BioCentury | Dec 31, 2020
Regulation

Dec. 30 Quick Takes: A first for Chi-Med; plus Orphazyme, BMS, FDA REMS, Pfizer, Astellas, Alkermes, Osmotica, Quanterix, Ampio and Novartis

Chi-Med’s surufatinib approved in ChinaChina’s National Medical Products Administration (NMPA) approved an NDA from Hutchison China MediTech Ltd. (NASDAQ:HCM; LSE:HCM) for Sulanda surufatinib to treat non-pancreatic neuroendocrine tumors. The FGFR, VEGFR and CSF1R inhibitor is...
BioCentury | Jun 30, 2020
Product Development

Second death in Audentes gene therapy trial may highlight need for more careful dosing

The report of a second patient death in a trial of an AAV8-based gene therapy from Audentes and Astellas renewed decades-old concerns over the modality’s safety, but the adverse events may reflect a need for...
BioCentury | Apr 2, 2020
Finance

Dynacure taps U.S. crossovers in €50M series C to fund orphan muscle disease studies

Dynacure expects its €50 million ($55.4 million) series C round to fund an ongoing trial of its lead program for myotubular and centronuclear myopathies in adults, as well as a planned pediatric study. Together, the...
BioCentury | May 2, 2019
Clinical News

Modest differences in outcomes for doses of Audentes myopathy gene therapy

Audentes presented updated data from the Phase I/II ASPIRO trial of AT132 to treat X-linked myotubular myopathy (XLMTM) showing that the trial's low and high doses had comparable safety and efficacy profiles. Chairman and CEO...
BioCentury | Apr 8, 2019
Company News

Audentes to develop vectorized antisense therapies with Nationwide

Audentes partnered with Nationwide Children's Hospital to develop antisense oligonucleotides delivered with the biotech's adeno-associated viral (AAV) vectors to treat muscular dystrophies. Audentes Therapeutics Inc. (NASDAQ:BOLD) Chairman and CEO Matt Patterson told BioCentury the decision...
BioCentury | Dec 14, 2018
Product R&D

Gene therapy’s make or buy choice

Whether or not to bring manufacturing in-house for gene therapy companies is becoming a critical choice, as contract manufacturers struggle to keep up with demand. The trade-off for the upfront investment risk is control over...
BioCentury | Oct 12, 2018
Financial News

Gene therapy company Audentes raises $151M

On the heels of data for one of its gene therapies, Audentes Therapeutics Inc. (NASDAQ:BOLD) raised $150.8 million on Oct. 10 through the sale of 5.2 million shares at $29 in a follow-on underwritten by...
BioCentury | Oct 11, 2018
Financial News

Gene therapy company Audentes raises $151M

On the heels of data for one of its gene therapies, Audentes Therapeutics Inc. (NASDAQ:BOLD) raised $150.8 million Wednesday through the sale of 5.2 million shares at $29 in a follow-on underwritten by BofA Merrill...
BioCentury | Aug 10, 2018
Clinical News

Audentes reports Phase I/II muscle biopsy data for myopathy gene therapy

Audentes Therapeutics Inc. (NASDAQ:BOLD) reported data from its 24-week muscle biopsy analyses of the first dose cohort and said an IDMC recommended proceeding to the second dose cohort of the Phase I/II ASPIRO trial evaluating...
BioCentury | May 25, 2018
Clinical News

Audentes reports updated Phase I/II myopathy data for AT132

Audentes Therapeutics Inc. (NASDAQ:BOLD) reported updated interim data from the first cohort of the open-label, dose-escalation, U.S. Phase I/II ASPIRO trial testing AT132 in patients with X-linked myotubular myopathy (XLMTM). The presentation at the American...
Items per page:
1 - 10 of 59
BioCentury | Dec 31, 2020
Regulation

Dec. 30 Quick Takes: A first for Chi-Med; plus Orphazyme, BMS, FDA REMS, Pfizer, Astellas, Alkermes, Osmotica, Quanterix, Ampio and Novartis

Chi-Med’s surufatinib approved in ChinaChina’s National Medical Products Administration (NMPA) approved an NDA from Hutchison China MediTech Ltd. (NASDAQ:HCM; LSE:HCM) for Sulanda surufatinib to treat non-pancreatic neuroendocrine tumors. The FGFR, VEGFR and CSF1R inhibitor is...
BioCentury | Jun 30, 2020
Product Development

Second death in Audentes gene therapy trial may highlight need for more careful dosing

The report of a second patient death in a trial of an AAV8-based gene therapy from Audentes and Astellas renewed decades-old concerns over the modality’s safety, but the adverse events may reflect a need for...
BioCentury | Apr 2, 2020
Finance

Dynacure taps U.S. crossovers in €50M series C to fund orphan muscle disease studies

Dynacure expects its €50 million ($55.4 million) series C round to fund an ongoing trial of its lead program for myotubular and centronuclear myopathies in adults, as well as a planned pediatric study. Together, the...
BioCentury | May 2, 2019
Clinical News

Modest differences in outcomes for doses of Audentes myopathy gene therapy

Audentes presented updated data from the Phase I/II ASPIRO trial of AT132 to treat X-linked myotubular myopathy (XLMTM) showing that the trial's low and high doses had comparable safety and efficacy profiles. Chairman and CEO...
BioCentury | Apr 8, 2019
Company News

Audentes to develop vectorized antisense therapies with Nationwide

Audentes partnered with Nationwide Children's Hospital to develop antisense oligonucleotides delivered with the biotech's adeno-associated viral (AAV) vectors to treat muscular dystrophies. Audentes Therapeutics Inc. (NASDAQ:BOLD) Chairman and CEO Matt Patterson told BioCentury the decision...
BioCentury | Dec 14, 2018
Product R&D

Gene therapy’s make or buy choice

Whether or not to bring manufacturing in-house for gene therapy companies is becoming a critical choice, as contract manufacturers struggle to keep up with demand. The trade-off for the upfront investment risk is control over...
BioCentury | Oct 12, 2018
Financial News

Gene therapy company Audentes raises $151M

On the heels of data for one of its gene therapies, Audentes Therapeutics Inc. (NASDAQ:BOLD) raised $150.8 million on Oct. 10 through the sale of 5.2 million shares at $29 in a follow-on underwritten by...
BioCentury | Oct 11, 2018
Financial News

Gene therapy company Audentes raises $151M

On the heels of data for one of its gene therapies, Audentes Therapeutics Inc. (NASDAQ:BOLD) raised $150.8 million Wednesday through the sale of 5.2 million shares at $29 in a follow-on underwritten by BofA Merrill...
BioCentury | Aug 10, 2018
Clinical News

Audentes reports Phase I/II muscle biopsy data for myopathy gene therapy

Audentes Therapeutics Inc. (NASDAQ:BOLD) reported data from its 24-week muscle biopsy analyses of the first dose cohort and said an IDMC recommended proceeding to the second dose cohort of the Phase I/II ASPIRO trial evaluating...
BioCentury | May 25, 2018
Clinical News

Audentes reports updated Phase I/II myopathy data for AT132

Audentes Therapeutics Inc. (NASDAQ:BOLD) reported updated interim data from the first cohort of the open-label, dose-escalation, U.S. Phase I/II ASPIRO trial testing AT132 in patients with X-linked myotubular myopathy (XLMTM). The presentation at the American...
Items per page:
1 - 10 of 59