RP001v63_Dosage, benefits & legal status
Also known as 21-amino acid peptide targeting expanded CAG repeat RNA, BIND peptide, P3L
A Hong Kong university spin-off's lead candidate, built on a published 21-residue peptide called BIND.
Common vial size is what vendors typically sell, not a recommendation. Enter your own vial in the calculator.
What is RP001v63?
Plain language first. The technical label stays, but it never stands alone.Overview
Almost every Huntington's drug in development goes after the faulty protein or the message that codes for it. This one treats the repeating RNA itself as the thing causing harm, and is built to clamp onto the shape that RNA folds into. The FDA granted it orphan drug designation in August 2026. There is no trial yet, and nothing published under the name RP001v63. The parent peptide does have a published record going back to 2018, including a sequence, a binding affinity that strengthens as the repeat gets longer, and rescue of eye degeneration and climbing ability in fruit flies.
RP001v63 is a 21-amino-acid peptide developed by Rare Power Limited, a Chinese University of Hong Kong spin-off, with the university's School of Life Sciences. It is designed to bind the folded structure of expanded CAG-repeat RNA, the transcript behind Huntington's disease, rather than the mutant huntingtin protein or its messenger RNA. No publication names RP001v63 itself, but the technology Rare Power licensed from the university is published: a 21-residue peptide called BIND, derived from the protein nucleolin, sequence AEIRLVSKDGKSKGIAYIEFK.
What it's used for
Nothing yet. It is a preclinical candidate for Huntington's disease. Orphan drug designation is a development incentive, not permission to sell or an approval.
How does RP001v63 work?
What it does in the body, and where.Huntington's disease comes from a stretch of DNA where the letters CAG repeat far too many times. The usual drug targets are the damaged protein that results, or the messenger RNA that carries the instructions for it. This peptide skips both.
- 01The repeat RNA as the problemA long run of CAG repeats makes an RNA that folds into a hairpin and traps proteins the cell needs elsewhere. The developers describe treating that RNA as a harmful agent in its own right rather than as a messenger.
- 02Shape recognition, not sequence readingRather than matching the RNA letter by letter, the peptide is described as recognizing the folded structure, with a flat sheet of its backbone sitting against the groove of the RNA helix. That is a different design approach from the antisense drugs tried in this disease.
- 03What has actually been shownFor RP001v63 itself: one statement, from the company's chief technology officer, that it was tested in neurons grown from patients and showed promising activity. No figures, no study design, no publication. For the parent peptide there is more: it blocked RNA-driven cell death at about 0.7 micromolar, bound more tightly as the repeat got longer, and in fruit flies carrying an expanded repeat it eased eye degeneration, improved climbing and extended lifespan.
A schematic of the compound's mechanism, not a diagnostic image.
What else is it used for?
Each group carries its own evidence level.Approved means regulators have cleared it for that purpose. Investigational means it is in trials. Early research means it is being studied and is not established.
What is verified
Investigational- RegulatoryThe FDA orphan designation exists and is dated August 3, 2026, to Rare Power Limited, for Huntington's disease. The record was read directly rather than taken from the announcement.
- RegulatoryThe FDA's own entry has no trade name and identifies the drug only by description: a 21-amino acid peptide targeting expanded CAG repeat RNA. The name RP001v63 comes from the university, not the regulator.
- No dataOrphan designation is granted on the basis of a rare disease and a plausible scientific rationale. It is not evidence that a drug works.
What is not
none- No dataNo peer-reviewed publication is cited for this candidate by name, and no trial is registered.
- No dataThe only experimental claim made for RP001v63 is a quoted statement about promising activity in patient-derived neurons, with no data behind it.
- No dataNo publication, patent or registry entry ties the name RP001v63 to a specific sequence. The v63 suffix suggests an engineered variant of the parent, and which variant is not public.
- No dataFirst-in-human trials are described as one to two years away, after manufacturing, safety and large-animal toxicology work that has not been done.
- No dataThe published work on the parent peptide stops at fruit flies and cells. Nothing in any species larger than a mouse has been reported for this family.
Orphan drug designation is not FDA approval and is not a finding that a drug works. It is an incentive granted for rare-disease programs, and the FDA's own record for this one says it is not approved for the orphan indication. Coverage of the announcement also treats the activity in patient-derived neurons as a result; it is a quote with no published data behind it. The designation was granted on August 3, 2026, almost two months before the September 23 announcement that made it news. Going the other way, this is not a program with no science behind it: the laboratory it came from published the parent peptide in 2018 and holds a granted US patent naming its sequence. What has not been published is which variant of that peptide RP001v63 is.
The published record
Papers and trials about RP001v63
4 papers tagged human · 3 not reviews or lab · 0 clinical trial publications
Furthest stage reached: No clinical trial on record
Counted from Europe PMC indexing, not read by a person; a human tag can land on reviews, commentary or lab papers; clinical trial publications is the narrower count.
At a glance
Four things to know about RP001v63
Evidence
Investigational
Category
Brain, mood and sleep
Common vial
Varies
Half-life
Nothing published
How much, and do you cycle it?
The range, and whether you take breaks.No human dose exists, not established. Not established No range
Nothing has been given to a person, so no schedule exists.
Nothing publishedNo figure
No pharmacokinetic data exist in any species. For a peptide meant to act inside brain cells, getting across the blood-brain barrier would be the first problem to solve, and nothing has been published about it.
You can compare this against the whole library to see where it sits.
What does it cost?
Typical dose and cost, in plain numbers.RP001v63 is not injected
It is taken not established in people. No human dose exists and no animal dose has been published. Any figure quoted for it would be invented.
How do you store it?
Before mixing, and after.Nothing to store: it is not sold anywhere.
RP001v63 has never been marketed, so there is no vial, pen or bottle to keep at home. Supplies for a trial are made, stored and dispensed by the sponsor and the trial pharmacy under conditions set in the protocol. Anything offered for sale under this name did not come from that supply chain.
What are the side effects and cautions?
Known cautions and warnings for this compound.Who should avoid it
- Any human use: there is no safety data, no dose and no product
- Anything sold under this name: the parent peptide's sequence is public, but nothing ties the designation RP001v63 to a specific variant of it, so a vial could not be checked against a reference
Stop and get help
- Not applicable. No product exists
Common effects
- Unknown for this candidate. The parent peptide was tested in cells and fruit flies, and this one in patient-derived neurons with no numbers published
Where this page says nothing is established, that means nobody has studied it, not that a compound is safe.
Is RP001v63 approved?
And where the numbers on this page come from.No. RP001v63 is not approved anywhere and is currently in company-run clinical trials. There is no legal route to obtain it outside a trial.
- Approval statusInvestigational
- RouteNot established in people
- Checked23 October 2026
- DoseNo dose has been established in people.
- Vial sizeFrom what vendors typically list; not a recommendation.
- SourcesFDA orphan drug designation record, 21-amino acid peptide targeting expanded CAG repeat RNAZhang et al., a peptidylic inhibitor for expanded CAG RNA-induced nucleolar stress, RNA, 2018US patent 11,639,371, Chinese University of Hong KongChinese University of Hong Kong announcement, September 23, 2026
What has happened recently
Not approved anywhere and not in human testing. The FDA's Office of Orphan Products Development lists a designation granted on August 3, 2026 to Rare Power Limited for the treatment of Huntington's disease, under the generic name "21-amino acid peptide targeting expanded CAG repeat RNA" with no trade name, and records it as not approved for the orphan indication. The Chinese University of Hong Kong announced the designation on September 23, 2026, 51 days later, and that announcement is where the name RP001v63 appears. Orphan designation can bring tax credits, fee waivers and up to seven years of market exclusivity after an approval, and it says nothing about whether a drug works. No trial is registered, there is no FDA substance record and no INN. The underlying technology is covered by US patent 11,639,371, granted to the Chinese University of Hong Kong on May 2, 2023 with a 2016 priority date, naming the 21-residue sequence as P3L.
You can compare this against the whole library, or read how to check a seller.
What else is like RP001v63?
Others in Brain, mood and sleep, side by side.If you're weighing RP001v63, it's worth reading Neurotensin, Semax, and Selank in the same class.
A Hong Kong university spin-off's lead candidate, built on a published 21-residue peptide called BIND.
- Dose
- No human dose exists
- How often
- Not established
One of the foundational neuropeptides. Decades of biology, no approved use.
- Dose
- No established dose
- How often
- Not applicable
A peptide used for sharper focus, memory, and mental clarity.
- Dose
- 0.2 up to 0.6 mg per day
- How often
- 1-2x daily
A peptide that eases anxiety while keeping you clear-headed.
- Dose
- 0.25 up to 0.5 mg per day
- How often
- 1-2x daily
Guides for this compound
- How to Reconstitute a Peptide
Mix peptide powder with bacteriostatic water, step by step.
- How to Read an Insulin Syringe
Read the units, convert to millilitres, and draw the exact dose.
- mcg vs mg: The Peptide Dosing Mistake to Avoid
Your vial says mg and your protocol says mcg. Here is the conversion and how to check it.
- How to Read a Peptide COA
What the purity number does and does not tell you, and how to spot an edited report.
- How to Vet a Peptide Vendor
Independent COAs, HPLC purity, and the red flags that mean walk away.
Sources
- FDA orphan drug designation record, 21-amino acid peptide targeting expanded CAG repeat RNA(2026)Designated August 3, 2026 to Rare Power Limited for the treatment of Huntington's disease. No trade name listed. Orphan designation status designated, and the record shows it is not FDA approved for the orphan indication.
- Zhang et al., a peptidylic inhibitor for expanded CAG RNA-induced nucleolar stress, RNA, 2018(2018)RNA 24(4):486-498. Describes BIND, a 21-amino-acid peptide, blocking expanded-CAG-RNA-driven cell death at about 0.7 micromolar. Binding strengthened with repeat length, from 17.28 micromolar for 22 repeats to 0.66 for 72. In fruit flies it eased retinal degeneration, improved climbing and extended lifespan. Corresponding authors Chan Ho-yin and Ngo Chi-Ki, the same laboratory behind the Rare Power candidate.
- US patent 11,639,371, Chinese University of Hong Kong(2023)Granted May 2, 2023 with a February 2016 priority date, inventors Chan, Ngo and Zhang. Names the 21-residue sequence AEIRLVSKDGKSKGIAYIEFK as SEQ ID NO:1, designated P3L, a nucleolin-derived peptide that binds CAG-repeat RNA with a measured affinity of 0.66 micromolar against a 72-repeat transcript. Covers Huntington's disease and other polyglutamine diseases.
- Chinese University of Hong Kong announcement, September 23, 2026(2026)Names the candidate RP001v63 and describes it as 21 amino acids binding the folded structure of expanded CAG-repeat RNA. Credits Rare Power Limited, a 2023 CUHK spin-off, with Professor Edwin Chan Ho-yin and Dr Maggie Leong Lok-i among the co-founders. Reports testing in patient-derived neurons with no numbers, and an aim to begin first-in-human trials within one to two years.
Beyond these, 5 records are indexed. Browse the records