DETAILED BUDGET FOR INITIAL BUDGET PERIOD DIRECT COSTS ONLY						FROM 01/01/05		THROUGH 12/31/05
PERSONNEL (Applicant organization only)			%		DOLLAR AMOUNT REQUESTED (omit cents)
NAME	ROLE ON PROJECT	TYPE APPT. (months)	EFFORT ON PROJ.	INST. BASE SALARY	SALARY REQUESTED		FRINGE BENEFITS		TOTAL
TBA (MD or Ph.D.)	FDA Sponsor, Principal Investigator, RAC Reviewer	FORMTEXT 12	50	120,000	60,000		19,200		79,200
TBA	Clinical Coordinator.	FORMTEXT 12	100	75,000	75,000		24,000		99,000
TBA	Molecular Biologist	12	100	55,000	55,000		17,600		72,600
TBA	Research Scientist	12	50	55,000	27,500		8,800		36,300
TBA	Technician	12	100	45,000	45,000		14,400		59,400
SUBTOTALS					262,500		84,000		346,500
CONSULTANT COSTS Biostatistician (100 hrs X $150.00 per hour)
									$ 15,000

 

EQUIPMENT  (Itemize)  FORMTEXT       Available
					FORMTEXT
SUPPLIES  (Itemize by category)					$ 400,000
Molecular biology, Cell culture, Cloning and Packaging supplies (DNA included)   Rodents Studies (350 total)   Primates Studies (20 total)
TRAVEL
PATIENT CARE COSTS	INPATIENT  (N/A)
	OUTPATIENT  (N/A)
OTHER EXPENSES  (Itemize by category)
FORMTEXT
SUBTOTAL DIRECT COSTS FOR INITIAL BUDGET PERIOD			763,500
CONSORTIUM/CONTRACTUAL		DIRECT COSTS
COSTS		FACILITIES AND ADMINISTRATIVE COSTS]
TOTAL DIRECT COSTS FOR INITIAL BUDGET PERIOD  (Item 7a, Face Page)			763,500
TOTAL DIRECT COSTS FOR 2 YEARS BUDGET $763,500  X 2 [ from 01/01/06 to 12/31/06]				$ 1,527,000

LETTER FROM DR. PAOLA LEONE, DIRECTOR OF CELL & GENE THERAPY AT UMDNJ / RWJ Medical School in New Jersey.

Dear Sherri,

I am sorry if I took so long to reply to you but I have been in-and-out of
town and awfully busy.

I would like to clarify that unfortunately my laboratory does not have any
ongoing research efforts for the gene transfer project for Tay Sachs mainly
because we do not have any funding for this project. The only funding
support that I received from the NTSAD nearly 2 yrs ago was to test the
efficacy of anti-inflammatory drugs on Tay Sachs and Sandhoff Diseases (i.e.
$45,000). This funding has now ran out.

The major two problems that we are facing with any clinical development for
Tay Sachs are time and funding. With the Canavan project the families joined
efforts several years ago and have raised 1.2 M. In 2 1/2 yrs we were able
to complete the preclinical studies and receive approval to go ahead.
However we did not have any funding to actually start the clinical study. We
applied for a NIH grant and we were able to start the clinical protocol only
after receiving approval for funding (i.e the awarded federal grant was 2.3
M for three years).

The clinical data collected in the Canavan patients that received gene
transfer are clearly showing that the therapy has much more effect in less
affected (younger) patients. In other words I want you to know that the gene
therapy does not do miracles but definitely has improved the quality of
life of the youngest patients.

I hope you are all well.
Warm regards,

Paola Leone