Catalyst Biosciences Announces Publication of CB 2679d Preclinical FIX Gene Therapy Data in Blood, the Journal of the America...
April 19 2021 - 8:00AM
Catalyst Biosciences, Inc. (NASDAQ: CBIO) today announced the
publication of preclinical data from its study of CB 2679d-GT in
Hemophilia B mice in Blood, the peer-reviewed, official journal of
the American Society of Hematology. The paper, entitled: "Gene
Therapy for Hemophilia B Using CB 2679d-GT: A Novel Factor IX
Variant with Higher Potency than Factor IX Padua," demonstrated
superiority of the Company's CB 2679d-GT gene therapy candidate
over that of the R338L-Padua variant, which is currently used in
clinical trials.
The study, conducted at the Vrije Universiteit Brussel,
Brussels, Belgium, by the team of Thierry VandenDriessche and
Marinee Chuah in collaboration with Catalyst Biosciences, showed
gene therapy with CB 2679d-GT safely and effectively achieved
sustained factor IX (FIX) levels and significantly outperformed the
R338L-Padua variant in hemophilia B mice. A significantly reduced
bleeding time and total blood loss with CB 2679d-GT gene therapy
compared to mice treated with R338L-Padua demonstrated a more rapid
and robust hemostatic correction. Thus, CB 2679d-GT could
potentially allow for lower and safer vector doses in future human
trials.
"The data demonstrate the strong potential of
our gene therapy candidate," said Grant Blouse, Ph.D., Senior
Vice President, Translational Research at Catalyst Biosciences. "We
are thrilled to have the study results published in Blood to share
with the medical and scientific communities
Thierry VandenDriessche, the lead investigator
at Vrije Universiteit Brussel, Belgium, said, "Collectively, these
data support the efficacy and safety of CB 2679d-GT as a promising
novel alternative to R338L-Padua for hemophilia B gene therapy. Its
potential to obtain higher FIX activity levels and superior
hemostatic efficacy following AAV directed gene therapy in
hemophilia B patients than what is currently achievable with the
R338L-Padua variant validates further clinical development."
About Catalyst Biosciences, the Protease
Medicines companyCatalyst is a research and clinical
development biopharmaceutical company focused on addressing unmet
medical needs in rare disorders of the complement and coagulation
systems. Our protease engineering platform has generated two
late-stage clinical programs, including MarzAA, a subcutaneously
(SQ) administered next-generation engineered coagulation Factor
VIIa (FVIIa) for the treatment of episodic bleeding in subjects
with rare bleeding disorders. Our complement pipeline includes a
preclinical C3-degrader program partnered with Biogen for dry
age-related macular degeneration, an improved complement factor I
protease for SQ replacement therapy in patients with CFI deficiency
and C4b-degraders designed to target disorders of the classical
complement pathway as well as other complement programs in
development.
Forward-Looking Statements This
press release contains forward-looking statements that involve
substantial risks and uncertainties. Forward-looking statements
include statements about the potential benefits of products based
on Catalyst's engineered protease platform, and the superiority of
CB 2679d-GT over the Padua variant. Actual results or events could
differ materially from the plans, intentions, expectations and
projections disclosed in the forward-looking statements. Various
important factors could cause actual results or events to differ
materially, including, but not limited to, the risk that that
additional human trials will not replicate the results from earlier
trials, that potential adverse effects may arise from the testing
or use of the Company’s product candidates, including the
generation of neutralizing antibodies, the risk that clinical
trials and studies may be delayed as a result of COVID-19,
competitive products and other factors, that trials may not have
satisfactory outcomes, costs required to develop or manufacture the
Company's products will be higher than anticipated, including as a
result of delays in trial enrollment, development and manufacturing
resulting from COVID-19 and other factors, the risk that Biogen
will terminate Catalyst's agreement, competition and other risks
described in the "Risk Factors" section of the Company's Annual
Report on Form 10-K filed with the Securities and Exchange
Commission on March 4, 2021, and in other filings with
the Securities and Exchange Commission. The Company does not
assume any obligation to update any forward-looking statements,
except as required by law.
Contact:
Ana KaporCatalyst Biosciences, Inc.investors@catbio.com
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