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Seres Therapeutics Announces Initiation of Enrollment in SER-155 Phase Ib Study Cohort 2 in Individuals Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

[Seres Therapeutics, Inc.] Seres Therapeutics, Inc. announced that enrollment in Cohort 2 in its SER-155 Phase Ib study was underway. SER-155, an investigational oral microbiome therapeutic, was designed to reduce the incidence of gastrointestinal infections, bloodstream infections, and graft versus host disease in individuals undergoing allogeneic hematopoietic stem cell transplantation.

Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-A501 Gene Therapy for Danon Disease

[Rocket Pharmaceuticals, Inc.] Rocket Pharmaceuticals, Inc. announced that the FDA has granted RMAT designation to RP-A501, the Company’s investigational adeno-associated virus-based gene therapy for the treatment of Danon Disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available.

Disgraced CRISPR-Baby Scientist’s ‘Publicity Stunt’ Frustrates Researchers

[Nature] He Jiankui, the Chinese biophysicist who shocked the world by creating the first children with edited genomes, says research must accept moral and ethical constraints, but is otherwise refusing to speak about the work that landed him in jail for three years.

Grants Awarded to Better Understand the No. 1 Birth Defect

[American Heart Association] Six promising researchers will advance their work to better understand and treat the most common birth defect in the US, congenital heart defects (CHDs), with support from the American Heart Association and The Children’s Heart Foundation’s Congenital Heart Defect Research Awards program.

Onconova Therapeutics Announces Additional Clinical Data Demonstrating Rigosertib’s Monotherapy Activity In RDEB-Associated Squamous Cell Carcinoma

[Onconova Therapeutics, Inc.] Onconova Therapeutics, Inc. announced that the second of two evaluable participants in an investigator-initiated Phase II program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC) achieved a complete response of all cancerous skin lesions following 4 treatment cycles and remains on oral rigosertib.

Kiora Pharmaceuticals Receives Investigational New Drug Approval for Phase II Study of KIO-101 for Treatment of Ocular Presentation of Autoimmune Diseases

[Kiora Pharmaceuticals Inc. (Yahoo News)] The study will enroll approximately 120 patients in a multi-center, controlled, randomized, double-masked trial assessing the safety and efficacy of KIO-101 eye drops in patients living with autoimmune disease who have signs and symptoms of ocular surface disease.

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