PPD
OCTOBER 12, 2023
These treatments use a vector to introduce the desired nucleic acid code to replace or modify protein expression or use cells to alter/restore a specific cell type. Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics.
The Pharma Data
MARCH 8, 2021
OspA is one of the most dominant surface proteins expressed by the bacteria when present in a tick. Food and Drug Administration (FDA) in July 2017 3. VLA15 has demonstrated strong immunogenicity and safety data in pre-clinical and clinical studies so far. The program was granted Fast Track designation by the U.S.
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The Pharma Data
DECEMBER 8, 2020
CEO and founder Min Li knows a thing or two from his time running GSK’s neuro R&D group before the company shuttered the program in 2017. As we reach the limits of what is easily druggable with protein targeting therapies, reprogramming RNA processing represents an exciting new therapeutic opportunity. Pear Therapeutics.
Alta Sciences
APRIL 17, 2024
The FDA approved the first gene therapy in 2017 for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL), and as of December 2023, there are over 30 approved cell and gene therapies on the market in the U.S.A.
The Pharma Data
OCTOBER 28, 2020
In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. About Ultragenyx.
The Pharma Data
APRIL 8, 2021
EVRYSDI was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively. Food and Drug Administration (FDA) approved EVRYSDI for the treatment of SMA in adults and children 2 months of age and older. About OCREVUS ® (ocrelizumab).
The Pharma Data
SEPTEMBER 2, 2020
Characteristics and Patient Reported Outcomes of Patients Initiating Ocrelizumab in the NARCOMS Registry from 2017 to 2019. Based on preclinical studies, OCREVUS binds to CD20 cell surface proteins expressed on certain B cells, but not on stem cells or plasma cells, suggesting that important functions of the immune system may be preserved.
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