Applied genetics

World's Leading Biobanks Join BC Platforms Global Data Partner Network to Advance Personalized Healthcare Research

Monday, April 6, 2020 - 8:00am

ZURICH and BOSTON, April 6, 2020 /PRNewswire/ -- BC Platforms , a global leader in providing a powerful data and technology platform for personalized healthcare, today announced it has added three new leading biobanks to its Global Data Partner Network, BCRQUEST.com, including Generation Scotland, UK, Bialystok Biobank, Poland, and Biobank Graz Austria.

Key Points: 
  • ZURICH and BOSTON, April 6, 2020 /PRNewswire/ -- BC Platforms , a global leader in providing a powerful data and technology platform for personalized healthcare, today announced it has added three new leading biobanks to its Global Data Partner Network, BCRQUEST.com, including Generation Scotland, UK, Bialystok Biobank, Poland, and Biobank Graz Austria.
  • This will progress the development of personalized healthcare for the benefit of patients, healthcare systems and research."
  • BC Platforms is a global leader in providing a powerful data discovery and analytics platform as well as data science solutions for personalized healthcare.
  • The company has developed and supports a Global Data Partner Network BCRQUEST.com, to provide genomic and clinical cohort data for pharmaceutical and medical research and development.

World's Leading Biobanks Join BC Platforms Global Data Partner Network to Advance Personalized Healthcare Research

Monday, April 6, 2020 - 8:00am

ZURICH and BOSTON, April 6, 2020 /PRNewswire/ -- BC Platforms , a global leader in providing a powerful data and technology platform for personalized healthcare, today announced it has added three new leading biobanks to its Global Data Partner Network, BCRQUEST.com, including Generation Scotland, UK, Bialystok Biobank, Poland, and Biobank Graz Austria.

Key Points: 
  • ZURICH and BOSTON, April 6, 2020 /PRNewswire/ -- BC Platforms , a global leader in providing a powerful data and technology platform for personalized healthcare, today announced it has added three new leading biobanks to its Global Data Partner Network, BCRQUEST.com, including Generation Scotland, UK, Bialystok Biobank, Poland, and Biobank Graz Austria.
  • This will progress the development of personalized healthcare for the benefit of patients, healthcare systems and research."
  • BC Platforms is a global leader in providing a powerful data discovery and analytics platform as well as data science solutions for personalized healthcare.
  • The company has developed and supports a Global Data Partner Network BCRQUEST.com, to provide genomic and clinical cohort data for pharmaceutical and medical research and development.

Rocket Pharmaceuticals Provides Business Update in Light of COVID-19 Pandemic

Thursday, April 2, 2020 - 2:00pm

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders.
  • The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients contending with rare genetic diseases.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.
  • Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes.

StrideBio Appoints Maritza Mcintyre, Ph.D., As Chief Development Officer

Thursday, April 2, 2020 - 1:00pm

RESEARCH TRIANGLE PARK, N.C., April 2, 2020 /PRNewswire/ -- StrideBio, Inc., a leading developer of novel adeno-associated viral (AAV) based gene therapies, today announced the appointment of Maritza McIntyre, Ph.D., as Chief Development Officer.

Key Points: 
  • RESEARCH TRIANGLE PARK, N.C., April 2, 2020 /PRNewswire/ -- StrideBio, Inc., a leading developer of novel adeno-associated viral (AAV) based gene therapies, today announced the appointment of Maritza McIntyre, Ph.D., as Chief Development Officer.
  • Dr. McIntyre is a virologist with over 20 years of experience in the development, evaluation and regulation of gene therapy, biological and small molecule products.
  • "We are delighted to have Dr. McIntyre join the StrideBio team as Chief Development Officer," said Sapan Shah, Ph.D., Chief Executive Officer.
  • I look forward to leading StrideBio's programs through this next phase of translational development and into the clinic."

Rocket Pharmaceuticals Announces Participation at the Guggenheim Healthcare Talks: Genomic Medicines and Rare Disease Day

Thursday, April 2, 2020 - 12:30pm

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that management will participate in the virtual Guggenheim Healthcare Talks: Genomic Medicines and Rare Disease Day on Friday, April 3, 2020.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that management will participate in the virtual Guggenheim Healthcare Talks: Genomic Medicines and Rare Disease Day on Friday, April 3, 2020.
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders.
  • The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients contending with rare genetic diseases.
  • Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition.

Applied Therapeutics Announces IND and Investigator-Initiated Studies of AT-001 in Critical COVID-19 Patients

Thursday, April 2, 2020 - 12:00pm

Multiple AT-001 investigator-initiated trials are now underway to address acute lung inflammation and cardiomyopathy in critical COVID-19 patients.

Key Points: 
  • Multiple AT-001 investigator-initiated trials are now underway to address acute lung inflammation and cardiomyopathy in critical COVID-19 patients.
  • Several New York City hospitals have initiated Emergency Investigational Drug applications for AT-001 use in critical COVID-19 patients.
  • Additional data is being gathered through studies on the effect of AT-001 therapy in critical COVID-19 patients.
  • Both Applied Therapeutics and the FDA understand the urgency to treat critical patients, and the unusual pandemic circumstances surrounding COVID-19.

Unchained Labs jumps into gene therapy and vaccines with new AAV stability application!

Wednesday, April 1, 2020 - 1:00pm

Uncle is already the gold standard for measuring protein stability and, with this new application, jumps in to support researchers in pursuit of novel gene therapies and better vaccines.

Key Points: 
  • Uncle is already the gold standard for measuring protein stability and, with this new application, jumps in to support researchers in pursuit of novel gene therapies and better vaccines.
  • Many gene therapies, immunotherapies and vaccines being developed use adeno-associated viruses or AAVs.
  • A stable AAV stays intact until it finds its target and won't aggregate-or worse, leak potentially toxic DNA into the body.
  • "With this new application, Uncle lends a helping hand to researchers working on life-changing gene therapies and vaccines," said Taegen Clary, VP of Marketing at Unchained Labs.

Unchained Labs jumps into gene therapy and vaccines with new AAV stability application!

Wednesday, April 1, 2020 - 1:00pm

Uncle is already the gold standard for measuring protein stability and, with this new application, jumps in to support researchers in pursuit of novel gene therapies and better vaccines.

Key Points: 
  • Uncle is already the gold standard for measuring protein stability and, with this new application, jumps in to support researchers in pursuit of novel gene therapies and better vaccines.
  • Many gene therapies, immunotherapies and vaccines being developed use adeno-associated viruses or AAVs.
  • A stable AAV stays intact until it finds its target and won't aggregate-or worse, leak potentially toxic DNA into the body.
  • "With this new application, Uncle lends a helping hand to researchers working on life-changing gene therapies and vaccines," said Taegen Clary, VP of Marketing at Unchained Labs.

REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV® Technology Platform for the Treatment of Rare Metabolic Disorder

Tuesday, March 31, 2020 - 9:05pm

Under the terms of the agreement, REGENXBIO has granted Ultragenyx an exclusive, worldwide license, with rights to sublicense, to REGENXBIO's NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder.

Key Points: 
  • Under the terms of the agreement, REGENXBIO has granted Ultragenyx an exclusive, worldwide license, with rights to sublicense, to REGENXBIO's NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder.
  • REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10.
  • REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.
  • Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases.

Ultragenyx Enters into Strategic Partnership with Daiichi Sankyo for Gene Therapy Manufacturing Technology

Wednesday, April 1, 2020 - 1:00am

Under the license and technology access agreement, Ultragenyx granted Daiichi Sankyo a non-exclusive license to intellectual property, including know-how and patent applications, with respect to its HeLa PCL and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products.

Key Points: 
  • Under the license and technology access agreement, Ultragenyx granted Daiichi Sankyo a non-exclusive license to intellectual property, including know-how and patent applications, with respect to its HeLa PCL and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products.
  • The parties will collaborate closely as part of a technology transfer plan to enable Daiichi Sankyo to use the technologies for its internal gene therapy programs.
  • Ultragenyx will also provide strategic consultation to Daiichi Sankyo on the development of both AAV-based gene therapy products and other products for rare diseases.
  • Daiichi Sankyo will reimburse Ultragenyx for all costs associated with the transfer of the manufacturing technology.