Engineered for Patients
Dystrogen Therapeutics
Is a clinical stage chimeric cell therapy company focusing on rare diseases. The company is currently testing Dystrophic Expressing Chimeric (DEC) cell therapy for Duchenne Muscular Dystrophy.
how it works
Duchenne Muscular Dystrophy
Dystrophin Expressing Chimeric (DEC) cell based therapy developed by Dystrogen Therapeutics aims to target all patients suffering from Duchenne muscular dystrophy, irrespective of existing genetic mutation.
Distinct Advantages Over Other DMD Approaches
- Universal therapy for all DMD genetic mutations
- Produces the dystrophin protein without genetic manipulation , unlike microdystrophin, the viral vector modified therapy approach targeting DMD
- Does not modify genes; no related toxicity or off-target mutations and no viral vector integration issues
- Does not require pretreatment or conditioning
- Does not require immunosuppression to support engraftment
- Redosing possible without sensitization and no immunosuppression required
- Can be used as a monotherapy or in combination with other therapeutics
Chimeric Cell Engineering
Chimeric Cell Summary
Chimeric cell therapy represents a unique and novel modality with broad applications across multiple therapeutic categories which could revolutionize the treatment paradigm for broad spectrum of genetic disorders
- Engineered Cell Technology – for Duchenne muscular dystrophy (DMD), a rare, progressive disease without a cure (caused by dystrophin gene mutations) – the therapy is created by “fusing” myoblasts of normal donor origin (allogenic) with myoblasts of the DMD-affected patient (autologous), to create a new generation of dystrophin expressing chimeric (DEC) cells which contain the phenotype of the normal donor while preserving the surface markers of the DMD recipient. DEC cells express significantly higher levels of CD56 and dystrophin when compared to DMD affected myoblasts, thus more closely resembling healthy myoblasts. Because they are chimeras, DEC cells display the recipients’ immune phenotype, thus does not require imunosupression to support engraftment
- Acts like a Trojan horse – once injected, DEC cells are recognized by the recipient as “self” and evade the patient’s immune system (are not rejected), and thus engraft, producing the relevant and missing protein from a full-length, healthy gene (delivered by the normal donor)