Voyager shares take a hit as FDA slaps a hold on the IND for Huntington's gene therapy — their #2 play in the pipeline

Voyager shares take a hit as FDA slaps a hold on the IND for Huntington’s gene therapy — their #2 play in the pipeline

A little more than a year after Sanofi handed back rights to Voyager’s $VYGR gene therapy program for Huntington’s in exchange for a cash payment, the biotech says the FDA has placed a hold on the IND needed to get into the clinic. This program is now listed in the number 2 spot on Voyager’s pipeline, behind its lead effort in a pivotal study for Parkinson’s. Voyager is part of a wave of upstarts focused on gene therapy tech as a cure for a variety of diseases. But the field has had more than its fair share of setbacks in ...
Read More
DARPA Awards Moderna up to $56 Million to Enable Small-Scale, Rapid Mobile Manufacturing of Nucleic Acid Vaccines and Therapeutics

DARPA Awards Moderna up to $56 Million to Enable Small-Scale, Rapid Mobile Manufacturing of Nucleic Acid Vaccines and Therapeutics

Moderna, Inc. , (Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced an agreement for a commitment of up to $56 million from the Defense Advanced Research Projects Agency (DARPA) to fund development of a mobile manufacturing protype leveraging Moderna’s existing manufacturing technology that is capable of rapidly producing vaccines and therapeutics. The agreement builds on a previous assistance grant with DARPA established in 2013. The award is part of DARPA’s Nucleic Acids On Demand World-Wide (NOW) initiative to develop a mobile, end-to-end automated manufacturing platform to provide in-field, ...
Read More
Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia A Gene Therapy Treatment

Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia A Gene Therapy Treatment

Pfizer Inc. and Sangamo Therapeutics, Inc. a genomic medicines company, announced on October 07th that the first participant has been dosed in the Phase 3 AFFINE study of giroctocogene fitelparvovec (SB-525), an investigational gene therapy for hemophilia A patients. AFFINE is a global Phase 3, open-label, multicenter, single arm study that will evaluate the efficacy and safety of giroctocogene fitelparvovec in patients with moderately severe to severe hemophilia A. The primary endpoint is impact on annual bleed rate (ABR) through 12 months following treatment with giroctocogene fitelparvovec, compared to ABR on Factor VIII (FVIII) replacement therapy collected in the Phase ...
Read More
Solid Biosciences Announces FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial

Solid Biosciences Announces FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial

Solid Biosciences Inc. announced on October 01st that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Company’s IGNITE DMD Phase I/II clinical trial. As announced in July 2020, the FDA had requested further manufacturing information, updated safety and efficacy data for all patients dosed, and provided direction on total viral load to be administered per patient. Based on the Company’s response to these requests, the FDA acknowledged that the Company satisfactorily addressed all clinical hold questions. As part of its commitment to continuously improve its manufacturing processes, Solid implemented and shared with the FDA manufacturing process changes that ...
Read More
G&CTI, premier think tank indépendant sur les thérapies avancées

G&CTI, premier think tank indépendant sur les thérapies avancées

Dans l’environnement actuel où les thérapies géniques et cellulaires apparaissent certes comme une véritable révolution médicale, mais où de nombreux défis éthiques, sociétaux, politiques, économiques se posent, la quête de réponses opérationnelles s’impose !L’ensemble des acteurs (sociétés savantes, patients, industriels, acteurs émergents, chercheurs, grand public…) est unanime sur la nécessité d’apporter des solutions concrètes aux questions, aux attentes et aux des besoins de chacun via un espace de dialogue et de réflexion libre et indépendant. C’est le positionnement qu’a choisi G&CTi (Gene and Cell Therapy Institute), structure à but non lucratif, au service de l’intérêt général, dont l’ambition est d’apporter ...
Read More
Breaking News: Trend Chart on Innovative Biotherapies - 17th september

Breaking News: Trend Chart on Innovative Biotherapies – 17th september

Trend Chart on Innovative Biotherapies is a FREE NEWSLETTER published by https://www.octopusyx.com/ and http://biopharmanalyses.fr/ Do not hesitate to share this informative document with colleagues and anybody who may be interested by these topics Contents Feature Story -Non Clinical Studies that support viral vector-delivered gene therapies: an EFPIA gene therapy working group perspective BASIC SCIENCE -Paving the road for RNA Therapeutics CLINICAL TRIALS/DATA -Akcea: positive phase II clinical data of Vupanorsen -The NEJM publishes study results evaluating Ionis antisenses therapy -BluebirdBio presents new clinical results with angioedema autostemcell (eli-cell, LentiD) gene therapy for CALD INDUSTRIAL LANDSCAPE & AGREEMENTS -CombiGene ssigns production ...
Read More
CAR T-cell Therapy for Solid Tumors: Bright Future or Dark Reality?

CAR T-cell Therapy for Solid Tumors: Bright Future or Dark Reality?

CAR T-cell therapy has garnered significant excitement due to its success for hematological malignancies in clinical studies leading to the FDA approval of three CD19-targeted CAR T-cell products. In contrast, the clinical experience with CAR T-cell therapy for solid tumors and brain tumors has been less encouraging with only a few patients achieving complete responses. Clinical and preclinical studies have identified multiple ‘roadblocks’ including i) a limited array of targetable antigens and heterogenous antigen expression, ii) limited T-cell fitness and survival before reaching tumor sites, iii) inability of T cells to efficiently traffic to tumor sites and penetrate physical barriers, ...
Read More

Voyager shares take a hit as FDA slaps a hold on the IND for Huntington's gene therapy — their #2 play in the pipeline

Voyager shares take a hit as FDA slaps a hold on the IND for Huntington’s gene therapy — their #2 play in the pipeline

A little more than a year after Sanofi handed back rights to Voyager’s $VYGR gene therapy program for Huntington’s in exchange for a cash payment, the biotech says the FDA has placed a hold on the IND needed to get into the clinic. This program is now listed in the number 2 spot on Voyager’s pipeline, behind its lead effort in a pivotal study for Parkinson’s. Voyager is part of a wave of upstarts focused on gene therapy tech as a cure for a variety of diseases. But the field has had more than its fair share of setbacks in ...
Read More
DARPA Awards Moderna up to $56 Million to Enable Small-Scale, Rapid Mobile Manufacturing of Nucleic Acid Vaccines and Therapeutics

DARPA Awards Moderna up to $56 Million to Enable Small-Scale, Rapid Mobile Manufacturing of Nucleic Acid Vaccines and Therapeutics

Moderna, Inc. , (Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced an agreement for a commitment of up to $56 million from the Defense Advanced Research Projects Agency (DARPA) to fund development of a mobile manufacturing protype leveraging Moderna’s existing manufacturing technology that is capable of rapidly producing vaccines and therapeutics. The agreement builds on a previous assistance grant with DARPA established in 2013. The award is part of DARPA’s Nucleic Acids On Demand World-Wide (NOW) initiative to develop a mobile, end-to-end automated manufacturing platform to provide in-field, ...
Read More
Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia A Gene Therapy Treatment

Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia A Gene Therapy Treatment

Pfizer Inc. and Sangamo Therapeutics, Inc. a genomic medicines company, announced on October 07th that the first participant has been dosed in the Phase 3 AFFINE study of giroctocogene fitelparvovec (SB-525), an investigational gene therapy for hemophilia A patients. AFFINE is a global Phase 3, open-label, multicenter, single arm study that will evaluate the efficacy and safety of giroctocogene fitelparvovec in patients with moderately severe to severe hemophilia A. The primary endpoint is impact on annual bleed rate (ABR) through 12 months following treatment with giroctocogene fitelparvovec, compared to ABR on Factor VIII (FVIII) replacement therapy collected in the Phase ...
Read More
Solid Biosciences Announces FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial

Solid Biosciences Announces FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial

Solid Biosciences Inc. announced on October 01st that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Company’s IGNITE DMD Phase I/II clinical trial. As announced in July 2020, the FDA had requested further manufacturing information, updated safety and efficacy data for all patients dosed, and provided direction on total viral load to be administered per patient. Based on the Company’s response to these requests, the FDA acknowledged that the Company satisfactorily addressed all clinical hold questions. As part of its commitment to continuously improve its manufacturing processes, Solid implemented and shared with the FDA manufacturing process changes that ...
Read More
G&CTI, premier think tank indépendant sur les thérapies avancées

G&CTI, premier think tank indépendant sur les thérapies avancées

Dans l’environnement actuel où les thérapies géniques et cellulaires apparaissent certes comme une véritable révolution médicale, mais où de nombreux défis éthiques, sociétaux, politiques, économiques se posent, la quête de réponses opérationnelles s’impose !L’ensemble des acteurs (sociétés savantes, patients, industriels, acteurs émergents, chercheurs, grand public…) est unanime sur la nécessité d’apporter des solutions concrètes aux questions, aux attentes et aux des besoins de chacun via un espace de dialogue et de réflexion libre et indépendant. C’est le positionnement qu’a choisi G&CTi (Gene and Cell Therapy Institute), structure à but non lucratif, au service de l’intérêt général, dont l’ambition est d’apporter ...
Read More
Breaking News: Trend Chart on Innovative Biotherapies - 17th september

Breaking News: Trend Chart on Innovative Biotherapies – 17th september

Trend Chart on Innovative Biotherapies is a FREE NEWSLETTER published by https://www.octopusyx.com/ and http://biopharmanalyses.fr/ Do not hesitate to share this informative document with colleagues and anybody who may be interested by these topics Contents Feature Story -Non Clinical Studies that support viral vector-delivered gene therapies: an EFPIA gene therapy working group perspective BASIC SCIENCE -Paving the road for RNA Therapeutics CLINICAL TRIALS/DATA -Akcea: positive phase II clinical data of Vupanorsen -The NEJM publishes study results evaluating Ionis antisenses therapy -BluebirdBio presents new clinical results with angioedema autostemcell (eli-cell, LentiD) gene therapy for CALD INDUSTRIAL LANDSCAPE & AGREEMENTS -CombiGene ssigns production ...
Read More
CAR T-cell Therapy for Solid Tumors: Bright Future or Dark Reality?

CAR T-cell Therapy for Solid Tumors: Bright Future or Dark Reality?

CAR T-cell therapy has garnered significant excitement due to its success for hematological malignancies in clinical studies leading to the FDA approval of three CD19-targeted CAR T-cell products. In contrast, the clinical experience with CAR T-cell therapy for solid tumors and brain tumors has been less encouraging with only a few patients achieving complete responses. Clinical and preclinical studies have identified multiple ‘roadblocks’ including i) a limited array of targetable antigens and heterogenous antigen expression, ii) limited T-cell fitness and survival before reaching tumor sites, iii) inability of T cells to efficiently traffic to tumor sites and penetrate physical barriers, ...
Read More
Voyager shares take a hit as FDA slaps a hold on the IND for Huntington's gene therapy — their #2 play in the pipeline

Voyager shares take a hit as FDA slaps a hold on the IND for Huntington’s gene therapy — their #2 play in the pipeline

A little more than a year after Sanofi handed back rights to Voyager’s $VYGR gene therapy program for Huntington’s in exchange for a cash payment, the biotech says the FDA has placed a hold on the IND needed to get into the clinic. This program is now listed in the number 2 spot on Voyager’s pipeline, behind its lead effort in a pivotal study for Parkinson’s. Voyager is part of a wave of upstarts focused on gene therapy tech as a cure for a variety of diseases. But the field has had more than its fair share of setbacks in ...
Read More
DARPA Awards Moderna up to $56 Million to Enable Small-Scale, Rapid Mobile Manufacturing of Nucleic Acid Vaccines and Therapeutics

DARPA Awards Moderna up to $56 Million to Enable Small-Scale, Rapid Mobile Manufacturing of Nucleic Acid Vaccines and Therapeutics

Moderna, Inc. , (Nasdaq: MRNA) a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines to create a new generation of transformative medicines for patients, today announced an agreement for a commitment of up to $56 million from the Defense Advanced Research Projects Agency (DARPA) to fund development of a mobile manufacturing protype leveraging Moderna’s existing manufacturing technology that is capable of rapidly producing vaccines and therapeutics. The agreement builds on a previous assistance grant with DARPA established in 2013. The award is part of DARPA’s Nucleic Acids On Demand World-Wide (NOW) initiative to develop a mobile, end-to-end automated manufacturing platform to provide in-field, ...
Read More
Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia A Gene Therapy Treatment

Pfizer and Sangamo Dose First Participant in Phase 3 Study Evaluating Hemophilia A Gene Therapy Treatment

Pfizer Inc. and Sangamo Therapeutics, Inc. a genomic medicines company, announced on October 07th that the first participant has been dosed in the Phase 3 AFFINE study of giroctocogene fitelparvovec (SB-525), an investigational gene therapy for hemophilia A patients. AFFINE is a global Phase 3, open-label, multicenter, single arm study that will evaluate the efficacy and safety of giroctocogene fitelparvovec in patients with moderately severe to severe hemophilia A. The primary endpoint is impact on annual bleed rate (ABR) through 12 months following treatment with giroctocogene fitelparvovec, compared to ABR on Factor VIII (FVIII) replacement therapy collected in the Phase ...
Read More
Solid Biosciences Announces FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial

Solid Biosciences Announces FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial

Solid Biosciences Inc. announced on October 01st that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Company’s IGNITE DMD Phase I/II clinical trial. As announced in July 2020, the FDA had requested further manufacturing information, updated safety and efficacy data for all patients dosed, and provided direction on total viral load to be administered per patient. Based on the Company’s response to these requests, the FDA acknowledged that the Company satisfactorily addressed all clinical hold questions. As part of its commitment to continuously improve its manufacturing processes, Solid implemented and shared with the FDA manufacturing process changes that ...
Read More
G&CTI, premier think tank indépendant sur les thérapies avancées

G&CTI, premier think tank indépendant sur les thérapies avancées

Dans l’environnement actuel où les thérapies géniques et cellulaires apparaissent certes comme une véritable révolution médicale, mais où de nombreux défis éthiques, sociétaux, politiques, économiques se posent, la quête de réponses opérationnelles s’impose !L’ensemble des acteurs (sociétés savantes, patients, industriels, acteurs émergents, chercheurs, grand public…) est unanime sur la nécessité d’apporter des solutions concrètes aux questions, aux attentes et aux des besoins de chacun via un espace de dialogue et de réflexion libre et indépendant. C’est le positionnement qu’a choisi G&CTi (Gene and Cell Therapy Institute), structure à but non lucratif, au service de l’intérêt général, dont l’ambition est d’apporter ...
Read More
Breaking News: Trend Chart on Innovative Biotherapies - 17th september

Breaking News: Trend Chart on Innovative Biotherapies – 17th september

Trend Chart on Innovative Biotherapies is a FREE NEWSLETTER published by https://www.octopusyx.com/ and http://biopharmanalyses.fr/ Do not hesitate to share this informative document with colleagues and anybody who may be interested by these topics Contents Feature Story -Non Clinical Studies that support viral vector-delivered gene therapies: an EFPIA gene therapy working group perspective BASIC SCIENCE -Paving the road for RNA Therapeutics CLINICAL TRIALS/DATA -Akcea: positive phase II clinical data of Vupanorsen -The NEJM publishes study results evaluating Ionis antisenses therapy -BluebirdBio presents new clinical results with angioedema autostemcell (eli-cell, LentiD) gene therapy for CALD INDUSTRIAL LANDSCAPE & AGREEMENTS -CombiGene ssigns production ...
Read More
CAR T-cell Therapy for Solid Tumors: Bright Future or Dark Reality?

CAR T-cell Therapy for Solid Tumors: Bright Future or Dark Reality?

CAR T-cell therapy has garnered significant excitement due to its success for hematological malignancies in clinical studies leading to the FDA approval of three CD19-targeted CAR T-cell products. In contrast, the clinical experience with CAR T-cell therapy for solid tumors and brain tumors has been less encouraging with only a few patients achieving complete responses. Clinical and preclinical studies have identified multiple ‘roadblocks’ including i) a limited array of targetable antigens and heterogenous antigen expression, ii) limited T-cell fitness and survival before reaching tumor sites, iii) inability of T cells to efficiently traffic to tumor sites and penetrate physical barriers, ...
Read More