Yale University has teamed up with cancer biotech X4 Pharmaceuticals to work on its therapy for WHIM syndrome, a rare genetic disorder that plays havoc with the immune system but has no approved treatment.
The Cambridge, Massachusetts, biotechwhich specializes in drugs targeting the CXCR4 receptorsays it will help Yale investigate the molecular mechanisms behind WHIM syndrome, specifically the role that CXCR4 mutations may play in the disease. CXCR4 is the receptor for the chemokine CXCL12, and research has suggested that the interplay of the receptor and its ligand can downplay immune responses.
In patients with WHIM syndrome, reduced immunity makes them vulnerable to a range of symptoms, notablyas the acronym indicateswarts, hypogammaglobulinemia (low antibody levels), infections, and myelokathexis (a severe deficiency in white blood cells).
Some patients are treated off-label with GCSF drugs such as filgrastim to improve white blood cell counts, while antibiotics and infusions of immune globulin are used to try to reduce infections. Still, there are no drugs available to treat the underlying disease mechanism.
X4 was launched with financial backing from Genzyme founder Henri Termeer before his death earlier this year, and came out of stealth mode in 2015 around lead drug X4P-001, a CXCR4 inhibitor that is being tested in phase 1/2 trials in clear cell renal cell carcinoma (ccRCC), melanomaand other solid tumors.
The biotech has always had ambitions outside of cancer, however, and started a phase 2/3 trial of a low-dose formulation of X4P-001 in WHIM syndrome January, with the aim of enrolling 33 patients ages 13 or older with the disorder. It is hoping to complete the trial in August 2019.
Linking with Yale gives X4 the opportunity to collaborate with Joo Pedro Pereira, Ph.D., an associate professor of immunobiology who is involved with Yale's stem cell and cancer centers. His research focuses on the process that generates many different cell types including all immune cells, and its role in conferring immunity.
"The incorrect positioning of immune cells in primary and secondary immune organs due to CXCR4 mutations has been well documented," said Pereira.
"This research will elucidate the fundamental mechanisms that lead to chronic impairment of the immune system, particularly of long-term immunity, as a result of aberrant immune cell positioning and trafficking."
The number of patients with WHIM is hard to estimate, but X4 reckons that there could be several thousand people worldwide affected with the disorder.
Another group with WHIM syndrome in its sights is the National Institute of Allergy and Infectious Diseases (NIAID), which is running a study comparing Sanofi Genzyme's Mozobil (plerixafor) with filgrastim that is due to generate results in 2021.
Go here to see the original:
X4 joins hands with Yale on rare disease program - FierceBiotech
- Heres How Fasting Benefits Your Mental and Physical Wellbeing - News18 - October 4th, 2022
- Asymmetrex Gives First Cell Culture Core Facility Introduction to Online Rapid Stem Cell Counting in the Institute for Applied Life Sciences at... - September 25th, 2022
- Asymmetrex's Introduction of Online Calculators for Determination of the Dosage of Therapeutic Stem Cells Announced as a Reformation in Stem Cell... - September 16th, 2022
- UMass Dartmouth awarded $750000 Massachusetts Life Science grant to diversify the field - New Bedford Guide - September 16th, 2022
- ALS Thought Leaders Weigh in Ahead of Second Amylyx Adcomm - BioSpace - September 8th, 2022
- Scientists convert kidney to universal O blood type - Freethink - August 30th, 2022
- Case Study: SARS-CoV-2 Virus Infecting the Inner Ear - Victoria News - August 14th, 2022
- Evidence Is Growing That LSD Improves Learning and Memory - Futurism - August 14th, 2022
- Rice University: Rice, Baylor developing implants to heal heart attack injuries | India Education | Latest Education News | Global Educational News |... - August 14th, 2022
- Iconic everyday inventions and the women behind them - YourStory - August 14th, 2022
- Head to Head Analysis: VolitionRx (NYSE:VNRX) vs. Intellia Therapeutics (NASDAQ:NTLA) - Defense World - August 14th, 2022
- Man who saved life as stem cell donor urges others to help his friend find a match after five-year search - Lancashire Evening Post - August 5th, 2022
- The 3D Cell Culture Market is expected to reach a value of USD 3721.86 Million by 2027, at a CAGR of 13.4% (2021 2027) - Digital Journal - August 5th, 2022
- Flow Cytometry Market is expected to reach a value of USD 11467.80 Million by 2027, at a CAGR of 8.88% over the forecast period (2021 2027) - Digital... - July 27th, 2022
- Stem Cells Market Competitive Insights And Global Outlook 2022 To 2027 Vcanbio, Boyalife, Beikebiotech, Thermo Fisher Scientific (Massachusetts, US),... - July 11th, 2022
- Seven UMass Amherst Faculty Members Receive NSF CAREER Awards in 2021-22 Academic Year - UMass News and Media Relations - July 11th, 2022
- BrainStorm Strengthens Executive Team with Key Appointments in R&D and Legal - GuruFocus.com - June 22nd, 2022
- Asymmetrex Presents New Developments in its Rapid Stem Cell Counting Technology at the ARMI|BioFabUSA Meeting in the Millyard on Regenerative Medicine... - June 13th, 2022
- Investigators Seek to Push Combination Therapy to the Front-line Treatment of aGVHD - OncLive - June 4th, 2022
- Stem Cells Market 2022 Industry Analysis, Segmentation, Share, Size, Opportunities and Forecast to 2027 The Greater Binghamton Business Journal - The... - June 4th, 2022
- Sheldon Krimsky, Who Warned of Profit Motive in Science, Dies at 80 - The New York Times - May 15th, 2022
- We met during cancer treatments, fell in love and got married - New York Post - May 15th, 2022
- Ticking time bombs of DNA mutation may dictate when animals die - Livescience.com - May 2nd, 2022
- Sen. Orrin Hatch's legacy tracks the GOP's evolution on health - Wisconsin Public Radio - May 2nd, 2022
- Emerging interactions between skin stem cells and their ... - April 6th, 2022
- Priothera Receives R&D Innovation Loan from Bpifrance - PR Newswire - April 6th, 2022
- What is Regeneration? review: A dive into the science of regrowth - New Scientist - April 6th, 2022
- 5 FDA decisions to watch in the second quarter - BioPharma Dive - April 6th, 2022
- Takeda and the New York Academy of Sciences Announce 2022 Innovators in Science Award Winners - The New York Academy of Sciences - April 6th, 2022
- MorphoSys and Incyte Announce Swissmedic Temporary Approval of Minjuvi(R) (tafasitamab) in Combination with Lenalidomide for the Treatment of Adults... - March 25th, 2022
- January 2022: 2021 Papers of the year - Environmental Factor Newsletter - January 5th, 2022
- Liso-Cel Outperforms Standard Therapy in Improving QoL in Relapsed/Refractory LBCL - www.oncnursingnews.com/ - January 5th, 2022
- Stem cells: Sources, types, and uses - Medical News Today - December 24th, 2021
- cGVHD Paradigm Gains Systemic Options Beyond Steroids, But Real-World Data Are Required - OncLive - October 28th, 2021
- Asymmetrex Will Present a New Test for Therapeutic Stem Cell Potency at the ISSCR 2021 Annual Meeting - PRNewswire - June 23rd, 2021
- Infertility: Men account for at least half of cases. So why have women shouldered the blame? - The Irish Times - June 23rd, 2021
- On systemic sources of early life stress, and empathetic responses - MIT News - June 6th, 2021
- How Coronavirus Damages Lung Cells Within Mere Hours And What Drugs Could Halt COVID-19 Infection - SciTechDaily - February 1st, 2021
- Orchard Therapeutics Outlines Comprehensive Presence at 2021 WORLDSymposium - GlobeNewswire - February 1st, 2021
- NurOwn May Be Given to Early ALS Patients in US Who Finished Phase... - ALS News Today - December 20th, 2020
- Be Biopharma debuts with $52M to advance engineered B-cell therapies - FierceBiotech - October 24th, 2020
- Targeted Therapy for Non-Hodgkin Lymphoma: Current Progress and Future Plans - Cancer Therapy Advisor - September 5th, 2020
- Massachusetts Eye and Ear Enters Licensing Agreement with Biogen to Develop Treatment for Inherited Retinal Disorder - Newswise - July 2nd, 2020
- Startup targets glioblastoma tumors with CAR-T therapy - FierceBiotech - May 28th, 2020
- Infrared Laser Treatment of TBI, PTSD, and Depression: An Expert Perspective - Psychiatry Advisor - April 3rd, 2020
- 10 Of The Biggest World Revelations In The 21st Century - World Atlas - February 29th, 2020
- Where Are They Now? Top 3 Biotech Startups From NextGen Bio Class of 2018 - BioSpace - January 10th, 2020
- Firm adds a new wrinkle to anti-aging products - Williamson Daily News - January 5th, 2020
- Top Emerging Technologies of the Year - Technowize - December 29th, 2019
- New Podcast Sponsored by Asymmetrex Increases Awareness to the Need for Stem Cell Dose in Stem Cell Treatments - PR Web - November 9th, 2019
- Exercise found to block chronic inflammation in mice - Harvard Gazette - November 9th, 2019
- Arkuda bags $44M to target progranulin and head off inherited dementia - FierceBiotech - November 9th, 2019
- New study reveals why breast cancer spreads to the brain - USC News - October 23rd, 2019
- Comparison of Merus N.V. (MRUS) and Sage Therapeutics Inc. (NASDAQ:SAGE) - MS Wkly - October 23rd, 2019
- Reviewing Cellectis S.A. (CLLS)'s and Magenta Therapeutics Inc. (NASDAQ:MGTA)'s results - MS Wkly - October 23rd, 2019
- Massachusetts Stem Cells | Stem Cell TV - September 10th, 2019
- Chronic variable stress activates hematopoietic stem cells ... - April 12th, 2019
- Boston, MA, Stem Cell Transplant, Weston, Nantucket ... - January 10th, 2019
- Cloning/Embryonic Stem Cells - National Human Genome ... - July 5th, 2018
- Alternate Methods for Preparing Pluripotent Stem Cells ... - September 25th, 2017
- Doubts raised about CRISPR gene-editing study in human embryos - Nature.com - September 2nd, 2017
- For Lowell native, stem cell match becomes a match as friends - Lowell Sun - September 2nd, 2017
- Lymphoma Patient's Brain Tumor Disappeared After She Received JCAR017, Study Reports - Lymphoma News Today - September 2nd, 2017
- Asymmetrex Introduces New Contract Service For Producing ... - PR Web (press release) - August 29th, 2017
- ORGANOID - Science Magazine - August 27th, 2017
- Current humanized mice not good models for studying stem cell transplants, say researchers - Scope (blog) - August 27th, 2017
- Mouse Model of Human Immune System Inadequate for Stem Cell ... - Technology Networks - August 27th, 2017
- CRISPR fixes disease gene in viable human embryos - Nature.com - August 6th, 2017
- TGF-1: ALS Astrocytes' Secret Sauce? - ALS Research Forum - August 6th, 2017
- From Stem Cells to Human Development - September 2016 ... - December 4th, 2016
- Stem cell controversy - Wikipedia - December 1st, 2016
- What Are Stem Cells? - Massachusetts General Hospital ... - November 22nd, 2016
- New England Cord Blood Bank - Cord Blood and Cord Tissue ... - November 22nd, 2016
- Stem-cell-based therapy promising for treatment of breast ... - September 26th, 2016
- Stem Cell FAQ - Massachusetts General Hospital, Boston, MA - July 27th, 2016
- Stem Cell Facts - University of Massachusetts Medical School - July 27th, 2016
- Scientists engineer toxin-secreting stem cells to treat ... - October 19th, 2015
- Cloning/Embryonic Stem Cells - Genome.gov - October 19th, 2015
- Biomedical engineer developing nanomaterial for healing broken bones - March 18th, 2015
- Researchers identify a vital protein that can determine head and brain development - March 13th, 2015