Aavantibio Inc., with the help of a large check from Sarepta Therapeutics Inc., has launched with a $107 million series A to pursue gene therapies for treating rare genetic diseases. The lead program is in Friedreich’s ataxia (FA), a rare genetic disease that typically begins in childhood and causes progressive damage to the nervous system.
At the virtual Cell & Gene Meeting on the Mesa, panelists talked about the challenges and benefits of developing cell and gene therapies to treat chronic conditions.
As with many conferences, the Cleveland Clinic’s 2020 Medical Innovation Summit went virtual this year. Still, the event featured the hotly anticipated top 10 list of innovations for 2021 that saw a range of therapies. Ranked in order of expected importance, the list was led by gene therapy for hemoglobinopathies. The top three innovations, including a novel drug for primary-progressive multiple sclerosis and smartphone-connected pacemaker devices, were highlighted in a special presentation.
DUBLIN – Versant Ventures, the founding investor of Crispr Therapeutics AG, is teaming up with one of the academic founders of that company, Stanford University’s Matt Porteus, to start a new gene editing firm, Graphite Bio Inc., which is focused on targeted integration of DNA to achieve a therapeutic benefit.
DUBLIN – Mina Therapeutics Ltd. raised £23 million (US$29.9 million) in a series A round to take forward a pipeline and a platform based on a novel regulatory RNA species, small activating RNAs, which selectively activate gene expression.
DUBLIN – Shares in Amryt Pharma plc surged by more than 36% Sept. 9 on news that Filsuvez (AP-101, Oleogel-S10) attained the primary endpoint of a phase III trial in epidermolysis bullosa.
LONDON – Gyroscope Therapeutics Ltd. is poised to move the field of ocular gene therapy on from the treatment of inherited rare diseases to address more common eye conditions, after receiving FDA 510(k) clearance for its Orbit SDS subretinal delivery device.
A first half of the year progress report from the international advocacy group Alliance for Regenerative Medicine finds that the regenerative medicine and advanced therapy sector is in very good shape and has performed well in terms of both clinical development and fundraising.
A third child with X-linked myotubular myopathy (XLMTM) has died after receiving the higher of two doses of an experimental gene therapy for the rare disease under development at Audentes Therapeutics Inc. Preliminary findings indicate that the immediate cause of death was gastrointestinal bleeding, Audentes said. The trial, testing AT-132 (resamirigene bilparvovec), was already on an FDA clinical hold.
A first half of the year progress report from the international advocacy group Alliance for Regenerative Medicine (ARM), finds that the regenerative medicine and advanced therapy sector is in very good shape and has performed well in terms of both clinical development and fundraising despite the challenges posed by the ongoing COVID-19 pandemic.
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