17:40 , Apr 25, 2019 |  BC Innovations  |  Translation in Brief

Risking CRISPR correction before birth

A team from CHOP and UPenn has published its second study showing CRISPR-based gene editing can treat deadly diseases in the womb but has yet to address the safety repercussions for the mother and fetus....
23:20 , Mar 15, 2019 |  BioCentury  |  Emerging Company Profile

Neurogene: Genes over ERT for monogenic CNS diseases

Neurogene is developing disease-modifying gene therapies for two monogenic, pediatric disorders in which enzyme replacement is not feasible or hasn't been explored. Aspartylglucosaminuria (AGU) is a lysosomal storage disorder caused by autosomal recessive AGA mutations...
18:02 , Mar 13, 2019 |  BC Innovations  |  Distillery Therapeutics

Cocktails of AAV vectors encoding anti-HIV bNAbs to treat the infection

DISEASE CATEGORY: Infectious disease INDICATION: HIV/AIDS Non-human primate studies suggest cocktails of viral vectors encoding broadly neutralizing antibodies (bNAbs) against multiple HIV antigens could help treat HIV. The antibodies consist of variable regions of previously...
18:41 , Feb 15, 2019 |  BC Week In Review  |  Financial News

Neurogene raises $68.5M series A for neurological disease gene therapies

Neurogene Inc. (New York, N.Y.) raised $68.5 million in a series A round to fund development of its adeno-associated viral (AAV)-based gene therapies to treat rare neurological diseases. Samsara BioCapital, EcoR1 Capital, Cormorant Asset Management,...
12:05 , Feb 12, 2019 |  BC Extra  |  Financial News

Neurogene raises $68.5M series A for neurological disease gene therapies

Neurogene Inc. (New York, N.Y.) raised $68.5 million in a series A round to fund development of its adeno-associated viral (AAV)-based gene therapies to treat rare neurological diseases. Samsara BioCapital, EcoR1 Capital, Cormorant Asset Management,...
04:20 , Dec 21, 2018 |  BC Innovations  |  Product R&D

Cell therapies seek solid ground

To make cell therapies work in solid tumors, drug companies will have to either master combination therapies or figure out how to make their treatments trigger broad immune responses that extend beyond the antigen specificity...
01:14 , Nov 16, 2018 |  BC Innovations  |  Emerging Company Profile

Virion: Harnessing viral defects

Virion Biotherapeutics Ltd. has capitalized on a naturally occurring viral mutation to develop broad-spectrum antivirals with lower potential for resistance than marketed products. Its lead compound, VH244, is in preclinical development to treat respiratory infections...
20:24 , Oct 31, 2018 |  BC Innovations  |  Distillery Therapeutics

Cancer; infectious disease

INDICATION: Cancer; melanoma; cervical cancer; influenza virus Mouse and monkey studies suggest simvastatin could be used as an adjuvant to boost the efficacy of cancer and influenza vaccines. In mice, immunization with the antigen ovalbumin...
00:36 , Oct 19, 2018 |  BC Innovations  |  Tools & Techniques

Allogeneic CARs on the horizon

With the first generation of autologous, personalized CAR T cell therapies on the market, the field is turning to allogeneic technologies to provide better scale and lower costs. But drug makers are learning that as...
00:12 , Oct 10, 2018 |  BC Extra  |  Preclinical News

In utero base editing corrects metabolic birth defect

Researchers from Children's Hospital of Philadelphia and University of Pennsylvania opened the door to prenatal genome editing by using a relatively new CRISPR-based approach called base editor 3 to correct tyrosinemia type I, a rare...