Gene therapies, peptides & devices.

• CRISPR / gene editing

  Exagamglogene autotemcel (CRISPR ex vivo)

  Brand: Casgevy

  FDA approved · 2023Show details
  Specialist gene therapy centers only. Not a wellness intervention.

  Ex vivo CRISPR/Cas9 disruption of the BCL11A erythroid enhancer in autologous hematopoietic stem cells, reactivating fetal hemoglobin (HbF).

  Use area: Sickle cell disease and transfusion-dependent β-thalassemia.
• Lentiviral gene therapy

  Lovotibeglogene autotemcel (lentiviral)

  Brand: Lyfgenia

  FDA approved · 2023Show details
  Specialist gene therapy centers only. Boxed warning applies.

  Autologous CD34+ HSCs transduced with a lentiviral vector encoding a modified β-globin gene (βA-T87Q) that produces an anti-sickling hemoglobin.

  Use area: Sickle cell disease.
• AAV gene therapy

  Onasemnogene abeparvovec (AAV9)

  Brand: Zolgensma

  FDA approved · 2019Show details

  Single IV infusion of AAV9 carrying a functional SMN1 transgene.

  Use area: Spinal muscular atrophy in young children.
• AAV gene therapy

  Voretigene neparvovec (AAV2)

  Brand: Luxturna

  FDA approved · 2017Show details

  Subretinal AAV2 delivery of a functional RPE65 gene.

  Use area: Inherited retinal dystrophy.
• AAV gene therapy

  Etranacogene dezaparvovec (AAV5)

  Brand: Hemgenix

  FDA approved · 2022Show details

  AAV5 vector delivering a Padua variant of factor IX.

  Use area: Hemophilia B.
• Cell therapy

  Tisagenlecleucel (CD19 CAR-T)

  Brand: Kymriah

  FDA approved · 2017Show details

  Autologous T cells engineered to express a CD19-directed CAR.

  Use area: B-cell hematologic cancers.
• Antisense oligonucleotide

  Nusinersen (ASO)

  Brand: Spinraza

  FDA approved · 2016Show details

  Intrathecal ASO that promotes SMN2 exon 7 inclusion.

  Use area: Spinal muscular atrophy across ages.
• siRNA

  Inclisiran (siRNA)

  Brand: Leqvio

  FDA approved · 2021Show details

  GalNAc-conjugated siRNA silencing hepatic PCSK9 mRNA.

  Use area: Hypercholesterolemia.
• siRNA

  Patisiran (siRNA)

  Brand: Onpattro

  FDA approved · 2018Show details

  LNP-delivered siRNA targeting hepatic TTR mRNA.

  Use area: Hereditary ATTR amyloidosis with polyneuropathy.
• CRISPR / gene editing

  Base editing (investigational)

  Phase 1/2 trialShow details

  Cytidine or adenine deaminase fused to nickase Cas enables single-base conversion without a double-strand break.

  Use area: Hypercholesterolemia (e.g., hepatic PCSK9 knockdown), hematologic disease.
• CRISPR / gene editing

  Prime editing (preclinical/early trial)

  PreclinicalShow details

  Cas9 nickase fused to reverse transcriptase enables programmable insertions, deletions, and substitutions.

  Use area: Broad rare-disease potential; first human trials emerging.
• Engineered microbiome

  Engineered live biotherapeutic products

  Phase 1/2 trialShow details

  Defined consortia or genetically modified strains delivering metabolic or immune-modulating function.

  Use area: Metabolic, inflammatory, and GI indications.
• Phage therapy

  Bacteriophage therapy

  Phase 1/2 trialShow details

  Targeted lytic phages against specific bacterial pathogens.

  Use area: Multidrug-resistant infections (compassionate use, trials).
• Other

  DIY / wellness CRISPR

  Unsafe / DIY prohibitedShow details
  Never offer, recommend, or condone.

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  Use area: —

Compounds & devices

• Peptide therapy
  GLP-1 RA (semaglutide / liraglutide)

  Approved indications; monitor labs and tolerability.

• Peptide therapy
  GLP-1/GIP co-agonist (tirzepatide)

  Approved indications; body composition focus.

• Investigational peptide
  BPC-157

  Preclinical signals only; sourcing and safety unverified.

• Vagus device
  tVNS device (auricular)

  Emerging evidence in select DGBI / inflammatory contexts.

• Behavioral / device
  HRV biofeedback

  Use trends, not single values.

• Microbiome
  Targeted live biotherapeutic products

  Strain- and indication-specific evidence.