Gene

Krystal Biotech Announces First Patient Dosed in Phase 1 Clinical Trial of Inhaled KB707 for the Treatment of Locally Advanced or Metastatic Solid Tumors of the Lung

Retrieved on: 
Monday, April 22, 2024
FDA, Cancer, Inhalation, Interleukin, Suma, Food, Cystic fibrosis, Drug, KRYS, NCT, Senior, Research and development, Patient, Lung, Clinical trial, Krystal, Gene, Pharmaceutical industry

PITTSBURGH, April 22, 2024 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company, announced today that the first patient was dosed in its Phase 1 clinical trial (KYANITE-1) evaluating inhaled KB707, a modified HSV-1 vector designed to deliver genes encoding both human interleukin-12 (IL-12) and interleukin-2 (IL-2) to the lung, for the treatment of patients with locally advanced or metastatic solid tumors of the lung.

Key Points: 
  • “Cytokine delivery via inhalation is a first-of-its-kind therapeutic approach made possible by the unique attributes of Krystal’s HSV-1-based vector platform.
  • Together with intratumoral KB707, inhaled KB707 has the potential to significantly expand the clinical utility of cytokine therapy to treat a wide range of otherwise difficult-to-treat and standard of care refractory solid tumors.
  • “Dosing the first patient in our inhaled KB707 trial is another important milestone for our oncology program and for Krystal,” said Suma Krishnan, President, Research & Development, Krystal Biotech.
  • In July 2023, the FDA granted intratumoral KB707 Fast Track Designation for the treatment of anti-PD-1 relapsed/refractory locally advanced or metastatic melanoma.

Ring Therapeutics Announces Presentations at the 27th Annual American Society of Gene and Cell Therapy

Retrieved on: 
Monday, April 22, 2024
Tropism, Therapy, Flagship Pioneering, Baltimore Convention Center, Gene, Society, Cell, Conference, Vaccine

CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Ring Therapeutics, a life sciences company founded by Flagship Pioneering to revolutionize genetic medicines with its commensal virome platform, today announced an oral presentation and two poster presentations at the 27th Annual American Society of Gene & Cell Therapy (ASGCT) Conference, to be held in Baltimore, Maryland from May 7 – 11, 2024. These presentations showcase the immense promise of a new class of viral vectors from Ring’s Anellogy™ platform to overcome some of the largest barriers associated with delivery of current genetic medicines including redosability and tissue tropism.

Key Points: 
  • CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Ring Therapeutics , a life sciences company founded by Flagship Pioneering to revolutionize genetic medicines with its commensal virome platform, today announced an oral presentation and two poster presentations at the 27th Annual American Society of Gene & Cell Therapy (ASGCT) Conference, to be held in Baltimore, Maryland from May 7 – 11, 2024.
  • These presentations showcase the immense promise of a new class of viral vectors from Ring’s Anellogy™ platform to overcome some of the largest barriers associated with delivery of current genetic medicines including redosability and tissue tropism.
  • Session Title and Location: Emerging Viral Vectors – Ballroom 4, Baltimore Convention Center

ProQR Highlights Upcoming Presentations on Axiomer™ RNA Editing at ASGCT 27th Annual Meeting

Retrieved on: 
Monday, April 22, 2024
EON, NTCP, RNA, Cell, Cholestasis, Oligonucleotide, ACTB, Gene, Eli Lilly, Society, Metabolism, Biomarker, NHP, Annual general meeting, ProQR, Vaccine

LEIDEN, Netherlands & CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will present new preclinical data for its proprietary Axiomer™ RNA editing technology platform, including the first preclinical data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 7-11, 2024, in Baltimore, Maryland.

Key Points: 
  • LEIDEN, Netherlands & CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- ProQR Therapeutics NV (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies, today announced it will present new preclinical data for its proprietary Axiomer™ RNA editing technology platform, including the first preclinical data for its AX-0810 pipeline program for cholestatic diseases targeting NTCP at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 7-11, 2024, in Baltimore, Maryland.
  • “The in vitro and in vivo data that we will be presenting at ASGCT further build upon our experience with Axiomer and highlight the significant potential we see with the platform, including now preclinical proof of concept for cholestatic diseases targeting NTCP,” said Gerard Platenburg, Chief Scientific Officer and co-founder, ProQR.
  • “We continue to be impressed by the robust editing efficiency of Axiomer editing oligonucleotides and look forward to leveraging these data as we advance AX-0810 toward the clinic.”
    Building on its robust platform data, the Company will present additional non-human primate (NHP) data including with ACTB and NTCP and initial in vitro and in vivo preclinical proof of concept for its AX-0810 program targeting NTCP for cholestatic diseases, including hepatic editing in NHP with strong correlating impact on disease-relevant biomarkers.
  • P-726, partner poster presentation by Eli Lilly: “Complex Metabolism and Prolonged PK/PD of a GalNAc-Conjugated Editing Oligonucleotide (EON) in Mice”

MeiraGTx Announces Oral Presentation at the 2024 American Academy of Oral Medicine (AAOM) Annual Conference

Retrieved on: 
Thursday, April 18, 2024
Oral medicine, American Academy, Poster, Safety, Radiation, Month, PRO, CFB, Xerostomia, Conference, Treatment, Gene, Head, Parotid gland, Pharmaceutical industry

LONDON and NEW YORK, April 18, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced the Company gave an oral presentation at the American Academy of Oral Medicine Annual Conference, being held from April 16-20, 2024, at the Hyatt Regency Grand Cypress in Orlando, FL.

Key Points: 
  • LONDON and NEW YORK, April 18, 2024 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced the Company gave an oral presentation at the American Academy of Oral Medicine Annual Conference, being held from April 16-20, 2024, at the Hyatt Regency Grand Cypress in Orlando, FL.
  • We assessed the safety and efficacy of AAV2-hAQP1 gene therapy as a treatment for this condition.
  • Twenty-four participants with Grade 2/3 xerostomia at least five years after completing radiotherapy (2 years if HPV+) were enrolled in this multi-center, open-label, dose-escalation study.
  • The presentation will be available on the Posters and Publications page of the Company’s website after the respective presentation session has concluded.

AskBio receives FDA Fast Track Designation for AB-1002 investigational gene therapy program in congestive heart failure

Retrieved on: 
Thursday, April 18, 2024
FDA, Phase, Bayer, Priority review, Safety, Food, Heart, Research and development, Doctor of Philosophy, Fast track (FDA), Protein phosphatase 1, Therapy, Accelerated approval (FDA), Patient, Gene, CHF, Clinical trial, Heart failure, Pharmaceutical industry

Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the AB-1002 program.

Key Points: 
  • Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the AB-1002 program.
  • AB-1002 is an investigational one-time gene therapy that is administered to the heart with the intention of helping to promote the production of a constitutively active form of protein inhibitor 1 (I-1c) designed to block the action of protein phosphatase 1.
  • “The Fast Track Designation for AB-1002 emphasizes the need to rapidly advance new therapeutic modalities such as gene therapy for people living with congestive heart failure,” said Christian Rommel, PhD, Head of Research and Development at Bayer’s Pharmaceuticals Division.
  • AskBio is currently enrolling patients in the Phase II GenePHIT (Gene PHosphatase Inhibition Therapy) trial of AB-1002 (also known as NAN-101) for the treatment of CHF.4

Telesis Bio Inc. announces strategic focus on game-changing Gibson SOLA enzymatic DNA synthesis (EDS) platform and BioXp mRNA solutions and announces new leadership

Retrieved on: 
Thursday, April 18, 2024
EDS, 9600, Doctor of Philosophy, Growth, Entrepreneurship, Automation, Avantor, DNA, Messenger, Drug discovery, SAN, Gene, Telesis

SAN DIEGO, April 18, 2024 (GLOBE NEWSWIRE) -- Telesis Bio Inc. (NASDAQ: TBIO), a leader in automated benchtop DNA and mRNA synthesis solutions, today announced a focus in strategy enabled by the commercial availability of its groundbreaking Gibson SOLA platform.

Key Points: 
  • SAN DIEGO, April 18, 2024 (GLOBE NEWSWIRE) -- Telesis Bio Inc. (NASDAQ: TBIO), a leader in automated benchtop DNA and mRNA synthesis solutions, today announced a focus in strategy enabled by the commercial availability of its groundbreaking Gibson SOLA platform.
  • “We believe our advances in enzymatic synthesis will ultimately accelerate the current drug discovery paradigm by creating mRNA-based vaccine and therapeutic candidates in days rather than weeks or months,” said Dan Gibson, PhD, Co-Founder of Telesis Bio.
  • Gibson SOLA can deliver on-demand DNA and mRNA in 1 – 2 days, enabling high throughput production at near 100% fidelity in unprecedented time.
  • Expanded the BioXp selling channel to broaden reach and provide easier access to its advanced benchtop synthesis technology.

Jaya Biosciences Presents Updated Preclinical Data in Alzheimer’s Disease at the 45th SIMD Annual Meeting

Retrieved on: 
Tuesday, April 16, 2024
GUSB, Society, PPT1, IDUA, Haploinsufficiency, Gene dosage, Central Waqf Council, Animal, GALC, Research, AAV, IND, CNS, Disease, Science, NAGLU, Patient, DSM-IV codes, SIMD, Toxicology, Annual general meeting, Plaque, SAN, Gene, APP, Antibiotics

During a podium presentation, Jaya Biosciences’ scientific founder and science advisory board chair, Professor Mark Sands, reported on recently updated human genetic analyses suggesting that heterozygous loss-of-function mutations in lysosomal enzyme genes are enriched in Alzheimer’s disease (AD) patients.

Key Points: 
  • During a podium presentation, Jaya Biosciences’ scientific founder and science advisory board chair, Professor Mark Sands, reported on recently updated human genetic analyses suggesting that heterozygous loss-of-function mutations in lysosomal enzyme genes are enriched in Alzheimer’s disease (AD) patients.
  • This new analysis generated from a much larger whole genome sequence database confirmed their previous human genetic findings from a smaller whole exome database.
  • “The updated human genetic data confirmed that heterozygous deleterious mutations in a subset of lysosomal genes are enriched in patients with Alzheimer’s disease,” said Prof. Mark Sands.
  • For more information about the 45th SIMD Annual Meeting, please go to SIMD2024 Meeting .

Eloxx Pharmaceuticals Provides ELX-02 and ZKN-013 Program Updates

Retrieved on: 
Tuesday, April 16, 2024
FDA, Genetics, Drug discovery, ADPKD, Mutation, IPS, Food, Prevalence, Proteinuria, Organoid, OTC, Kidney, Gene, Orphan, RNA, Partnership, UPCR, Almirall, U.S. FDA, FAP, IND, RDEB, Disease, SAD, Patient, Biopsy, Clinical trial, Polycystic kidney disease, ODD, Food and Drug Administration, PKD, Pharmaceutical industry

WATERTOWN, Mass., April 16, 2024 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today provided program updates for ELX-02 and ZKN-013, including Orphan Drug Designation (ODD) for ELX-02.

Key Points: 
  • Eloxx has significantly advanced the development of ELX-02 for the treatment of Alport syndrome with Nonsense Mutations (NMAS).
  • “The recent ELX-02 program updates, including Orphan Drug Designation for ELX-02 for the treatment of Alport Syndrome highlights the significant unmet medical needs of Alport Syndrome patients with Nonsense Mutations,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx.
  • The designation was based on a review of the prevalence of NMAS and the data from the Proof-of-concept Phase 2 Study that Eloxx announced top-line results for in 2023.
  • In March 2024, Eloxx announced an exclusive license agreement with Almirall to develop and commercialize ZKN-013 in orphan indications including RDEB and FAP with nonsense mutations.

TFF Pharmaceuticals Announces Additional Positive Data from the Tacrolimus Inhalation Powder (TFF TAC) Phase 2 Trial Following Oral Presentation at the ISHLT 44th Annual Late Breaking Clinical Science Abstract Sessions

Retrieved on: 
Monday, April 15, 2024
International Society, Part, Kidney, Lung transplantation, Risk, Safety, Immunosuppression, IND, Tacrolimus, Patient, Principal, The Alfred Hospital, Lead, Lung, Gene expression, TFF, Annual general meeting, Gene

FORT WORTH, Texas, April 15, 2024 (GLOBE NEWSWIRE) -- TFF Pharmaceuticals, Inc. (NASDAQ: TFFP) (“the Company”), a clinical-stage biopharmaceutical company focused on developing and commercializing innovative drug products based on its patented Thin Film Freezing (TFF) technology platform, today announced additional positive data from the ongoing Phase 2 study which is evaluating Tacrolimus Inhalation Powder (TFF TAC) for the prevention of organ rejection in lung transplant recipients. These new data were presented by Professor Gregory Snell, the lead Principal Investigator of the TFF TAC Phase 2 study, in an oral presentation at the International Society for Heart and Lung Transplantation (ISHLT) 44th Annual Meeting at a late breaking clinical science abstract session, on April 13, 2024, in Prague, Czech Republic.

Key Points: 
  • “The decreased expression of multiple, rejection-related genes provides compelling evidence at a molecular level that patients who have transitioned from oral tacrolimus onto TFF TAC are not experiencing rejection.
  • Part A of the trial is a 12-week treatment period, and Part B is an optional safety extension period.
  • In March 2024, the Company announced updated data from the first eight patients (N=8) enrolled in the trial.
  • Based on these highly encouraging results, TFF Pharmaceuticals plans to open an IND in the US to explore the use of TFF TAC early post-transplant in preparation for a registrational trial.

Immix Biopharma Announces Late-Breaking NXC-201 Clinical Data Abstract Accepted for Oral Presentation at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

Retrieved on: 
Monday, April 15, 2024
AL amyloidosis, Amyloidosis, MD, Cell, Patient, Gene, Society, LOS, Hematopoietic stem cell transplantation, Cell therapy, Dentistry

Updated clinical data for next-generation CAR-T NXC-201 in relapsed/refractory AL amyloidosis will be presented in Baltimore May 7-11, 2024

Key Points: 
  • Updated clinical data for next-generation CAR-T NXC-201 in relapsed/refractory AL amyloidosis will be presented in Baltimore May 7-11, 2024
    LOS ANGELES, April 15, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (Nasdaq: IMMX) (“ImmixBio”, “Company”, “We” or “Us” or “IMMX”), a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and other autoimmune diseases, today announced that updated NXC-201 clinical data has been selected for presentation at the upcoming 27th Annual Meeting of The American Society of Gene & Cell Therapy (ASGCT) to be held in Baltimore May 7-11, 2024.
  • “We are delighted to present continued clinical progress developing what we believe is the only CAR-T in AL Amyloidosis at the upcoming 27th Annual Meeting of The American Society of Gene & Cell Therapy,” said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and principal study investigator for the NEXICART-1 Phase 1b/2a clinical trial of NXC-201.
  • “A potential one-time treatment such as CAR-T NXC-201 for relapsed/refractory AL amyloidosis patients would be a welcome option for this devastating disease.”
    Event: 27th Annual Meeting of The American Society of Gene and Cell Therapy, Baltimore, MD