Alder Therapeutics – Our Therapies

Hope for those with Retinitis Pigmentosa

Retinitis Pigmentosa (RP) is a debilitating inherited blindness disease affecting 1 in 4,000 people. Current treatments are limited to one approved single-mutation gene therapy, with only a fraction of patients eligible.

ALD01 is our lead program, a retinal cell therapy that could revolutionize RP treatment, offering a mutation-agnostic therapeutic option.

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Promising preclinical data

‍ALD01 is supported by promising preclinical data across three relevant animal models:

ALD01 improved sight in a mouse RP model in a water maze task
Successful engraftment of ALD01 cells and synaptic connectivity in a rabbit RP model
Most non-human primate models show >40% mfERG improvement four weeks post-transplantation

Read the full story

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Benefits over other cell therapies

‍ALD01 outcompetes many other cell therapy options on therapeutic dose requirements as well as the simplicity and scalability of its manufacturing process:

Proprietary, regulatory friendly process
Shorter manufacturing timeline
Low therapeutic doses
No purification step required
Identical product identity across several different PSC lines

Potential impact

Based on preclinical data, our research suggests that ALD01 could profoundly impact RP patients’ lives:

Halted degenerative vision loss with possible improved vision
Mutation agnostic option for the vast majority of RP patients

We aim to conduct first-in-human trials for ALD01 in 2026

Find out how you can help us get there

Tackling heart failure

Heart failure is a major global health burden and is the most common cause of death worldwide. Treatment options are donor transplantation, other forms of surgical intervention, or lifelong symptomatic pharmacological treatment, which costs the US alone $20 billion per year.

ALD02 will be our second program, a cardiac cell therapy that aims to regenerate functional heart tissue.

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Promising preclinical data

Preclinical data shows that ALD02 improves heart function in pigs with induced heart disease — without fatal arrythmia:

1–3 fold thickening of the ventricular wall
5-fold improvement in wall motion
>20% increase in left ventricular ejection fraction

Read the full story

Benefits over other cell therapies

As well as being supported by promising, high-quality preclinical data, ALD02 offers other benefits relative to other cell therapies:

Xeno-free, shorter, and regulatory friendly manufacturing process
Low cell dose
No purification step required
Engraftment of ALD02 cells into the host tissue
No fatal arrhythmia in animal models

Potential impact

Our research to date suggests that ALD02 could have a transformative impact on patients suffering with heart failure:

Halted further worsening with possible improved heart function
Improved quality of life

We aim to conduct further proof-of-concept studies before planning first-in-human trials.

Find out how you can help us get there

Hope for those with Retinitis Pigmentosa

Retinitis Pigmentosa (RP) is a debilitating inherited blindness disease affecting 1 in 3,000 people. Treatments are limited to one approved single-mutation gene therapy, with only a fraction of patients eligible.

ALD-01 is a retinal cell therapy that could revolutionize RP treatment, offering a mutation-agnostic therapeutic option.

Promising pre-clinical data
ALD-01 is supported by promising preclinical data across three relevant animal models:
·        ALD-01 improved sight in a mouse RP model in a water maze task
·        Successful engraftment and synaptic connectivity in rabbit RP model
·        ¾ of non-human primate models show >40% mfERG improvement four
        weeks post-transplantation

Read the Full Study

Benefits over other cell therapies
ALD-01 outcompetes many other cell therapy options on therapeutic dose requirements as well as the simplicity and scalability of its manufacturing process:
·        Proprietary, regulatory friendly process
·        Shorter manufacturing timeline
·        Lower therapeutic doses
·        Higher yield (no purification step required)
·        Identical product identity across three different PSC lines

Potential impact
Our research suggests that ALD-01 could profoundly impact RP patients’ lives:
·        Halted degenerative vision loss with possible improved vision
·        Improved static perimetry and mobility testing scores at year 1
·        Mutation agnostic option for those not eligible for currently available
         gene therapies

We aim to conduct first-in-human trials for ALD-01 in 2026.
Find out how you can help us get there‍
‍

Tackling chronic heart failure

Chronic heart failure is a major global health burden and is the most common cause of death worldwide. Treatment options are donor transplantation, other forms of surgical intervention, or lifelong symptomatic pharmacological treatment, which costs the US alone $20 billion per year.

‍ALD-02 is a cardiac cell therapy that aims to change this.

Promising pre-clinical data
Pre-clinical data shows that ALD-02 improves heart function in pigs with induced heart disease — without fatal arrythmia:
·        1–3 fold thickening of the ventricular wall
·        5-fold improvement in wall motion
·        >20% increase in left ventricular ejection fraction
·        No fatal arrythmia

Read the Full Study

Benefits over other cell therapies
As well as being supported by promising, high-quality preclinical data, ALD-02 offers other benefits relative to other cell therapies:
·        Xeno-free, fast, robust, and regulatory friendly manufacturing process
·        Faster cell differentiation (10 vs. 12–18 days)
·        Higher yield (no purification step required)
·        4x fewer cells required per dose
·        No open-heart surgery for administration

Potential Impact
Our research suggests that ALD-02 could have a transformative impact on patients suffering with CHF:
·        10% mortality reduction at year 1
·        10% improvement in heart function
·        20% improvement in quality of life
·        50% reduction in hospitalization

‍We aim to conduct first-in-human trials for ALD-02 in 2026.
Find out how you can help us get there
‍

Our Therapies

Our promising stem cell therapies

We have two highly promising proof-of-concept pluripotent stem cell therapy programs in the pipeline. What's more, the manufacturing processes have now been successfully transferred to a CDMO and the economical calculations according to the AlderEdge principles have been completed.

Hope for those with Retinitis Pigmentosa

Retinitis Pigmentosa (RP) is a debilitating inherited blindness disease affecting 1 in 4,000 people. Current treatments are limited to one approved single-mutation gene therapy, with only a fraction of patients eligible.

ALD01 is our lead program, a retinal cell therapy that could revolutionize RP treatment, offering a mutation-agnostic therapeutic option.

‍

Promising preclinical data

Benefits over other cell therapies

Potential impact

We aim to conduct first-in-human trials for ALD01 in 2026

Tackling heart failure

Heart failure is a major global health burden and is the most common cause of death worldwide. Treatment options are donor transplantation, other forms of surgical intervention, or lifelong symptomatic pharmacological treatment, which costs the US alone $20 billion per year.

ALD02 will be our second program, a cardiac cell therapy that aims to regenerate functional heart tissue.

Promising preclinical data

Benefits over other cell therapies

Potential impact

We aim to conduct further proof-of-concept studies before planning first-in-human trials.

Hope for those with Retinitis Pigmentosa

Tackling chronic heart failure

Research Highlights

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