Top 5 biotechnology companies treating (the loss of proteostasis) Ageing.

1. resTORbio RB101
2. Navitor
3. Longevity Biotech
4. Juvenescence
5. Altos’ lab

resTORbio RB101

The process is indeed related to the loss of proteostasis. Proteostasis refers to the regulation
of the proteins within a cell, which includes their synthesis, folding, and degradation2.

In the context of neurodegenerative diseases, disturbances in proteostasis can lead to the

accumulation of misfolded or aberrant proteins, which can contribute to neuronal dysfunction
and disease progression2. This is particularly important in neurons, which are long-lived cells
with limited regenerative capacity3.

resTORbio, a US-based biopharmaceutical firm, is exploring the use of RTB101 for the
treatment of Parkinson’s disease. Here are the key details:

1. RTB101 (Mechanism of Action):

o RTB101 is an oral small molecule inhibitor that targets a protein complex
called TORC1 (target of rapamycin complex 1).
o It binds to the active site of mTOR (mammalian target of rapamycin) within
the TORC1 complex.
2. Clinical Trial:
o resTORbio has initiated a Phase Ib/IIa clinical trial to evaluate RTB101.
o The trial involves approximately 45 participants with mild Parkinson’s
disease who are already on standard-of-care therapy.
o Participants are randomized into five cohorts and administered:
 RTB101 alone (300mg dose).
 RTB101 in combination with sirolimus (2mg, 4mg, or 6mg doses).
3. Trial Endpoints:
o Primary Endpoint: Assessing the safety and tolerability of RTB101 alone
or in combination with sirolimus.
o Secondary Endpoints: Measuring exposure in blood, plasma, and
cerebrospinal fluid (CSF).
o Exploratory Endpoints: Investigating biomarkers in plasma and CSF, along
with various clinical assessments.
4. Expected Results:
o Data from the trial are anticipated to be available next year.
o The combination of RTB101 and sirolimus aims to trigger autophagy, which
may help eliminate protein aggregates in neurons and improve lysosomal
function.

In summary, resTORbio is exploring RTB101 as a potential therapeutic approach for

Parkinson’s disease by targeting TORC1 and promoting autophagy. Ongoing research will
provide insights into its safety and efficacy.
SIDE EFFECTS:

In clinical phase trials, RTB101 has been reported to cause respiratory problems in patients.

PRICING:

Because this drug is under clinical trials so, the pricing strategy is still unknown.

FUTURE PROSPECTS:

As for the prospects of RTB101, it's an oral mechanistic target of rapamycin (mTOR)
inhibitor that has been observed to increase IFN-induced antiviral gene expression and
decrease the incidence of respiratory tract infections (RTis) in older adults. However, the
clinical development of RTB101 in the prevention of clinically symptomatic respiratory
illness (CRI) was halted after its Phase III PROTECTOR 1 clinical trial failed to meet the
primary goal. Despite the current halt in production due to observed respiratory problems in
patients, the prospects of RTB101 seem promising. The company resTORbio intends to
develop RTB101, an orally administered, selective TORC1 inhibitor, for additional ageing-
related indications including heart failure or neurodegenerative diseases
REFERENCES
1. https://www.resTORbio.com/pipeline/
2. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8483240/
3. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7601929/
4. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9998692/
5. https://www.thelancet.com/journals/lanhl/article/PIIS2666-7568%2821%2900062-3/
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