal ganglion cells. Without properly functioning mitochondria, the retinal ganglion cells slowly die. This causes the optic nerves to become thinner, which (often slowly) leads to loss of vision.
PYC-001 is an RNA therapy in which a single, ascending dose of the drug is injected directly into the eye. This drug is intended to increase the amount of OPA1 protein that is produced, in order to achieve the correct balance of the OPA-1 molecule. The difference between RNA and DNA therapy is that DNA therapy is permanent. And the effect of RNA therapy is reversible when you stop the therapy.
If the PYC-001 treatment proves to be safe and tolerable during this study, follow-up research can take place. If the entire study goes well, the first treatment for ADOA could be available to everyone who qualifies for it in 2029.
Locations
The SUNDEW study will initially be conducted exclusively in Australia and Tasmania, and possibly later in the US. Initially, PYC is looking for participants who are already there. If you live in Australia or Tasmania, please send an email to sundew@pyctx.com to sign up.
BANKSIA: Natural History Study
In addition to the SUNDEW study, PYC is conducting research into the progression of ADOA. This two-year study is investigating two processes that play an important role in ADOA at the molecular level. By investigating this thoroughly, PYC will soon be able to use these processes to determine whether PYC-001 has an effect on the progression of ADOA.
Locations
BANKSIA takes place in various locations in the US, Australia and Europe, including Amsterdam:
- Sydney Eye Hospital, Sydney (Australia)
- Bascom Palmer Eye Institute University of Miami, Miami (Florida, USA)
- University of Washington, Seattle (Washington, USA)
- Rennes Hospital, Rennes (France)
- Amsterdam University Medical Centers, Amsterdam (Netherlands)
- Hannover Medical School, Hannover (Germany)
- Medical University of Graz, Graz (Austria)
Send an email to banksia@pyctx.com to register for this study.
About PYC
PYC is a pharmaceutical company. Their goal is to develop life-changing medicines for people who need them most: people with diseases for which there is no treatment. The ADOA medication is the second RNA therapy PYC is working on. The first therapy is for retinitis pigmentosa type 11. Everything the researchers have learned from the retinitis pigmentosa program can be applied to the ADOA program.
Sign in
Do you meet the criteria for the SUNDEW and BANKSIA examinations, which you can find on their detailed pages? Then register directly via the links below. Travel and accommodation costs incurred by you and any supervisor will be reimbursed by PYC.
