Allergan and Editas Medicine dose patient with world’s first ever in vivo CRISPR medicine

Date: 4th March 2020

Article in brief:

The gene-editing field is waiting with hope as the first wave of CRISPR-trials start to release patient data. Now Allergan and Editas Medicine have announced the treatment of the first patient in the BRILLIANCE clinical trial of AGN-151587 (EDIT-101), in a first ever attempt to edit the genome in vivo in humans.

To date all clinical trials involving CRISPRs have performed the gene editing steps in vitro, and then transplanted the edited cells back into the patient.

In September last year we saw an attempt to use CRISPR genome-edited donor cells to treat a HIV patient with blood cancer for the first time.

Then in November, came the release of patient data following clinical trials from CRISPR Therapeutics and Vertex Pharmaceuticals. Similarly, this trial used CRISPR-edited HSPCs, this time for the treatment of transfusion-dependent β thalassemia (TDT), and sickle cell disease (SCD).

The most recent report from last month saw scientists from the University of Pennsylvania and Stanford University School of Medicine, using multiplex CRISPR-Cas9 editing to engineer T cells in three patients with refractory (unresponsive) cancer.

In vivo gene editing

The BRILLIANCE Phase 1/2 clinical trial is designed to assess the safety, tolerability, and efficacy of AGN-151587 in approximately 18 patients with Leber congenital amaurosis 10 (LCA10).

LCA10 is an inherited form of blindness caused by mutations in the centrosomal protein 290 (CEP290) gene. The disorder primarily affects the retina, and typically people affected have severe visual impairment beginning in infancy. It occurs in 2 to 3 per 100,000 of newborns and is one of the most common causes of blindness in children. Currently there are no approved treatment options so this announcement offers hope for thousands of patients.

AGN-151587 is designed to eliminate a common intron 26 mutation in the CEP290 gene that results in retinal degeneration. It is administered directly into the eye via a subretinal injection and delivers the gene editing machinery directly to photoreceptor cells.

This world’s first human study of an in vivo, or inside the body, CRISPR genome editing medicine will no doubt attract much interest over the coming months.

How will this in vivo editing compare to those that occur in a relatively stable, controllable and measurable in vitro environment? With current patient data showing mixed but promising results, will in vivo editing prove safe, and more reliable? We will be waiting with anticipation for the initial patient data to emerge…

 

For more details please see the press release from Editas

Additional details are available on www.clinicaltrials.gov

  1. https://www.gustudentassociation.org/
  2. https://kimmerestaurant.com/
  3. https://www.nyonyafood.com/
  4. https://www.perfectotech.com/
  5. https://www.planetgapyear.com/
  6. https://whatcomvet.com/
  7. https://theclassicyachtexperience.com/
  8. https://www.batonrougerosesociety.org/
  9. https://www.finburysullivan.com/
  10. https://mikrofinanzinstitut.com/
  11. https://www.the-vision-of-harmony.org/
  12. https://www.pantheonpress.com/
  13. https://thefinancialgraduate.com/
  14. https://www.thenutkitchen.com/
  15. https://altiboutique.com/
  16. https://ambushsweden.com/
  17. https://goingonforgod.com/
  18. https://lasdopestattorney.com/
  19. https://www.sewardne.com/
  20. https://www.tehranfestival.com/
  21. https://brysonchristianmontessorischool.com/
  22. https://www.excalibureurope.com/
  23. https://www.originallotsoflox.com/
  24. https://www.wavespace-berlin.com/
  25. https://www.michiganmediates.org/
  26. https://www.yourmyrtlebeachproperty.com/
  27. https://metrcconference.com/
  28. https://biotechscope.com/
  29. https://jzbrasil.com/
  30. https://saafootball.org/
  31. https://griefergames.info/
  32. https://ampalauragarcianoblejas.com/
  33. sbobet
  34. judi parlay
  35. togel kamboja
  36. Pengeluaran Cambodia
  37. judi bola
  38. Togel Kamboja
  39. keluaran Kamboja
  40. slot thailand
  41. togel kamboja
  42. keluaran kamboja
  43. togel Kamboja
  44. slot demo
  45. keluaran cambodia
  46. togel cambodia
  47. live draw macau
  48. slot thailand
  49. pengeluaran kamboja
  50. judi bola
  51. sbobet
  52. slot demo
  53. togel sdy
  54. demo slot
  55. keluaran kamboja
  56. judi sbobet
  57. slot qris
  58. slot qris 5000
  59. slot qris
  60. slot deposit 5000
  61. slot qris
  62. pintarbersamamedan.org
  63. slot qris gacor
  64. slot qris 5000
  65. toto macau
  66. pengeluaran macau
  67. macau pools
  68. slot server thailand super gacor
  69. slot qris 5k
  70. togel hk
  71. togel hk
  72. togel hk
  73. togel hk
  74. data hk
  75. data sdy
  76. togel
  77. data sdy
  78. generasitogel
  79. generasitogel
  80. generasitogel
  81. Live Draw HK
  82. HK Lotto
  83. Live SDY Lotto
  84. Live HK
  85. Live Draw SDY
  86. Live SDY Lotto
  87. situs slot dana
  88. TOGEL SDY
  89. Live Toto Macau
  90. KELUARAN HK
  91. generasitogel
  92. Live Draw HK
  93. Live Draw Singapore
  94. Live HK
  95. pengeluaran macau
  96. Live Draw HK
  97. Live Togel Singapore
  98. Live Draw SDY
  99. Data HK
  100. Live Draw SDY
  101. Live Draw SGP
  102. Togel SDY
  103. Togel HK
  104. Live Togel SGP