C12Y401/01028

GENE THERAPY COMPOSITIONS AND METHODS FOR TREATING PARKINSON'S DISEASE

A method of improving motor function and reducing dyskinesia in a subject suffering from a neurodegenerative disease or a disease where endogenous dopamine levels are reduced in the subject comprising administering an effective amount of a viral vector comprising a nucleic acid construct comprising (i) a nucleotide sequence which encodes tyrosine hydroxylase (TH), (ii) a nucleotide sequence which encodes GTP-cyclohydrolase I (CH1), (iii) a nucleotide sequence which encodes Aromatic Amino Acid Dopa Decarboxylase (AADC), or any combination thereof to the subject.

MULTI-SUBSTITUENT PSILOCYBIN DERIVATIVES AND METHODS OF USING
20230270768 · 2023-08-31 ·

Disclosed are novel multi-substituent psilocybin derivative compounds and pharmaceutical and recreational drug formulations containing the same. The compounds may be produced by reacting a reactant psilocybin derivative with a substituent containing compound.

THERAPEUTIC MICROBES

The invention relates to microbial cells and microbial cells for use as a medicament, the cells expressing a recombinant nucleic acid encoding a eukaryotic tyrosine hydroxylase. The cells produce L-DOPA and dopamine.

METHODS AND SYSTEMS FOR PRODUCING AAV PARTICLES

The present disclosure describes methods and systems for use in the production of recombinant adeno-associated virus (rAAV) particles comprising a payload (e.g., a polynucleotide encoding aromatic L-amino acid decarboxylase (AADC) or a functional variant thereof). In certain embodiments, the production process uses Sf9 insect cells as viral production cells. In certain embodiments, the production process and system use Baculoviral Expression Vectors (BEVs) and/or Baculoviral Infected Insect Cells (BIICs) in the production of rAAV particles.

ENZYMES AND REGULATORY PROTEINS IN TRYPTAMINE METABOLISM

Provided are non-naturally occurring nucleic acids comprising a sequence encoding an enzyme or regulatory protein in tryptamine metabolism. Also provided are a recombinant microorganisms expressing the enzyme or regulatory protein. Methods of expressing the enzyme or regulatory protein are additionally provided.

ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN STRIATAL NEURONS

Artificial expression constructs for modulating gene expression in striatal neurons are described. The artificial expression constructs can be used to express heterologous genes in striatal neurons including in striatal medium spiny neuron-pan, striatal medium spiny neuron-indirect pathway, striatal medium spiny neuron-direct pathway, striatal interneuron-cholinergic, and Drd3+ medium spiny neurons in olfactory tubercle. The artificial expression constructs can be used for many purposes, including to research and treat movement disorders such as Parkinson's disease and Huntington's disease.

AADC polynucleotides for the treatment of Parkinson's disease

The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment of Parkinson's Disease.

AADC POLYNUCLEOTIDES FOR THE TREATMENT OF PARKINSON'S DISEASE
20210338786 · 2021-11-04 ·

The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment of Parkinson's Disease.

ENGINEERED TRYPTOPHAN DECARBOXYLASES AND USES THEREOF FOR SYNTHESIZING TRYPTAMINE ANALOGS

Unnatural, mutant tryptophan decarboxylase proteins and methods of using same. The mutants have an amino acid sequence at least 80% identical to SEQ ID NO:1 and have substitutions at one or more of amino acid positions 98, 99, 120, 126, 339, 349, and 355 of SEQ ID NO:1. The mutants can have enhanced activity and/or substrate promiscuity and be used for synthesizing tryptamine analogs.

METHOD FOR TREATING ALZHEIMER'S DISEASE BY REGULATING AMINO ACID LEVEL
20220280589 · 2022-09-08 ·

Provided is a use of an agent for regulating an amino acid level in preparing a drug for treating Alzheimer's disease in a subject.